2023 Corporate
Responsibility
ReportGoing beyond every day.

TM

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

Dr. Michelle Fynan’s Story

Forward-Looking Statements and Other Important Legal Information

Dr. Michelle Fynan, diagnosed with Osteogenesis Imperfecta

This document and the materials or websites
cross-referenced contain statements that are
aspirational or reflective of our views about our
future performance that constitute “forwardlooking statements” within the meaning of thePrivate Securities Litigation Reform Act of 1995.
Forward-looking statements are generally
identified through the inclusion of words such as“aim,” “anticipate,” “aspire,” “believe,”commit,” “endeavor,” “estimate,” “expect,”“goal,” “intend,” “may,” “plan,” “seek,” “strive,”“target,” “will,” vision,” “mission,” “strategy,”“commitment” and “work,” or similar statements
or variations of such terms and other similar
expressions. The forward-looking statements inthis document and the materials or websites
cross-referenced concern Ultragenyx’s goals,
progress or expectations with respect tocorporate responsibility, sustainability, patients,
products, product candidates, employees,
environmental matters, policy and business risks
and opportunities. Forward-looking statements
inherently involve risks and uncertainties that
could cause actual results to differ materiallyfrom those predicted in such statements. Thesestatements are based on numerous assumptions
that we believe are reasonable but are open to awide range of uncertainties and business risks. In
addition, these statements may be based onstandards for measuring progress that are stilldeveloping, controls and processes that continue
to evolve, and assumptions that are subject to(OI) in childhood, exemplifies resilience by having overcome
numerous fractures and surgeries to become a

psychotherapist and an advocate, raising awareness andsupporting others with OI, including her own daughters.
Learn more about Dr. Fynan© 2024 Ultragenyx Pharmaceutical Inc

SASB Indexchange in the future. Consequently, actual results
may vary materially from what is contained in a
forward-looking statement. For a further
description of the risks and uncertainties that
could cause actual results to differ from thoseexpressed in these forward-looking statements, aswell as risks relating to the business of Ultragenyx
in general, see Ultragenyx’s Annual Report on
Form 10-K filed with the Securities and Exchange
Commission (SEC) on February 21, 2024, and its
subsequent periodic reports filed with the SEC.
Forward-looking statements are aspirational andare not guarantees or promises that goals or
targets will be met. Ultragenyx undertakes noobligation to update any forward-looking or otherstatements, whether as a result of new
information, future events, or otherwise, andnotwithstanding any historical practice of doing
so. Ultragenyx may determine to adjust any goals
and targets or establish new ones to reflectchanges in our business. The information
included in, and any issues identified as material
for purposes of, this document may not be
considered material for SEC reporting purposes.
In the context of this report, the term “material”
is distinct from, and should not be confused with,such term as defined for SEC reporting purposes.
Historical clinical trial success rates are notnecessarily predictive, and should not be
considered a guarantee, of future success rates.
Website references and hyperlinks throughout
this document are provided for convenience only,

GRI Index

2023 Corporate Responsibility Report

and the content on the referenced third-party
websites is not incorporated by reference into thisreport nor does it constitute a part of this report.
Ultragenyx assumes no liability for the content
contained on the referenced third-party references.
This document and the materials or websites
cross-referenced include certain statisticalinformation and estimates relating to rarediseases that are based on publications of
independent sources. Ultragenyx believes thesethird-party sources to be reputable but has notindependently verified the underlying data
sources, methodologies or assumptions. The
third-party sources referenced are generally
available to the public and were notcommissioned by Ultragenyx. Information that isbased on estimates, forecasts, projections, market
research or similar methodologies is inherently
subject to uncertainties, and actual events orcircumstances may differ materially from eventsand circumstances reflected in this information.

2

Letter From Our CEO

About Us

Corporate Responsibility Approach

About this Report

Ultragenyx Pharmaceutical Inc. (Ultragenyx) is a raredisease drug development company.
Ultragenyx Pharmaceutical Inc. (Ultragenyx) is headquartered in
Novato, California. We have offices and laboratories in 12 countries
across North America, Europe, Asia and Latin America. In the U.S.,
we have offices and/or laboratories in California, Florida,
Massachusetts, Texas and Utah.
Ultragenyx prepares an annual corporate responsibility report.
This 2023 report contains disclosures for the period January 1
through December 31, 2023, unless otherwise noted. Data may
be restated from previous years of reporting for several reasons (e.g.,
information has been updated or was not available at the time ofa previous report, improvements in data collection or methodology
or data errors). In the case of changes in data or information that
results in a material restatement, a note is provided with therestated data or information.
The scope of this report is Ultragenyx’s wholly-owned operations
globally. Third-party manufacturing is not included.

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Table of Contents
A Letter From Our CEO................ 4

People............................................... 46

About Us............................................. 7

Communities.................................. 66

Corporate Responsibility
Approach..........................................10

Planet.................................................77

Innovation .......................................14
Patients.............................................33

Governance.................................... 84
SASB Index.......................................97
GRI Index.......................................100

The term “employees” refers to our full-time employees, while
the term “workforce” is used to refer to the wider groups of people
working for and with us, including full- and part-time employees
and contingent staff.
©April 2024
Contact
We welcome your feedback. Please contact us at cr@ultragenyx.com
with your comments and suggestions about this report.

© 2024 Ultragenyx Pharmaceutical Inc

3Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

A Letter From
Our CEO

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

In 2023, we made significant progress on multiple oftreatments. Moreover, the efficiencies we have achieved in

We are particularly excited about potential treatments forof gene therapies. This reduction in cost is vital for making

our key programs in diseases with no approved therapies.
osteogenesis imperfecta (OI) and Angelman syndrome,
where we will have additional data to report later thisEmil Kakkis, M.D., Ph.D.

year. In OI (“brittle bone disease”), our antibody has been

Founder, President and CEO

strength in a Phase 2 clinical trial and this has resulted inshown to significantly reduce fractures and improve bonesome patients in our study moving from wheelchairs to

the manufacturing process are crucial in reducing the costthese therapies more accessible worldwide, especiallyin developing countries where such treatments can belife changing. Our goal is to set a new standard in genetherapy, making these advanced treatments more widely
available for diseases that haveno other treatment options.

Dear Stakeholders,

walking unaided. For Angelman syndrome, a developmental

I feel it is a privilege to work in rare disease, yet our job comes with a great

our antisense oligonucleotide demonstrate promisinggrappling with as innovative therapeutic approaches are

skills in affected children.

equity and accessibility. We’re at an unprecedented time in

We also opened our gene therapy manufacturing facility

an array of tools to identify and target previously intractabledeal of responsibility. We must do whatever we can, as quickly as we can, tohelp the many families and individuals struggling every day to get access tothe treatments they need. We’re approaching this in multiple ways — throughour own innovation, our initiatives to influence rare disease policy, our support

brain disorder, early Phase 1/2 clinical trial results withThis brings me to one of the big topics our industry has been

improvements in cognition, communication and motor

maturing and entering the market, and that is healthcare

in Bedford, Massachusetts in 2023. It is equipped tohandle both traditional Human Embryonic Kidney (HEK)

manufacturing and our innovative Pinnacle PCLTM (Producer

for the parents and foundations working to develop new therapies, and ourCell Line) platform, enabling us to produce a wide range ofcommitment to making our therapies accessible to all who can benefit.

production cycles annually increases our capacity to develop© 2024 Ultragenyx Pharmaceutical Inc

gene therapies. This facility’s unique ability to run multiple

drug development where scientific discoveries have given us
diseases. It is important that our healthcare systems support
getting effective treatments to the individuals and families

that will benefit from them, and this has long been a crucialpart of our mission at Ultragenyx.

4Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

2023 Corporate Responsibility ReportGRI Index

A Letter From Our CEO (cont.)
A key lever for this is rare disease policy. Developing

Another critical area to support health equity and access is

I’m proud of these efforts and others at the company“Generosity” is a core value at Ultragenyx. It is fundamental

for myriad reasons: patient populations are small, theof disease is imperative to patients receiving optimal care

communities where we live and work. In 2023, we continued

every day by the commitment and drive our team members

therapies in rare and ultrarare diseases is challenging
progression of these diseases is often slow and varies

from person to person and clinical symptoms are often

irreversible. We have been pushing for the U.S. Food andDrug Administration (FDA) to use the accelerated approval
pathway in those areas where the science for using

through improved diagnosis. Accurate and timely diagnosis
and it should be a responsibility of the healthcare system.

That said, one of the greatest challenges in rare disease isgetting diseases properly diagnosed. Delays in diagnosis

can result in patients receiving ineffective treatments andunnecessary testing, as well as the possibility of missing

to continue to do what’s right for patients and for the

to run our Rare Bootcamp™ for parents and other patientadvocates seeking to develop rare disease therapies, andwe launched an inaugural global “Days of Service” initiative
at Ultragenyx.

their opportunity for effective treatment altogether.

This year we also have worked to improve our disclosuresand in February 2024 we supported a workshop with the

Unfortunately, we live in an environment where payers often

chain, and business continuity, reflecting feedback received

Biomarkers to Support Rare Disease Regulatory Pathways.”

to a proper diagnosis in rare and ultrarare disease areas.

to clear and consistent regulatory guidance that supportsthat companies like ours step up to fill this gap throughand our Sustainalytics Environmental, Social and Governancethat individuals will receive an accurate, timely diagnosis

welcome our communities to continue to engage withbiomarkers is clear and may be the only route to developing
new drugs. I’ve written a number of editorials on this topic,
Reagan-Udall Foundation for the FDA entitled, “Qualifying

decline reimbursement of genetic testing, which is critical

We are hopeful that public forums like this will pave the way

This clearly needs to change, and meanwhile it is imperative

the use of accelerated approval for ultrarare diseases.

sponsored testing programs, which are often the only way
and treatment.

© 2024 Ultragenyx Pharmaceutical Inc

in areas such as quality, clinical trials, cybersecurity, supply

to our purpose as a rare disease company, and I’m inspired
have to give back. Together, we will continue to push

for progress and change to support rare disease drug

development for the industry and the communities ofpatients waiting for proper care.
Sincerely,

from our stakeholders. We’re pleased that our efforts arebeing recognized in our ratings. Last year, S&P ranked us 5th

in North America and 8th globally across the biotech industry

Emil Kakkis, M.D., Ph.D.

(ESG) risk rating improved to a “Low Risk” category. I

Founder, President and CEO

us on our corporate responsibility initiatives.

5

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

2023 Awards© 2024 Ultragenyx Pharmaceutical Inc

6Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

About Us
Vision: Lead the future of rare disease medicine
Mission: Transform the lives of people with rare disease© 2024 Ultragenyx Pharmaceutical Inc

7

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

One Ultra TeamUltragenyx Pharmaceutical Inc. (Ultragenyx) isa biopharmaceutical company headquartered in

Novato, California, that is committed to bringing
novel products to patients for the treatment ofrare and ultrarare diseases, with a focus on serious,

debilitating genetic diseases. Our purpose is to leadthe future of rare disease medicine as we seek totreat individuals afflicted by diseases with limited
or no treatment options. We recognize that theirlives and well-being are dependent upon our efforts
to develop new therapies. For this reason, we are

passionate about developing these therapies withthe utmost urgency and care.

© 2024 Ultragenyx Pharmaceutical Inc

8Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

2023 Corporate Responsibility ReportGRI Index

Four Approved Treatments for Five Rare Diseases

2023 UPDATES
Evkeeza:
Successfully filed reimbursement dossiers in 11 markets,with access achieved in 8 markets. Received marketing
approval in Canada and Japan, and an expanded

indication in children aged 5 years and older fromthe European Commission.
Crysvita:
Achieved marketing authorization in Argentina. Received

expanded reimbursement for pediatric patients in Mexico
and Brazil, with Brazil moving to full reimbursement.

© 2024 Ultragenyx Pharmaceutical Inc

9Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Corporate
Responsibility
Approach
Ultragenyx’s corporate responsibility approach, guided
by a materiality assessment, focuses on six key pillars
to prioritize efforts and resources towards initiatives
that significantly impact our business and stakeholders.

© 2024 Ultragenyx Pharmaceutical Inc

10Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Strategic Framework
Ultragenyx is committed to ethics, integrity andcorporate responsibility, integrating these principles

across all levels of our business. Our leadership sets a

powerful example, passionately focused on enhancing
the lives of individuals with rare diseases, empowering
our employees, and contributing positively to thecommunities we serve.
Innovation© 2024 Ultragenyx Pharmaceutical Inc

Guided by the principle of doing the right thing, we continue to strive forof our corporate responsibility strategy, starting with the identification of broad

to consistently consider the sustainability and social impact implications ofThis report covers a wide range of business practices within our company and is

excellence and improvement. This deliberate approach supports our efforts
our actions and decisions.

In 2021, we formalized our corporate responsibility program and based on amateriality assessment, we developed a strategic framework with six pillars:

Innovation, Patients, People, Communities, Planet and Governance. These
pillars help focus our efforts and our communications with both internal andexternal audiences, and allocation of our resources towards initiatives that support
the creation of long-term value and positive impact for our shareholders andsociety. They also inform the structure of this report and guide the development

Patients

PeopleCommunities

aspirations and objectives, highlighted at the beginning of each chapter.

meant to convey a clear and authentic story about Ultragenyx. We are committed
to engaging meaningfully with our stakeholders. We have ongoing engagementswith a wide range of internal and external stakeholders, which help inform anddrive various aspects of our corporate responsibility program from enhancing
disclosure strategies to improving information transparency.

This year, we have broadened our disclosures on several key topics, including

clinical trials, quality and safety, public policy participation, business continuity,
cybersecurity, and supply chain management.

PlanetGovernance11

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Materiality Assessment
In 2021, we conducted a materiality analysis to determine

our priorities. This process took into account both internaland external perspectives and focused on identifying the

topics that could have the greatest impact on our businessand that matter most to our stakeholders.

Material topics are listed below each pillar; bolded topics have been identified as higher priority© 2024 Ultragenyx Pharmaceutical Inc

12Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

United Nations (UN) Sustainable Development Goals (SDGs)
In 2015, the UN introduced 17 SDGs

as a “shared blueprint for peace andprosperity for people and the planet

now and into the future.” Ultragenyx’s
mission, vision and values align withseveral of the UN SDGs.

UN SDG

Contribution
Ultragenyx is focused on advancing health equity by developing treatments for rare and ultrarare diseases that improve the health and qualityof life of individuals living with rare disease, and by broadly supporting rare disease communities.
We advocate for broad access to screening and treatment and strive to make our treatments accessible and affordable to as many patients
as we can.
We are also committed to advancing employee health, safety and wellness. We continue to provide our employees with wellness offeringsand comprehensive benefits to support their financial, familial, physical and mental health.
Ultragenyx provides the majority of its grants and philanthropic funding to organizations and initiatives that contribute meaningfully
to the health and well-being of local and at-risk communities, including our rare disease communities.
Ultragenyx promotes rare disease education and awareness among healthcare professionals and the rare disease community, and offers
employee education and training to support career advancement for our employees. We are also committed to expanding access to Science,
Technology, Engineering, Arts, and Mathematics (STEAM) education opportunities, particularly in economically disadvantaged communities.

Ultragenyx promotes gender equality in the workplace by fostering diversity, equity, inclusion and belonging (DEIB) at all levels of theorganization and is committed to equitable compensation practices.

© 2024 Ultragenyx Pharmaceutical Inc

13

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility ReportInnovation

Pioneering new approaches to drug development
for rare and ultrarare diseases.

We are committed to delivering novel,
disease-modifying treatments with speed
and urgency to rare disease communities
with limited or no treatment options.

© 2024 Ultragenyx Pharmaceutical Inc

14

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Aspiration
To optimize and acceleraterare disease drug researchand development (R&D),

Our Objectives

2023 Progress

Develop industry-leading clinical pipeline in rare andultrarare diseases that have limited or no treatment options• Initiated Phase 3 Cosmic study in patients aged 2 to <5 years and Phase 3 portion of Phase 2/3 Orbit study in patients
aged 5 to <26 years for UX143 in osteogenesis imperfecta (OI)
• Completed enrollment in Phase 1/2 study evaluating GTX-102 for Angelman syndrome

whether by us or others.

• Completed dosing of all patients in the Stage 1 portion of the seamless Cyprus2+ Phase 1/2/3 study evaluating UX701
in Wilson disease• Presented positive data from the pivotal Transpher A and long-term follow-up studies of UX111 for Sanfilippo
syndrome type A (MPS IIIA)• Completed enrollment in Phase 3 GlucoGene study of DTX401 for glycogen storage disease type Ia (GSDIa)
Foster industry-wide and community funded development
efforts in rare and ultrarare diseases• Hosted two Rare Bootcamps with 40 participants representing more than 30 organizations in attendance• Contributed to collaborative drug development by participating in multiple industry consortia, including theAngelman Biomarker and Outcome Measure Consortium (ABOM) and the Accelerating Medicines Partnership (AMP)
Bespoke Gene Therapy Consortium, among others• Supported more than 50 investigator-sponsored trials (ISTs) globally, fostering community and investigator-led
research initiatives• Opened a state-of-the-art Gene Therapy Manufacturing Facility (GTMF) in Bedford, Massachusetts© 2024 Ultragenyx Pharmaceutical Inc

15

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

R&D
Innovation refers to our processes,

initiatives, activities and investments
aimed at the efficient and rapid
advancement of investigational

therapies that maximize patient

health outcomes. Our patient-focused
drug development model and crossindustry collaborations support our
mission to transform the lives ofpeople with rare disease.

We believe innovation in healthcare is critical for the many

Our research aims to advance a new program into clinical development every one to two years. At the end of 2023, we had

approved treatment. We make significant investments in R&D

in clinical trials.

individuals living with rare diseases who are waiting for an

to innovate and develop new therapies. In 2023, we spent

four rare disease treatments approved by the U.S. Food and Drug Administration (FDA) in five indications and six programs

approximately 65% of our operating expenses on R&D.
We rely on patent protection, trade secrets, know-how

and continuing innovation to develop and maintain ourcompetitive position. Our policy is not to tolerate any unlawful
use or activity that violates the intellectual property rights
of others, as highlighted in our Global Code of Conduct.

We also expect suppliers with which we conduct businessto respect the intellectual property rights of others. We

seek patent protection in the U.S. and internationally forour products, investigational therapies and processes. Ourpolicy is to patent or in-license the technologies, inventions
and improvements that we consider important to thedevelopment of our business. As of December 31, 2023, weown, jointly own or have exclusive rights to more than 250
issued and in-force patents and more than 350 pending

patent applications, more than 50 of which are in the U.S.

Total R&D spending decreased by $57.3 million, reflecting changes in various program costs, including adecrease in upfront license, acquisition and milestone fees, and shifts in spending across gene therapy, biologic,
nucleic acid programs and approved products. For more details, please see our 2023 Annual Report.

© 2024 Ultragenyx Pharmaceutical Inc

16

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Patient-Focused Drug Development
More than 90% of rare diseases do

not have available treatments, so we
dedicate ourselves every day to thegoal of developing and delivering

new therapies with urgency. Three of

our four approved medicines are theonly FDA-approved therapy for theirrespective diseases.

Ultragenyx’s Dynamic Development Model (DDM) supports

effective decision-making in rare disease drug development

through a patient-focused approach that involves collecting
direct information and insights from patients. We engage
in various activities to help align our programs with theunmet medical needs and expectations of the rare diseasecommunity. These activities include informal and structured

patient interviews, clinical survey studies, disease monitoring
programs, natural history studies and patient engagement
plans (PEPs). Engaging early with patients and theircaregivers provides important insights that inform clinical

trial design, endpoint selection and treatment expectations,potentially resulting in clinically meaningful endpoints that

improve the lives of individuals living with rare diseases. The

DDM strategy also encourages inventing new approaches andhaving backup plans to help address unexpected challenges.
For more information on how we partner with the raredisease community and leverage insights, see Patient
Advocacy and Engagement.

~70% historical success rate to datein developing therapies from clinicalstudy initiation to receiving commercial
approval from regulatory authorities© 2024 Ultragenyx Pharmaceutical Inc

17

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

2023 Corporate Responsibility ReportGRI Index

Advancing Gene Therapy for Rare Diseases
In 2023, we opened our state-of-

the-art 112,500-square-foot Gene
Therapy Manufacturing Facility(GTMF) in Bedford, Massachusetts,providing us with end-to-end genetherapy R&D and manufacturing
capabilities. This fit-for-purpose

facility can enable future process

innovation and multi-modal process
configurations, supporting our goalof delivering new treatments topatients as quickly as possible.
A family affected by a rare disease, alongside Ultragenyx leadership and Middlesex
County representatives, officially unveiled the new GTMF in June 2023.

© 2024 Ultragenyx Pharmaceutical Inc

The decision to build our own GTMF was driven by astrategic goal to enhance flexibility and control over

our processes. Our Pinnacle PCLTM (Producer Cell Line)platform is a proprietary manufacturing system that isdesigned to produce adeno-associated virus (AAV) vectors
more efficiently and cost-effectively than traditional
methods to meet the increasing demand in today’s

gene therapy market. This high-titer, commercial-grade
platform is designed to enable us to address large andextrahepatic indications with greater efficiency and lower
manufacturing costs.

Our goals with the GTMF are to support the optimizationof our Pinnacle PCL platform and expand our opportunities
for future partnerships, maintain a stable and securesupply of gene therapies, and provide the agility needed

to support our gene therapy pipeline. Given it enables theswift transitions between products, we expect to be able toaccelerate the delivery of treatments to patients who needthem urgently.

18

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

A Fusion of Innovation and Flexibility
The GTMF transcends the traditional role of a manufacturing site; it is a hub

where pioneering research converges with cutting-edge technology. The facility’s
versatile multi-product design, complemented by 50L, 250L, 2x500L and

2000L single-use bioreactors, marks a significant advancement in gene therapy
production, emphasizing flexibility, efficiency and scalability.

The facility is equipped with cGMP-compliant spaces for both drug substance(DS) and drug product (DP) manufacturing, as well as warehouse and officespaces. Its design is both flexible and purpose-driven, employing single-use
mobile equipment and versatile utility panels to support ongoing process
innovation and enable various configuration modes.

2023 Corporate Responsibility Report

Overcoming Challenges Together
During COVID-19
The GTMF was completed both on time and under budget,
despite the unique challenges posed by the COVID-19

pandemic. Our collaborative culture and integrated projectdelivery method helped us navigate these challenges

effectively by taking strategic pauses to enable right-firsttime solutions. Our team’s emphasis on safety, problem-

solving and a no-blame culture highlights our commitment
to not just building a facility but fostering a community
dedicated to transforming lives.

Moreover, the facility was designed to maximize both efficiency andeffectiveness in our manufacturing operations. Having both DS and DP

manufacturing under one roof is expected to streamline our production
process, especially for small batches.

In addition, in the Greater Boston area, we operate a 500L production scale

Pilot Plant and advanced GMP QC laboratories. These facilities, located near theGTMF, support our fully integrated end-to-end gene therapy R&D capabilities
and position us at the forefront of industry developments in gene therapy.

As part of our strategic initiatives outlined in

our quality 5-year strategy planning, we haveestablished a state-of-the-art gene therapy quality
control (QC) laboratory. This lab represents asignificant investment in our goal of achieving thehighest quality of our gene therapy research. The

QC lab is equipped with advanced technologies for
rigorous testing and validation of gene therapies,aligning with our focus on quality and safety.

© 2024 Ultragenyx Pharmaceutical Inc

19

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Our Pipeline
Our approved therapies and clinical-stage pipeline consist of fourmodalities: biologics, small molecules, gene therapies and nucleic

acid therapies (antisense oligonucleotide [ASO] and messenger RNA[mRNA]). We have a broad translational research effort that works toturn observations in the laboratory and clinic into interventions that

improve the health of individuals with rare and ultrarare diseases. We
are advancing clinical and preclinical development programs acrossmultiple rare disease therapeutic areas. Currently, we are developing
six investigational therapies in pivotal clinical programs with thepotential to reach more than 150,000 patients. In addition to ourclinical efforts, we are actively working on advancing a number ofpreclinical programs, including UX055 for CDKL5 Deficiency Disorder(CDD) and UX810 for Duchenne Muscular Dystrophy (DMD).

© 2024 Ultragenyx Pharmaceutical Inc

20

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Update to Our Pipeline

UX143 (setrusumab) for
osteogenesis imperfecta (OI)

In July 2023, we initiated two late-stage clinical trials on setrusumab for OI types I, III, and IV. The Phase 3 part of the Phase 2/3 Orbit study assesses setrusumab’s impact on fracture rates versus
placebo in patients aged 5 to <26. The Phase 3 Cosmic study, an active-controlled trial, compares setrusumab to IV bisphosphonates in patients aged 2 to <5, focusing on total fracture rates. At theAmerican Society for Bone and Mineral Research (ASBMR) 2023 annual meeting, we presented interim Phase 2 Orbit study data that showed a significant (~67%) fracture rate reduction and bonedensity improvements after 6 months, with no serious adverse events reported as of the data cut-off date. UX143 has gained Rare Pediatric Disease and Orphan Drug designations in the U.S., and
PRIME and Orphan product designations in the EU.

GTX-102 for Angelman
syndrome

The Phase 1/2 study evaluating the safety and tolerability of GTX-102 in pediatric patients with Angelman syndrome completed enrollment in December 2023. Interim data presented in April 2024
demonstrated clinically meaningful improvements across multiple clinical domains, compared to natural history data, where available. GTX-102 has received Rare Pediatric Disease, Orphan Drug,and Fast Track designations in the U.S., plus PRIME designation in the EU.

UX701 for Wilson disease

The seamless Cyprus2+ Phase 1/2/3 study of UX701 for Wilson disease completed dosing the three dose-escalation cohorts of Stage 1 in January 2024 and is evaluating safety, efficacy, and dose selection forthe pivotal, randomized, placebo-controlled Stage 2. Initial data showed four out of five patients in the lowest-dose cohort experienced reductions in urinary copper and were tapering off chelators and/or
zinc therapy, with no serious adverse events reported as of the data cut-off date. UX701 has received Orphan Drug and Fast Track designations in the U.S., and Orphan medicinal product designation in the EU.

UX111 for Sanfilippo syndrome
type A (MPS IIIA)

Positive data from the pivotal Transpher A study, which is evaluating the efficacy and safety of UX111 in children with Sanfilippo syndrome type A, along with results from long-term follow-up studies,were presented at the WORLDSymposium 2024 20th annual research meeting. These findings showed rapid and sustained decreased levels of heparan sulfate (HS) levels in cerebrospinal fluid (CSF),
correlating with improved long-term cognitive development. UX111 has received Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Rare Pediatric Disease, and Orphan Drug designationsin the U.S., and PRIME and Orphan medicinal product designations in the EU.

DTX401 for glycogen storage
disease type Ia (GSDIa)

The Phase 3 GlucoGene study completed enrollment in May 2023. The 48-week study is evaluating patients aged 8 years and older randomized 1:1 to DTX401 or placebo. The primary endpoint is the
reduction in oral glucose replacement with cornstarch while maintaining glucose control. DTX401 has received Orphan Drug designation, Regenerative Medicine Advanced Therapy (RMAT) designation
and Fast Track designation in the U.S., and PRIME and Orphan medicinal product designations in the EU.

DTX301 for ornithinetranscarbamylase (OTC)
deficiency

The Phase 3 Enh3ance study is expected to complete enrollment in the first half of 2024. The pivotal, 64-week study will include approximately 50 patients, randomized 1:1 to DTX301 or placebo.
The primary endpoints are response as measured by removal of ammonia-scavenger medications and protein-restricted diet and change in 24-hour ammonia levels. DTX301 has been granted
Orphan Drug designation and Fast Track designation in the U.S., and Orphan medicinal product designation in the EU.

© 2024 Ultragenyx Pharmaceutical Inc

21

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

2023 Corporate Responsibility ReportGRI Index

Clinical Trials
Prior to receiving marketing approval

Regular monitoring of patient safety throughout our trials

Every clinical trial site is subject to a thorough vetting

profile for our investigational therapies. To support clinicalregulatory compliance. During the trials, routine monitoring

aims to prevent harm and maintain a positive benefit-riskfrom regulatory authorities for our

trial efforts, we partner with contract research organizations

investigational therapies, we must

(CROs) and other vendors, requiring them to undergo aevaluate and demonstrate their safety

vendor qualification audit, adhere to our policies as well

as applicable laws and regulations, and participate in ourand efficacy in clinical trials.

monitoring oversight and auditing program to sustain

compliance and alignment with our standards. By working
closely with our partners, we aim to confirm that study
We are committed to conducting trials in an ethical mannerand to adhering to our internal procedures and policies, theInternational Council for Harmonisation (ICH) of TechnicalRequirements for Pharmaceuticals for Human Use E6

Guideline for Good Clinical Practice (GCP), and applicablenational and local regulations for trial design and conduct.

We seek to protect patient safety and well-being by adhering
to appropriate informed consent procedures and good

policies and procedures, and that participants’ rights, safety
and well-being are upheld, in accordance with ethical andregulatory standards.

For more information on the risks related to the discovery
and development of our investigational therapies, pleasesee our 2023 Annual Report.

clinical practices. We work to maintain clinical trial safety

Clinical Site Assessment and Compliancesystems. We also document and report relevant safety

monitoring is essential to our goal for safeguarding theat each study site is conducted with the goal of confirming
adherence to protocols and Good Clinical Practice (GCP)

standards, while also focusing on site quality and the safety
and rights of patients.

Our quality team is responsible for conducting independentsite audits for each study, based on a risk-based approach.
Through our GCP audit program, we evaluate each site
for compliance risks. The frequency of these audits isadjusted according to the specific risk factors identified.
We consistently monitor for any deviations from GCP

standards, misconduct or violations of patient rights or safety.

In cases where such issues are identified, we require corrective
measures to be implemented, conduct severity impact

assessments and, if necessary, report these findings to the

FDA and comparable regulatory authorities in other countries.

through controlled processes, policies and management

Our approach to clinical site selection and routine site

In 2023

information and adverse events (AEs), such as any new orsafety and reliability of our clinical operations. Before

we had more than 130 clinical trialsites in operation across 19 countries.

worsening conditions, to the relevant regulatory authorities
and participating investigators.

© 2024 Ultragenyx Pharmaceutical Inc

conduct aligns with the clinical trial protocol, our internal

process designed to confirm both scientific quality andbeginning operations at any clinical site, we carefully

assess available resources, clinical trial experience andthe suitability of its facilities, staff and equipment.

22

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Clinical Trials (cont.)
Data Transparency
Data transparency is essential to fostering trust with patients,

We endeavor to make clinical trial information and results

for legitimate medical/scientific research purposes fromresearchers. We recognize this importance and support the

proprietary information and patient privacy. Ultragenyx

care and helping advance medical science.

healthcare professionals, regulatory agencies and medical
overall principles of greater clinical trial data transparency
as part of our patient-focused drug development model.

public in a timely manner while protecting essential

registers protocol information for company-sponsoredclinical trials of investigational therapies and marketed

We follow the standards and principles for clinical trial

medicines in accordance with applicable laws anddata transparency set forth by international industry

regulations. In the U.S., protocol information is registeredorganizations such as:

at www.clinicaltrials.gov.

• International Federation of Pharmaceutical
Manufacturers & Associations (IFPMA)

Ultragenyx discloses the results of company-sponsoredclinical trials in accordance with applicable laws and• European Federation of Pharmaceutical Industries
and Associations (EFPIA)

regulations. We also seek to publish results – regardlessof outcome – in peer-reviewed journals or at medical• Japan Pharmaceutical Manufacturers Association (JPMA)

and scientific meetings. We publish the results of both• Pharmaceutical Research and Manufacturersof America (PhRMA)

Our medical writing follows industry standards, such as the

Additionally, Ultragenyx is a member of the Biotechnology
Innovation Organization (BIO) and follows the BIO Position
Statement on Clinical Trial Registries and Disseminationof Clinical Trial Results.

successful and failed trials to advance scientific learning.

Good Publication Practice guidelines (GPP3) published by theInternational Society for Medical Publication Professionals,
and Recommendations for the Conduct, Reporting, Editing
and Publication of Scholarly work in Medical Journals

published by the International Committee of Medical Journal
Editors (ICMJE). We also prepare plain language summaries

of results to share with patients and the public. Additionally,

we make reasonable efforts to address clinical data requests© 2024 Ultragenyx Pharmaceutical Inc

qualified researchers in the interest of improving patient
These collective efforts help patients access trial result

In 2023
Registered 2 new protocols on
www.clinicaltrials.gov

information and facilitate compliant data sharing with

Published 11 peer-reviewed manuscripts

Trial Transparency Commitment and Ultragenyx Clinical

Submitted 99 abstracts to congresses

interested parties. For more information, see Our Clinical
Trial Results.

Data Protection, Anonymization and Security
Ultragenyx is committed to high standards of integrity andMade 135 oral and poster presentations
at medical and scientific meetingscompliance with applicable laws and regulations whenhandling patient data. Clinical trial data that is transferred to
Ultragenyx is required to be anonymized, meaning no names
or other personally identifiable information (PII) is provided.
Trial participants must be informed if Ultragenyx examines
their medical records. We require vendors supporting ourtrials to comply with applicable data protection laws and tohave a data breach response plan in place. These measures
are some of the ways that Ultragenyx works to protect theprivacy and rights of our clinical trial participants.

See Data Privacy in this report for more information.

23

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

2023 Corporate Responsibility ReportGRI Index

Diversity in Clinical Trials
We are committed to ethical,

responsible and inclusive clinicalresearch. We advocate and lobby forpeople living with a rare disease toreceive an accurate diagnosis, quality
care and available therapy as quickly
as possible.

Individuals and families impacted by rare and ultrarare

to identify potential participants, and we have clinical trial

and accessing optimal care, particularly when there is

We endeavor to make our clinical trials inclusive and diverse

diseases face challenges in securing accurate diagnoses
no approved therapy for a particular condition. This isfurther compounded by the fact that many physicianslack experience with these diseases and are often unawareof the options available for enrolling in clinical trials.

Given these challenges, we design our clinical trials withthe goal of maximizing diversity and heterogeneity in ourstudy populations. We strive to limit exclusions as much as
possible, weighing the benefit/risk balance in an effort topromote broad inclusivity.

rare and ultrarare diseases, regardless of gender, ethnicity
or socioeconomic status. As many of the diseases we are

studying have no approved treatments, we strive to makethese opportunities accessible to a broad range of patients.

To help minimize the financial burden on patients, we cover
expenses necessary for clinical trial participation, including

travel. We also prioritize reducing the burden of participation

research conducted within our sector and that building andseveral key measures in our endeavor to foster greater

rebuilding trust with these groups is a critical step. This lackof representation may compromise a research program’s

effectiveness. Emphasizing inclusive designs in clinical trials
is a crucial step toward enhancing both the quality andfairness of treatments for the populations most impacted.
As we continue to monitor and strive to improveparticipants’ diversity metrics within the U.S., our global
across diverse regions. Our dedicated Patient Find teamworks with clinical operations and development teams• Regularly collecting feedback from participants
for continued improvement
We also provide support for investigator-sponsored trials(ISTs) for our investigational or approved products worldwide.
We encourage proposals that align with our scientific areasof interest and include integrated evidence plans for our
products and programs.

through home visits and phone calls when possible.

Building on our global initiatives and the insights gained

expansion further amplifies our commitment to inclusivity© 2024 Ultragenyx Pharmaceutical Inc– available in multiple countries to individuals living with

We recognize that a significant number of racial and ethnic

groups in the U.S. are not adequately represented in clinicalMore than 50 clinical ISTs
approved and 495+ clinicalparticipants enrolled
globally since 2012

sites in both developed and developing countries.

• Investing in employee training programs for cultural
sensitivity and Diversity, Equity, Inclusion and Belonging(DEIB) to enhance our employees’ ability to engage
effectively with diverse populations

from diverse patient populations, we have implemented
inclusivity and diversity in our clinical trials. Examplesof these measures include:• Preparing multilingual materials to educate patientson each clinical trial• Utilizing additional techniques to further outreach topatient communities, including social media and otherdigital marketing channels• Engaging and building partnerships with the raredisease community through advisory panels toadequately represent their experiences and needs

Looking ahead to 2024, we are focusing on

supporting economically and racially marginalized

populations in entering the sciences. To that end, weare initiating a fellowship program that aims to bringin research fellows, to conduct, lead and publish

research with a particular emphasis on attracting
a diverse pool of candidates. This program is a

step toward broadening our impact and fostering agreater diversity of physicians, and benefits from awide range of perspectives and expertise.

24

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Disease Monitoring Programs and Post-Marketing Commitments
We utilize disease monitoring

programs (DMPs) to evaluate long-term outcomes for newly approved

therapies, facilitate knowledge sharing
with the rare disease community, andfulfill any post-marketing regulatory
requirements.

Disease registries and other post-marketing studies canprovide an organized way to collect patient data and allow forpost-marketing surveillance of approved medicines. However,rare disease registries are costly, may have incomplete or

missing data, and seldom provide compelling publishable

data, due to small patient numbers and/or patient attrition.
Traditional registries often fail to provide patients with theirown collected data, losing an opportunity to share useful

information for medical care with patients and physicians.
To address these challenges, Ultragenyx developed thenovel concept of a DMP. This is a global study that assembles
regulatory-quality data on a broad population of individuals

DMPs can also support the generation and transparent

access to a medicine – or not. DMPs go beyond clinicalcompliant data with patients, physicians, sponsors and theliving with a rare disease, whether treated – via commercial

trials by enhancing the understanding of the disease and its

treatments for the benefit of patients, physicians, payers andthe company. DMPs provide progress reports, broader patient
population data analysis, high-quality disease information,and supportive long-term outcome data, and promote
therapy science advancement and research.

As part of a DMP, the pharmaceutical sponsor may partnerwith an academic institution and a patient advocacy group,
if applicable, on the ownership and management of data.

© 2024 Ultragenyx Pharmaceutical Inc

sharing of high-quality, Good Clinical Practice (GCP)-

rare disease community. We provide participants in our DMPs
with the opportunity to receive their own data in easy-tounderstand language.

To date, we have initiated eight DMPs, including several for
Crysvita (burosumab-twza) for the treatment of X-linked

DMP Highlights
First DMP launched in 2012
1,380+ patients enrolled*
at nearly 57 sites in 12 countries*Enrollment as of December 31, 2023

hypophosphatemia (XLH). Our in-clinic DMP for XLH is ourlargest study, with more than 780 patients in its sixth year.

In 2022, we enrolled the first patient in our most recent DMP,
for Dojolvi (triheptanoin).

25

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

2023 Corporate Responsibility ReportGRI Index

Quality
Our commitment is to deliver

quality medicines to the rare diseasecommunity. We strive to upholdan engaged quality culture thatemphasizes the safety, efficacy andreliable quality of our medicines.

We have implemented a company-wide quality program
to manage product and safety risks, with the goal offull compliance with applicable laws, regulations andinternational standards. Our quality program, built on afoundation of safety, efficacy, product quality and network

reliability, embodies our core values in research, development
and manufacturing. This program is spearheaded by our chiefContinued improvement is a cornerstone of our QMS,

• Quality Culture
We combine a leadership-driven quality culture witha strong focus on safety and efficacy.

It involves systematic assessment, control, communication

quality operations officer. Our leadership is committed to

Ultragenyx works to adhere to “good practice” quality

quality management system (QMS). This system is designed

collectively referred to as GxP. This extends to our suppliers

all levels of operations and across all stages of development,

framework. We require our suppliers to comply with thesystem continues to evolve to meet business and quality

update our practices to align with industry trends andOur approach to quality is centered around three focus

Our QMS integrates people, processes and systems. It is afostering a responsible quality mindset, supported by a robust

guidelines, regulations and international standards,

to promote sound science and appropriate behaviors acrossand business partners, facilitating a unified compliancemanufacturing and distribution of our medicines. Our quality

Drug Supply Chain Security Act (DSCSA). We continue torequirements, as well as growth and scaling needs.

regulatory expectations.

areas that guide our performance:• Regulatory Compliance and Data-Driven Innovation
We design our programs to adhere to regulatory
standards and strive to use data to drive continued
quality improvement.

© 2024 Ultragenyx Pharmaceutical Inc• Integrated Quality and Risk Management
We are committed to integrating quality
throughout our operations and employ proactive
risk management strategies.

principles-based framework designed for quality assurance,
continuous improvement and compliance. The system

includes organizational structure, responsibilities, procedures

which evolves to meet business growth, scale and quality

requirements. Quality risk management (QRM) is an integral
part of our QMS, extending beyond ICH Q9 guidelines.
and review of risks throughout the product lifecycle.

This approach is intended to foster a risk-curious mindset
across the company, encouraging a proactive stancetoward risk management and to facilitate a cultureof learning and growth.

We aspire to become an industry leader in cultivating aquality learning culture, going beyond traditional training
methods. We focus on learner success and development

and strive to continue enhancing our learning modules andmaterials. This approach is key to our goal of embedding
a quality mindset across the company, promoting open
communication, employee ownership and data-driven
performance monitoring.

and resources, detailed in our Quality Manual, Standards and
SOPs, which are subject to regular review.

26

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Quality in Supply Chain
To maintain high standards, we havea robust supplier oversight program.
This program includes risk-based
auditing and monitoring of our
supply chain partners, with thegoal of ensuring compliance withregulatory and internal requirements.

Our third-party audits, essential for product quality andsafety, involve reviewing records, conducting interviews,
analyzing third-party reports such as RX-360 of The

International Pharmaceutical Supply Chain Consortium,and performing onsite inspections. In 2023, we licensed 6

RX-360 audit reports to satisfy the audit requirements for 6

supplier sites (3 in the U.S. and 3 international). Additionally,
we assess compliance with standards, contract adherence,and quality benchmarks. This proactive approach isdesigned to help us identify improvement areas anduphold our supply chain’s integrity.

Our Corrective and Preventative Actions (CAPA)Management System is central to our QMS, and we takea risk-based approach to managing incidents and quality
issues internally or with contract manufacturingorganizations (CMOs). We focus on identifying root causesof quality events, with the goal of implementing effective
corrective and preventive measures to avoid recurrence.

Counterfeit Products
We take the safety and effectiveness of our medicines

seriously and have implemented processes designed to

identify and address potential or known risks associatedwith counterfeit products. Our field action procedureallows for cross-functional collaboration to address

counterfeit or quality issues and communicate with

In 2023
Initiated over 100 GxP audits with plansto address all findings
Zero product recalls; no units recalledstakeholders as necessary.

In our endeavor to further mitigate the risk of counterfeit

products entering our supply chain, we have implemented

security features, such as tamper-evident seals and serializationof product labeling. Each commercial drug product is tracked
through a serialization process, which assigns a unique

identifier to each package. This is designed to allow ourpartners in the distribution network to verify that a given

package is a legitimate product of our company, providing

added protection to patients against the risk of counterfeiting.

We continue to assess and advance the maturity of ourOur commitment to transparency and proactive measuresenhancing training, with a cross-functional Community

products is aimed at maintaining the trust and confidence

CAPA practices, aimed at optimizing our systems andto identify and address risks associated with counterfeitof Practice driving these improvements.

of our stakeholders in the safety and effectiveness of our
products. We expect to continue to implement rigorous

procedures and security features designed to ensure theauthenticity and quality of our products© 2024 Ultragenyx Pharmaceutical Inc

27

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

SafetyOur reputation is dependent

on the trust that patients, healthcare
professionals, regulatory authorities
and the general public place in us.

Our reputation is dependent on the trust that patients,

Our pharmacovigilance (PV) system, supported by ourgeneral public place in us. We work to uphold this trust byprocess and evaluate available safety information for ourhealthcare professionals, regulatory authorities and thequality system, is designed to efficiently and accurately

striving to comply with all applicable laws, regulations andproducts to confirm the benefits outweigh the risks. Wheninternational standards, such as International Conference

there is a change that impacts the benefit/risk profile, weon Harmonization (ICH), Good Clinical Practices (GCP), Goodare committed to promptly communicating with regulatory

Distribution Practices (GDP), Good Laboratory Practices

authorities, patients and healthcare providers. Our diligent(GLP), Good Manufacturing Practices (GMP) and Goodapproach to patient safety and swift communication of

Pharmacovigilance Practices (GVP), collectively known as GxP,

safety data that informs benefit-risk of our medicines aims

with all Ultragenyx employees receiving annual GxP training.

to empower patients and healthcare providers to makeAt the heart of our mission to be the leader in rare disease

This information comes from many potential sources.

informed treatment decisions, consistent with our goal ofthrough a pharmacovigilance (PV) system designed with

• Clinical trials

All Ultragenyx workforce, representatives or agents working

medicine is our commitment to patient safety, upheld

comprehensive, inspection ready systems and procedures
designed to be in compliance with laws, regulations,standards and industry best practices. Our Global Drug

Safety & Pharmacovigilance (DSPV) department maintains
a robust medical and safety surveillance.

Safety information is received, collated and analyzed

throughout a product’s lifecycle, from early development
phase and throughout the post-approval phase.

These include:• Disease monitoring programs• Patient access programs such as compassionate andnamed patient use• Patients and their families and caregivers• Healthcare providers• Scientific literature• Regulatory agencies and their databases• Social media• Vendors or business partners with whom we have legal
agreements that include requirements to report relevant

contributing to safer and healthier lives.

on behalf of Ultragenyx play an important role in promoting

the safety of our products. Web-based training is required of
our workforce on how to report safety information including
AEs and product quality complaints (PQCs) associated withany Ultragenyx medicine.

Recognizing the value of proactive pharmacovigilance,

we assess industry standards and best practices. Throughcontinual learning from our data and the broader researchcommunity, we strive to enhance patient safety and upholdour commitment to public trust.

safety information they become aware of© 2024 Ultragenyx Pharmaceutical Inc

28

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Strategic Collaborations
We are working to develop a robustand diverse pipeline of investigational
therapies to support our mission
to transform the lives of people
with rare disease.

In addition to our translational research efforts, we have apartnership strategy that combines Ultragenyx’s expertiseand know-how in developing and commercializing rare andultrarare therapeutics with R&D efforts at other companies
and institutions. Ultragenyx seeks partners aligned withthe company’s core values and vision for developing andbringing transformative therapies to those living with rare
and ultrarare diseases worldwide. Our flexible partneringmodel is intended to allow us to collaborate broadly withacademics and biopharma industry partners in developing
rare disease therapeutics or platform technologies.

Ultragenyx has a successful history of partnering withother biotech companies to develop and commercialize

rare disease therapeutics. Since 2013, Ultragenyx and Kyowa
Kirin have been partners in the successful development

and worldwide commercialization of Crysvita across two

indications. Ultragenyx also has license and collaborationagreements with Mereo BioPharma on the developmentof setrusumab globally and with Regeneron to clinicallydevelop, commercialize and distribute Evkeeza (evinacumab)
in countries outside of the U.S.

Please see our 2023 Annual Report and our website
for a list of our collaborations.

© 2024 Ultragenyx Pharmaceutical Inc

29

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Research Collaborations
We participate in multiple industry

consortia and partnerships to fosterand support collaborative, industrywide drug development in rare andultrarare diseases.

We’re a member of the Angelman Biomarker and Outcome

We’re a member of the LouLou Foundation CDKL5

partner in the development of UX143 (setrusumab) for the

sharing, agree on the most important disease domains

Clinical Assessment of NeuroDevelopmental measures In

from the IMPACT Survey, “The patient clinical journey

Measure Consortium (ABOM), launched to drive informationin Angelman syndrome, and develop or modify existing

endpoints or biomarkers in order to know how to measurechange in clinical studies.

We’re a founding sponsor of BeginNGSTM (newborn genomic

sequencing), a public-private coalition led by Rady Children’s

CDD (CANDID), a three-year observational study for thedevelopment of disease-modifying therapeutics for CDD.

The CANDID study is anticipated to enroll more than 100

participants, with sites in the U.S., Canada, U.K., Germany,potential treatment of OI. The first article based on dataand socioeconomic impact of osteogenesis imperfecta:a systematic scoping review,” was published in Orphanet
Journal of Rare Diseases in February 2023.

France, Spain, Italy and UAE. CANDID study results will be

We’re a partner in the n-Lorem Foundation, whose mission

design and inform therapeutic development for related

oligonucleotide (ASO) medicines for diseases that affectshared with the entire community to aid CDD clinical trial

is to discover, develop and provide experimental antisense

neurodevelopmental disorders.

nano-rare patients (one to 30 patients worldwide) for free,conduct genomic screening on 3.7 million newborns in the

We’re a supporter of the “Living with Osteogenesis

into n-Lorem’s drug development program, six patients

information on our support of newborn screening (NBS).

Community Insight and Experience” (IMPACT) Survey,

Institute that is piloting a program to use genetic testing

technology to screen newborns for rare genetic diseases.
The ultimate goal is to test for up to 1,000 disorders andU.S. annually. See Public Policy Participation for more

We’re a member of the Accelerating Medicines Partnership®

(AMP®) Bespoke Gene Therapy Consortium, a public-private
partnership with the National Institutes of Health (NIH), the

FDA, and multiple public and private organizations to createa standardized approach to help reduce upfront costs andlower barriers to developing new gene therapies for rare andultrarare diseases.

© 2024 Ultragenyx Pharmaceutical Inc

Deficiency Disorder Consortium, which is directing theImperfecta: Understanding Experiences Based on

the largest collection of data about osteogenesis imperfecta

for life. To date, more than 100 patients have been accepted
are on treatment, and nine Investigational New Drug (IND)
applications have been submitted to the FDA.

(OI) and its impact on patients, their families and care givers.
OI is a rare genetic condition that leads to abnormal bone

structure, decreased bone mass, bone fragility and weakness.
The IMPACT Survey is a joint research project between the

Osteogenesis Imperfecta Foundation (OIF) and the umbrellaorganization Osteogenesis Imperfecta Federation Europe(OIFE), with support from Mereo BioPharma, Ultragenyx’s

30Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Zoe’s Inspiring Journey with Sanfilippo Syndrome Type A
Zoe’s life story is a testament to resilience and hope in the face ofNow 11 years old, Zoe is a vibrant child who loves Disney movies,

Diagnosed at just three months old, Zoe’s journey was marked

which has impacted her ability to speak, Zoe’s joy for life remains

Sanfilippo syndrome type A, an ultrarare neurodegenerative disorder.
by early developmental delays. Unlike typical language learning

challenges in bilingual households, Zoe’s late walking and mild tomoderate hearing loss were early signs of a deeper, more complex
condition.

The journey to diagnosis was fraught with uncertainty and lackof support, leaving Zoe’s family to navigate the complexities ofan ultrarare disease affecting one in 70,000 newborns. Her family
initially assumed her bilingual environment was the cause of her

baking cookies and music. Despite the progression of her condition,
undiminished. Her story is not just about the difficulties of living witha rare disease, but about the strength and love of a family adjusting
their lives to support their daughter’s needs.

Zoe’s journey highlights the importance of early diagnosis, the needfor a supportive community, and the relentless spirit of families

dealing with rare diseases. Her smile continues to inspire hope andresilience, serving as a beacon for others on a similar path.

developmental delays and was shocked when she was diagnosed

with Sanfilippo syndrome type A. They desperately needed resourcesand emotional support and found it challenging to appear strong,but they found solace and community through the Cure Sanfilippo
Foundation and the National MPS Society.

Zoe’s story is not just about the difficulties
of living with a rare disease, but about thestrength and love of a family adjusting theirlives to support their daughter’s needs.

© 2024 Ultragenyx Pharmaceutical Inc

31

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Rare BootcampTM
We host a recurring Rare Bootcamp ,TMwhere we share our knowledge,

expertise, insights and connections

to help patient families, foundations

and organizations seeking to develop
novel treatments for rare diseases.

Our Rare Bootcamp is designed for incredibly determinedpatient families and advocates who have started funding
their own rare disease research and are looking to better
coordinate and build structure around their efforts.
What began as a half day meeting in 2017 is now amulti-day event and takes place twice a year. Topics

include therapeutic modalities, manufacturing strategy,
development strategy (including clinical endpoints anddiagnostics), regulatory considerations, raising capitaland partnering. We also allow time for one-on-one
meetings so experts can share their advice.

Rare Bootcamp
Highlights
In 2023, we hosted 2
Rare Bootcamps with 40
participants from morethan 30 organizations
attending.

The event has evolved based on participant feedback. For
example, on the research side, we now conduct a working

group to assess scientific gaps in knowledge associated witheach disease and the appropriate therapeutic modality toprioritize. In addition, we have added a panel discussion andlecture topics focused on selecting an academic partner to

conduct this research as well as how to define contract terms
governing data ownership and research progress.

© 2024 Ultragenyx Pharmaceutical Inc

Since 2017, we haveheld 8 rare Bootcampswith more than 135
participants from morethan 100 organizationsin attendance.

32

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Patients

Propelling the entire rare disease community forward
to transform as many lives as possible

We are committed to supporting the rare diseasecommunity through our efforts to develop novel therapies,
share our science and expertise, achieve broad access toscreening and treatment, and partner with policymakers formeaningful change.
Sadie and her mother, Ashley.

Sadie is a very happy girl who lights up a room withher beautiful smile and her eyes full of joy. She lives
with Sanfilippo Syndrome, also known as MPS III.

© 2024 Ultragenyx Pharmaceutical Inc

33

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Aspiration
To achieve meaningful benefit

for people living with rare diseases
by delivering on the promise of

Our Objectives

2023 Progress

Achieve majority access through responsible pricing
and support services• We continue to align U.S. price increases consistent with the Consumer Price Index

our science and our therapies.

• More than 600 patients in over 45 countries have been approved for access to Ultragenyx treatments through various
global expanded access and patient assistance programs since 2013
• As of December 31, 2023, more than 4,700 patients have received Ultragenyx treatments through commercial
or expanded access in more than 50 countries

Use our expertise to amplify the voices of the rare diseasecommunity to inform and influence key decision-makers in
the field of rare disease• Successfully advocated for the inclusion of more rare diseases in newborn screening (NBS) programs across several
U.S. states

Incorporate the perspectives and experiences of patientsand caregivers in our decision-making, with a focus onaddressing unmet needs for basic necessities and improving
quality of life

• Attended over 25 patient advocacy conferences, meetings, and town halls worldwide, engaging with advocacy leaders
and rare disease community members, fostering awareness and collaboration• Contributed to policy discussions and advocated for legislation that supports the development of treatments for rarediseases, with the goal of ensuring that the voices of the rare disease community are heard at the legislative level• Hosted regular patient and community leadership councils, including new councils for emerging therapeutic areas,
to gather insights and feedback on the company’s strategies, research priorities and patient support services• Co-authored and presented research findings, including patient advocacy perspectives on gene therapy, at key
industry conferences, highlighting the challenges and opportunities within the rare disease field to a broad audienceof stakeholders, including healthcare professionals, researchers and policymakers© 2024 Ultragenyx Pharmaceutical Inc

34

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Access and Affordability
We believe the greatest impact we can have on the
lives of individuals with rare disease is to make ourtreatments accessible and affordable to anyone who
can benefit from them and to engage and support

rare disease families along every step of their journey.
To date, more than 4,700 patients have received

Ultragenyx treatments via commercial or expanded
access in more than 50 countries.

We have taken a responsible approach to pricing our therapies from thestart with the goal of enabling as many appropriate patients as possible toaccess treatment. Our focus is on expanding patient access while maintaining
investment in innovation. We evaluate each medicine’s value based onhealthcare economics, clinical data and comparisons to similar therapies.

Additionally, we consider the costs of producing high-quality medicines andsustaining a robust global supply chain. We set our medicine prices with global

pricing in mind. In the U.S., we leverage effective reimbursement strategies anduse our best efforts to prevent patients from foregoing Ultragenyx therapy for

In the last five years, Health Technology Assessment (HTA) Agencies
around the world have evaluated and recommended our therapies
for reimbursement, which is a testament to the potential for thesetreatments to enhance healthcare outcomes globally.

• Mepsevii (vestronidase alfa-vjbk) was recommended by Brazil’s
CONITEC, England’s NICE, Germany’s G-BA, Italy’s AIFA, Spain’s AEMPS
and Portugal’s INFARMED.

financial reasons. Our patient assistance programs include co-pay support and• Crysvita was recommended by Canada’s CADTH and INESSS, Brazil’s
CONITEC and Mexico’s Federal Reimbursement List.

products for which we lead promotion were guided by the Consumer Price Index.

• Dojolvi was recommended by Canada’s CADTH and INESSS and
Mexico’s Federal Reimbursement List.

free access where necessary. In 2023, U.S. price increases for the commercial

As we look ahead to our next-generation gene therapies, we have invested in

our proprietary Pinnacle PCL gene therapy technology platform and our GTMF
in Bedford, Massachusetts. Gene therapy manufacturing issues have been a• Evkeeza was recommended by France’s HAS, Italy’s AIFA and
Canada’s CADTH.

known barrier to treatment access, and our goal in establishing end-to-end genetherapy R&D and manufacturing capabilities is to improve our control over costs,
production and our ability to scale.

© 2024 Ultragenyx Pharmaceutical Inc

35

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Access and Affordability (cont.)

Timely Care for Rare Disease
Patients Anywhere
Our product supply team’s strategy is designed
to address the unique challenges of the raredisease community, which is characterized by low

volume and high complexity demands and urgent
needs. We have enhanced our supply network

and implemented a strategic logistical approach,with the goal of providing swift deliveries for raredisease patients globally. To optimize operational
efficiency and minimize extensive travel, we have
set up regional hubs staffed with local experts.

In collaboration with our managed access partner,
we streamline processes by consolidating smallerorders. This is designed to reduce shipment

frequency and focus on supplying therapies for three

to six months at a time. Where possible, we also offer
direct-to-patient deliveries. This strategy is intended
to maintain continuous access and a better delivery
experience while enhancing cost efficiency.

© 2024 Ultragenyx Pharmaceutical Inc

36

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Access and Affordability (cont.)
UltraCare® Programs

Expanded Access

We created our UltraCare programs to help patients andWe are committed to supporting individuals with rare disease and their families in receiving proper

eligibility for our financial assistance programs, navigate

medicines for both children and adults with rare and ultrarare diseases is matched by our efforts tocaregivers understand their insurance coverage, determine
access to treatment and find patient support programs.

UltraCare programs, based on local regulations, are currently
in place in the U.S., Canada, Argentina, Brazil, Colombia

and Mexico, and programs are in development in additionalcountries. Visit Ultracaresupport.com for more information
about our U.S. programs.

diagnoses and optimal care following a rare disease diagnosis. Our commitment to developing new
make our commercial therapies accessible to patients via appropriate mechanisms, particularly incountries where regulatory authorities have yet to approve such treatments.

Although clinical trials offer access to our investigational therapies, some patients who suffer fromserious or life-threatening diseases may be ineligible to participate in such studies and may not haveother viable treatment options. In such cases, where able, we offer our investigational therapies on acompassionate use basis to qualified patients worldwide via our early access program. Our evaluationof requests for individual patients to receive investigational therapies outside of a clinical study isconducted on a case-by-case basis. Given the urgency associated with treating patients with rarediseases, we aim to respond to compassionate use requests within 24 hours and to ship therapies within
48 hours wherever feasible. We are committed to supporting physicians in the timely and excellent careof their patients and patient families.

Over 40 patients weretreated with Ultragenyx
medicines throughcompassionate use
in 2023© 2024 Ultragenyx Pharmaceutical Inc

More than 600 patients in over 45
countries have been approved foraccess to Ultragenyx medicines

through various global expanded

Sponsored testing programs shorten
the diagnostic odyssey
A typical rare disease patient requires 6 years and an average of

17 clinical encounters in the quest for an accurate diagnosis after

symptom onset. Genetic testing is often necessary to confirm a raredisease diagnosis and for a patient to qualify for reimbursement of

optimal therapy. However, genetic testing itself is often not reimbursed.

Ultragenyx is focused on differentially diagnosing 9 different rare diseases
to give physicians information that will support clinically-appropriate
decision making and disease management. These sponsored testing

programs can reduce the time to diagnosis for patients and help provide
patients with timely care. Ultragenyx sponsored testing programs havespecific eligibility requirements and tests are only offered to appropriate,
clinically-suspected patients who are already embedded in the medical
system on a diagnostic odyssey. At Ultragenyx, we are committed tosharing the learnings from our sponsored testing programs with the

public. This includes information about what gene changes (or variants)
are associated with a given disease. We have created a website thatmakes this data available to the public and we encourage users of thewebsite to not only explore our data but also contribute their own newvariant data.

access and patient assistanceprograms since 201337

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Patient Advocacy and Engagement
Ultragenyx was built hand-in-handwith the rare disease communities
we serve. Our priority is to partnerwith patients and their families fromthe earliest stage of drug development
through clinical research and support

patients and their families throughout
the treatment life cycle.

We also have the opportunity to listen to first-handexperiences from invited speakers living with rare diseases,
which allows us to see the real-world impact of our
medicines on patients and families.

Our patient advocacy and engagement work includes
the following:• Establishing partnerships with patient organizations

Patient Engagement Plans (PEPs)
PEPs are collaboratively developed by a cross-functional groupled by our patient advocacy department to include the patientand patient community perspectives and lived experiences

across all functional areas of product development. Multiple
teams explore and address various aspects of a disease withpatients, caregivers and the affected community, including

to best support patients and patient communities andspecific care landscapes and the associated healthcare

development of therapies to address unmet needs.

experts may not have comprehensive insight into how patients

incorporate community insights and priorities in the• Educating the broader rare disease community by

sharing information about rare and ultrarare diseases
and their impact.

• Engaging with key patient community members toamplify the voices of the rare disease community.

• Supporting patients, their families and the rare diseaseenvironment and services. Since physicians and disease

perceive the disease’s impact on their quality of life, thePEP process involves regularly reassessing existing sourcesof patient experience data. Patient involvement is critical tomaking informed decisions in drug development and PEPs

A real-life example of an effective PEP
is the one we used with our UX143
program for osteogenesis imperfecta (OI).
The cross-functional team met early in
product development to gather patient
insights and perspectives, and thework with the patient community has
evolved simultaneously with the overall
program. Patient insights from the OI
community were included in the work
done by our Clinical Operations, ClinicalDevelopment, Regulatory and Global
Brand Strategy departments.

provide a framework for cross-functional leaders to seek out
insights and incorporate patient feedback into their work.

community through medical education and health-related grants. Please see the Grants section of thisreport for information on our support of medical

education grants and health-related grants to patientadvocacy groups and our website for a list of patientadvocacy groups we partner with to provide education,
support and periodic updates of our clinical programs.

© 2024 Ultragenyx Pharmaceutical Inc

38

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

2023 Corporate Responsibility ReportGRI Index

Partnering with Patient Organizations
The Ultragenyx Patient Advocacy andPatient Engagement team is dedicatedto advancing global rare diseaseadvocacy through inclusive patientengagement and partnerships.

To further this purpose, in 2023, we attended more than

25 patient advocacy conferences, meetings and town halls

across the globe. We engaged with advocacy leaders and raredisease community members, sponsored educational events,and shared information on our pipeline research programs.
Ultragenyx also hosts regular patient and community

We currently host the following community leadership councils:• LC-FAOD Patient Leadership Council• OI Leadership Council• XLH Patient Education Advisory Council• Sanfilippo Caregiver Council• CTD Caregiver Leadership Council• Global Gene Therapy Advisory Council• Angelman Caregiver Leadership Council

leadership councils with the goal of having a consistent andstructured way to gain insights from the patient community
on our work. These leadership councils are a forum for

Global Gene Therapy Advisory Council

Ultragenyx to provide updates on our work and to receive

In 2021, Ultragenyx founded our Global Gene Therapy Advisory Council, which includes nine patient advocacy leadersthem. The insights from these efforts help inform ourcouncil are to garner insight from council members on priority challenges, knowledge gaps and unmet needs related tolifecycle and place patient and community perspectives

therapy, and identify potential collaboration opportunities to address unmet community needs regarding gene therapy.

feedback from council members on areas of interest tofrom the U.S., Mexico, Netherlands, U.K. and Sweden, representing various rare disease communities. The goals of thestrategies and decision-making throughout the productgene therapy within rare disease communities, increase community awareness, knowledge and understanding of geneat the center of our work.

The council met several times in 2023. At the end of the year, we developed a framework and messaging for

educational resources on gene therapy based on the diverse feedback we received from council members. The council
also identified a series of strategic issues that can be addressed across communities to increase patient understanding
and acceptance of gene therapies on a global scale. In conjunction with Ultragenyx, several members of the council

co-authored and presented a poster titled “Rare Disease Patient Advocacy Perspectives on the Promise and Challengesof Gene Therapy” at the 2023 American Society of Gene & Cell Therapy Annual Meeting.

© 2024 Ultragenyx Pharmaceutical Inc

39

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Providing Educational Resources
Our patient-focused websites provide
customized education and raredisease awareness to patients andtheir families. We also prepare plain

language summaries of clinical trial
and DMP results.

Our Educational Resources:
www.mpsviiinfocus.com

Our Rare Journey Resources, at Ultrarareadvocacy.com, are designed to provide access to information(MPS) VII available to the global community in English,

connect and get involved.

A disease education website about mucopolysaccharidoses
Spanish, Portuguese, Italian, Polish, Romanian, Hungarian

for many of the common phases of the “rare” journey and empower patients and their caregivers to

and Croatian

www.OneXLHvoice.pt

A disease education website about X-linked

hypophosphatemia (XLH) available to the community
in Mexico and Brazil

www.faodinfocus.com

A disease education website about long-chain fatty acidoxidation disorder (LC-FAOD) available in English and Spanish
ultraclinicaltrials.com

A global clinical trial recruitment website for caregivers
and people living with rare diseases© 2024 Ultragenyx Pharmaceutical Inc

40

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Resilience and Advocacy: Dr. Michelle Fynan’s Journeywith Osteogenesis Imperfecta (OI)
Meet Dr. Michelle Fynan, a distinguished psychotherapist and advocate, whose

Moreover, Michelle’s advocacy extends beyond her personal experiences.

characterized by fragile bones. Diagnosed in early childhood, Michelle has notchampioning awareness for OI and underscoring the importance of community

life is a testament to resilience and strength in the face of OI, a genetic disorder
only overcome immense physical challenges but also become a beacon of hopeand change for others facing similar struggles.

She actively supports Ultragenyx’s mission in rare genetic disorder research,
support, self-advocacy and innovative treatments.

From her earliest years, Michelle faced the daunting challenges of OI. Herlife has been marked by more than 30 fractures and numerous surgeries.
Despite facing both physical and psychological impacts, including post-traumatic stress disorder, Michelle has achieved remarkable milestones,
showcasing her relentless spirit and adaptability.

Michelle’s family, initially unfamiliar with OI, quickly became her bedrock ofsupport. Their unwavering determination and love have been pivotal in shaping
her journey. Today, as a mother to Addison and Avery, both diagnosed with OI,

Michelle instills the same spirit of resilience and empowerment in her daughters.

© 2024 Ultragenyx Pharmaceutical Inc“For me, resilience means more than justovercoming; it’s about thriving in the face ofchallenges. It’s about transforming obstacles into
opportunities for growth and personal evolution.”– Dr. Michelle Fynan

41

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

2023 Corporate Responsibility ReportGRI Index

Public Policy Participation
Ultragenyx recognizes the vital
importance of public policy

engagement in our mission to serve
individuals living with rare diseases.

We understand that policy and regulations are essential

pricing, and accelerating the development timelines for

As part of our commitment to transparency, we disclose in acommercialization of treatments for the 1 in 10 Americans

processes through newborn screening (NBS), contributing

California candidates, and the Ultragenyx Political Action

for the cost-effective and timely development andliving with rare diseases. They also play a critical role inhelping people with rare diseases receive accurate diagnoses,
quality care and timely access to therapies. Our commitment
to public health and medical innovation is reflected in our
advocacy for patient access to FDA-approved medicines,
awareness of these treatments, and reforms to foster
patient-centered care.

Our Global Policy Committee identifies priority areas for our
engagement and advocacy, aiming to educate, inspire andinfluence key decision-makers in the rare disease field. The

committee’s objective is to enable all individuals with a raredisease to receive an accurate diagnosis, quality care andaccess to available therapy as quickly as possible.

© 2024 Ultragenyx Pharmaceutical Inc

new medicines. Efforts also include improving diagnostic

to gene therapy development, and increasing awareness ofultrarare diseases in emerging markets. We establish thesepriorities and positions in consultation with our management
team, who are updated annually on our advocacy efforts.

timely fashion our limited corporate contributions to several
Committee (PAC) makes contributions to various lawmakers
and committees. The Ultragenyx PAC is registered with theFederal Election Commission (FEC) and works to adhere to allreporting requirements. Information about our contributions

Our activities in public policy are guided by our Global

is publicly available on the FEC’s website.

with all relevant laws in our engagements with public andCompany Coalition (RDCC), a coalition of 23 companies that

Code of Conduct, with the goal of acting in compliance

Additionally, we are a founding member of the Rare Disease

governmental entities. Our Government Affairs and Public

are collectively investing more than $15 billion annually in

Policy team is at the forefront of our interactions withlegislative and regulatory bodies, committed to contributing
responsibly and with a civic-minded approach to thescience of rare disease medicine. At the federal and statelevels, members of the Ultragenyx team engage in policy

The committee sets our positions and defines our priority

discussions with governments, trade associations, patienton public health policies. These priorities encompass

experiences and insights as a biopharmaceutical company

providing access to innovative therapies through responsible

medical needs, we believe we add valuable perspectives topolicy areas, focusing on making a significant impact

groups and other stakeholders. By sharing our uniqueenhancing the patient experience in drug development,

dedicated to developing therapies for patients with unmet
the ongoing dialogue about tackling rare diseases.

R&D and that have over 200 treatments either approved orin development. Betsy Ricketts, our vice president of Policy,

Government and Public Affairs, was elected in 2023 to serve

as secretary of RDCC’s Executive Committee. RDCC educatespolicymakers on the distinct considerations of life science
companies operating in the rare disease field and focuseson three priorities:• supporting robust development and innovation• promoting accessibility• enabling earlier diagnosis

42

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Public Policy Participation (cont.)
We amplify the voices of the rare disease community by supporting the following policy priorities:
Patient

Prioritizing patients in development and commercialization, including patient experience data

Market AccessHelping make rare diseases a priority in global markets

Pricing andProactively engaging with payers to achieve majority access to treatments while advocating

Diagnostic

Shortening the timeline from diagnosis to treatment access through initiatives such as NBS

GeneEngaging policymakers in innovative approaches to clinical and manufacturing development

Experience

Reimbursement

Odyssey

Therapyin drug applications and labels

for responsible pricing and flexible payment models for innovative therapies

and genetic testing

while characterizing unmet needs and burden of illness in peer-reviewed literature

Accelerated

Streamlining drug development timelines, including biomarker and endpoint development,

Emerging

Raising awareness of ultrarare diseases, encouraging local definitions, and educating on

Development

Markets© 2024 Ultragenyx Pharmaceutical Inc

Our CEO alongside members of the public affairs team during a public policy advocacy visit to Washington, D.C.

to speed up assessment and approval by global health authorities

advanced therapies, including gene therapy and mRNA

43

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

2023 Corporate Responsibility ReportGRI Index

Public Policy Participation (cont.)

Advocating for Newborn Screening (NBS)

Advocating for an UltraOrphan Drug Act

NBS is a vital part of treating rare diseases. With

January 4, 2023 – 40 Years of the Orphan Drug Act (ODA): This landmark legislation has been instrumental

treatment that can help prevent permanent disability,

1,000 rare diseases and significantly enhancing the lives of those with rare conditions.

early detection, affected infants can receive prompt

developmental delay and death. NBS programs in the

U.S. are state-run public health programs that identify

newborns with certain genetic, metabolic, hormonal orfunctional disorders. Because NBS programs are state

run, there are major discrepancies regarding the diseases
each state screens. The total number of conditions

included in screening ranges from 34 to 67, and no state
screens for all 37 core conditions and all 26 secondary
conditions on the Recommended Uniform Screening

Panel (RUSP). Ultragenyx advocates for these state-run programs to screen for all conditions on the RUSP so that

infants with rare diseases can receive prompt access to treatment no matter where they are born in this country. In

in fostering the development of treatments for rare diseases, leading to over 600 treatments for more than

Challenges in Ultrarare Disease Drug Development: Despite the success of the ODA, there are substantial regulatory

and commercial hurdles in creating treatments for ultrarare diseases, which affect extremely small patient populations.
This has led to the cessation of development of multiple investigational therapies by many companies, prompting

nonprofits, often spearheaded by parents of children with ultrarare diseases, to take charge of therapy development.
Ultragenyx’s Advocacy for the UltraOrphan Drug Act: In response to these challenges, Ultragenyx is proposing
the UltraOrphan Drug Act, legislation intended to implement a new framework for incentivizing development oftherapies for diseases with very small patient populations that will be a supplement to the existing ODA. This newact aims to facilitate the development of therapies for diseases affecting fewer than 2,000 people in the U.S.,
defined as ultrarare diseases.

Key elements of the proposed framework include:2023, Ultragenyx advocated for the passage of legislation in Texas so that when new conditions are added to the RUSP,• Recognition of qualifying biomarkers as primary
endpoints in pivotal clinical trials

do so (RUSP-alignment legislation).

• Acceptance of alternative study designs and analyses
for ultrarare diseases

Texas will add those conditions to its state panel within a specific period of time and that it has the requisite funding to
In 2024, Ultragenyx will be working to pass RUSP-alignment legislation in Alabama, Colorado, Tennessee and Washington.
See also Research Collaboration for information on Ultragenyx joining BeginNGS consortium, a Rady Children’s initiative
to develop genomic analysis tools for hospitals to significantly increase NBS and early diagnosis of rare diseases.

© 2024 Ultragenyx Pharmaceutical Inc• A 50% tax credit for clinical trial costs (capped at$100 million per program), 10 years of marketingexclusivity, and waiver of application user fees

• Implementation of a “sufficient efficacy standard”
akin to the emergency use authorization standard,
replacing the “substantial efficacy standard”• Assurance that approvals under this framework are
full, not experimental, and are recognized as suchby payers

44Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Gabrielle’s Journey with Angelman Syndrome (AS)When Gabrielle was 7 months old, her parents noticed that she was behind

Gabrielle loves to give hugs and finds joy in many things. Now at school, she iscategorization of “gross developmental delay.” At 18 months, she was given

doing their jobs. Gabrielle’s family motto is to always BELIEVE. Anna says thaton certain developmental milestones. Pediatric testing resulted in a generala blood test for AS following a health scare and the results were negative.

Gabrielle was misdiagnosed several times before finally being given whole-known as “Mayor Gabby” because she takes the time to observe if people arewhile life will be different, you shouldn’t give up on your dreams for your child.

exome sequencing, which finally resulted in a definitive diagnosis of AS atage 2. She has a rarer subtype of AS that didn’t show up with a blood test.
AS is a condition characterized by developmental delays and neurologicalchallenges and affects roughly 1 in 15,000 births. With no therapy available,

Gabrielle’s diagnosis catalyzed her family’s commitment to advocacy, researchsupport and community engagement. Her mother Anna’s advice for otherfamilies walking the same path is to know that AS is part of your child, but
it does not define who they are.

© 2024 Ultragenyx Pharmaceutical Inc

Gabrielle’s diagnosis catalyzed her family’scommitment to advocacy, research support
and community engagement.

45

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

PeopleSustaining and strengthening our generous and inclusive
culture while enhancing our health and safety practices

We are committed to working to maintain a diverse,
inclusive, safe and healthy environment. We are also
committed to fair and equitable compensation practices
that are transparent and free from bias.

© 2024 Ultragenyx Pharmaceutical Inc

46

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Aspiration
To be an inclusive, sought-after

Our Objectives

2023 Progress

first and feel motivated to bring theMaintain a positive workforce culture by achieving a total
turnover rate below the industry average and continuing tohave high employee engagement• Total turnover was 15.5%, which is below the U.S. and global averages for our industry (according to Aon Radford’s
Salary Increase and Turnover Studies)

Support internal career and leadership development
through our significant investment in customized employee
programs that build core competencies and bring ourcompany values and culture to life

• Successfully implemented an enhanced and bespoke framework for evaluating and rewarding performance

Continue to strengthen and expand diversity andinclusion through intentional talent acquisition andmanagement efforts, including candidate pipelining,interview processes, ongoing education, awards, promotions
and succession planning• Women accounted for 60% of promotions globally, while U.S. employees who self-identified as racially or ethnicallydiverse represented 46% of U.S. promotions

Implement a robust and comprehensive health and safety
management system framework and audit process• Introduced a New Hire EHS Checklist across all sites, completing 315 checklists to enhance safety awareness among
employees and contractors regarding evacuation routes and emergency equipment

company where employees comebest versions of themselves to work
each day, knowing they are making
a difference in the lives of the raredisease community.

• Maintained a high engagement score of 86% in our employee engagement survey• Nearly 22% of our global workforce participated in the on-demand career coaching services we offer• Hosted 24 interns across our emerging talent programs• Broadened our mission and evolved from Inclusion & Diversity (I&D) to Diversity, Equity, Inclusion andBelonging (DEIB)• Conducted DEIB Leaders’ Workshops to unite DEIB Action Team and ERG leaders, enhancing our 2020-2025
DEIB Roadmap• Launched a safety observation system, engaging employees in safety improvements, leading to over 300 reported
and addressed hazards• Completed more than 140 ergonomic hazard assessments© 2024 Ultragenyx Pharmaceutical Inc

47

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Culture and Values
We have intentionally built andconsistently nourished our company

culture so our employees can experiencea sense of purpose and fulfillment intheir work – while feeling connectedeach day to the bigger impact we haveon the rare disease community.

We aspire to be a company where our family, friends and children are proud to work. This means having
a steadfast commitment to working to create and sustain a healthy, inclusive company culture where
our people feel genuinely cared for and supported so they can thrive in all areas of their lives. As
foundational to our culture, we encourage generosity, curiosity and humility so we can continue
to learn together while fostering an environment that supports profound growth.

Our people are
Ultra-focused

Ultra-curious

Our team works together fearlessly to uncover new possibilities.
Our team applies their biggest ideas in courageous ways.
Instead of asking “Why?” they ask “Why not?”Ultra-impactful

Our team works hard to make a difference for those who need it most.

Ultra-dedicated

Our team recognizes their biggest challenges yield rare possibilities.

We carefully crafted our cultural values to empower our team members,

allow for their voices to be heard, and encourage them to strive to makea difference.

GENEROUS

DYNAMICWe are committed to helping –We learn and adapt –with our patients, our field andunderstanding and rapidly evolving

sharing our knowledge and skills

constantly searching for deeper

each other.

our plans based on our insights.

COURAGEOUS

POSSIBILITY

We go where others won’t –We seek the undiscoveredtaking on the challenges thatto finding options for those whotargeting untreated diseases anddiscoveries – we’re committed

move our field forward.

don’t have any.

RELENTLESS

We won’t give up fighting forthe rare disease community –Ultra-innovative© 2024 Ultragenyx Pharmaceutical Inc

Our team takes rare and dynamic challenges head on.

together always searchingfor solutions.

48

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Human Capital Development
Ultragenyx has more than 1,270

employees globally. We are dedicatedto building a global and diverse

team; maintaining a healthy, inclusivecompany culture where employees feel
respected and valued; and providing
opportunities for learning, personal
growth and career advancement.

We strive to provide employees with a workplace and workand structure. The goal is to support the development of

they want to stay long term. Our voluntary turnover was

leadership succession plans, and refine DEIB strategies. Our

environment where they can do their best work and where
10.5% in 2023, which is below the U.S. and global averages
for our industry (according to Aon Radford’s Salary Increase
and Turnover Studies).

future leaders, identify skills and capability needs, update
Executive Team meets annually to discuss the succession
plan for the company, and each Executive Team member
We also support and encourage team building with teamdisease. In an effort to enhance camaraderie and embed

lunches, UltraTalk Speaker series, group exercise, happy hours,

recognition into our culture, we have an internal platform
called “U Earned It,” where employees at all levels canrecognize colleagues for their contributions and award themwith points that they can redeem for rewards.

We actively nurture and develop our internal talentpipeline, providing opportunities for employees to growwithin their roles and beyond. This includes our Mentorship
Program, which pairs employees across the business toaccelerate growth and development, foster cross-functional
relationships, and strengthen our teaching, learning andnetworking skills. In 2023, more than 110 employees

participated in the program. Our executive leadership teamMore than 35% of new hires camethrough our employee referral program

updates the plan for their employees.

We push each other to perform at our very best to support
our mission to transform the lives of people with rare

In 2023and department offsites, weekly company-sponsored

milestone celebrations, summer and holiday events, andmore. These events are held both virtually and in-person.

Turnover in 2023
Total Turnover Rate

15.5%

Voluntary Turnover Rate

10.5%

Executive/Senior-Level Officials and Managers

0.5%First/Mid-Level Officials and Managers

6.7%

Others (Administrative Support Workers, Professionals, Technicians, and Laborers and Helpers)

3.3%

plays an important role in workforce planning by periodicallyassessing the company’s overall organizational design© 2024 Ultragenyx Pharmaceutical Inc

49

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

2023 Corporate Responsibility ReportGRI Index

UltraPerformance Management
We refer to our performance management
system as UltraPerformance, which

formal employee reviews twice a year where performanceis assessed against an employee’s objectives. We expect

management by objectives, aligning

our managers to have regular check-ins with their direct

individual efforts with the company’s

overarching goals. It plays a crucial rolein empowering team members to makesignificant contributions toward sharedobjectives while offering opportunities
the company.

periodically reviewing annual goals to help keep themaligned with the company’s strategic direction. We conduct

represents a holistic approach toto enhance skills and advance within

The core of UltraPerformance involves establishing andreports throughout the year and provide real-time feedback
and recognition. These interactions provide a broader
understanding of each employee’s strengths, career

aspirations and performance contributions. They also help
identify opportunities to accelerate career development
and align individual achievements with compensation
and rewards. This comprehensive approach helps tocultivate a culture of continued improvement and open
communication and is integral to our ongoing success,with the goal that individual contributions are not only

recognized but also directly linked to both personal andcompany-wide achievements.

See also Employee Compensation and Benefits.

In 2023
Women accounted for 60% of promotions globally, while U.S. employees who
self-identified as racially or ethnically diverse represented 46% of U.S. promotions© 2024 Ultragenyx Pharmaceutical Inc

Enhancing Performance Management
After an extensive three-year planning process, incorporating stakeholder feedback from employee and manager
focus groups, we have moved away from our prior five-point rating system and successfully implemented anenhanced framework for evaluating and rewarding performance. This new and bespoke framework, applicable toall employees, is designed to empower discussions and evaluation of individual performance and development in

a comprehensive and meaningful way. This new framework formally integrates three equally weighted categories,
historically fundamental to evaluating performance, that form the basis for calculating the annual bonus payout:

Goal Attainment

Outcomes achieved against
individual objectives.

Role Performance

Performance against key job
priorities and expectations.

Core Value Alignment

Behaviors consistent with integrity
and our core values at Ultragenyx.

This transition has delivered several benefits for our workforce:
Clarity: Employees now have a clearer understandingof their goals, job expectations, and how their actions
reflect our values on a day-to-day basis.

Empowerment: People managers now have greater
flexibility and nuance in recommending year-end
bonuses, enabling them to recognize and address
both successes and areas for improvement.

Alignment: A stronger correlation between
company, individual performance, and actual bonus
payout, reinforcing our commitment to the pay-forperformance framework.

Transparency: The new framework provides
a transparent structure for offering feedback,encompassing both celebratory achievements andopportunities for growth.

To facilitate the transition, we employed a thoughtful change management strategy. This included the developmentof guides, FAQs, and training videos to assist our employees in navigating the changes.

50Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

2023 Corporate Responsibility ReportGRI Index

Employee Learning and Development
Our culture continues to grow andevolve, and we believe that each

employee plays an important rolein shaping and sustaining it. That is
why we are deeply invested in the
personal and professional growthof our employees, making it astrategic focus of our company.

We have instituted a unique approach to employee

The goals of our employee development programs areexperience designers and facilitators. This team develops

curiosity and humility, teach effective feedback, reinforce

development, led by an in-house team of skilled learningand customizes the majority of our programs thatare designed to align with our culture and meet ourcommunity’s specific needs. These needs are identified

through a thorough process that includes behaviors andattitudes measured by our annual engagement and pulse
surveys, performance management processes, ongoing

dialogues with business leaders and teams, and assessmentof alignment with our company values, vision and strategy.
We also rigorously track the impact of our programs.

Each course undergoes real-time evaluation for relevance,

comprehensive, aiming to deepen self-awareness, encourage
an empowered mindset, strengthen compassion of/for self
and others, build a diverse and inclusive community, andsupport our company vision and strategy. These objectives
not only relate to the ability of our employees to excel

ImpactOur employee development initiatives havesignificantly boosted our engagement andretention key performance indicators (KPIs) above
industry benchmarks over the past six years:

in their roles and collaborate effectively but also are• Raised our annual engagement index
and DEIB engagement scores

increased engagement, high retention rates, career growth• Maintained high retention and job
satisfaction rates

leadership in corporate culture.

• Attained a high rate of manager effectivenessdesigned to contribute to crucial business outcomes such asopportunities, talent attraction, brand enhancement andapplication potential, and overall enjoyment. We aim tocontinue to refine our programs based on feedback sothat even the highest-rated courses are regularly updated.

© 2024 Ultragenyx Pharmaceutical Inc

51

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Employee Learning and Development (cont.)
We offer both required and optional workshops that

extend beyond conventional models, combining highengagement and impactful content with a mix ofin-person and digital formats. Required workshops include:
Dynamic Feedback

Seek, Offer, Receive: Encourages a culture of ongoing

improvement by teaching employees to effectively seek,
give and accept feedback
Empowered Mindset

Helps employees link their mindset with actions andoutcomes, promoting better decision-making
Managing at Ultragenyx

A comprehensive workshop series providing managers with
communication tools, management strategies and online
career development resources

How to Effectively Manage Declining Performance

A focused 90-minute training for managers to address
team underperformance

Leadership Development Program (LDP)

Helps enhance understanding of personal leadershipstyles, improves work relationships, and reinforces company
culture commitment© 2024 Ultragenyx Pharmaceutical Inc

Some of our other popular programs to support employees in their development include:
High Performing Teams

Insights 101: Introduction

quickly from forming and storming to performing

understand others by learning about the insights

Our customized model and approach to help teams move

Promotes enhanced self-awareness and ability to

People Manager Tools

working style assessment and the four-color energies

a resource library, monthly newsletters, essential workshops

Provides simple stress relief tools, online resources

Comprehensive support for people management, including

Mindfulness for Stress Relief

and topical lunch series

and insights into the scientific benefits of mindfulness

Cultivating Resilience in Everyday Life

Presenting with Impact

and promote well-being

memorable content and effective delivery style

Provides research-based tools to stay strong

Energy Management Wins – PeopleFuel

Helps employees use energy effectively, assess life areas

Teaches basic skills for developing clear messaging,to business presenters who do not have extensive
presentation experienceand manage emotions for resilience and well-being

Technical training, particularly in areas such as quality and compliance, is managed separately by a specialized technicalgroup. This approach maintains focused expertise and provides tailored training for specific technical needs, thereby
complementing our broader employee development efforts.

Additionally, we sponsor programs such as UltraTalks, our version of TED Talks, to bring new perspectives and insights to thecompany. These experiences are designed to build employee morale, stimulate innovation and invest employees in company

improvement. Some of our past speakers have included Dr. Cal Newport, Abby Wambach, Patrisse Cullors, Dr. Laurie Santos
and Paul Hawken.

In 2023
We offered 39 employee development
workshops, of which 29 were provided
by our in-house Organizational
Development team.
The average rating for in-house delivered
workshops in terms of relevance,
application potential and overall
enjoyment was
92 out of 100.
The average* training anddevelopment time per employee
across the 39 development workshops
we offered was over one hour. The
attendance breakdown is representativeof our employees’ gender andracial/ethnic diversity.
*Our average training hours per person exclude time fortechnical training, required document reviews, and continuing
education/accreditation from third parties, which certain
employees must complete for their roles

52

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Improving How We Work
We believe that by embedding thetools and principles of continuous

improvement and Lean Six Sigma

into the way we work at Ultragenyx,

we can achieve our near-term goals

and advance our vision and strategy.

We aspire for our employees to adopt
a continuous improvement mindset,

focusing on efficiency, effectiveness,

In 2023, we invested in Lean Six Sigma by partnering with
Acuity Institute to bring this high impact methodology

to help solve problems and improve efficiency across ourcompany. The objective is to identify and deliver operational
improvements in the form of speed, quality and cost,enabling us to refine our processes, increase capacity andadvance our strategic goals. Additionally, we curated andcustomized a set of Lean Six Sigma tools and resources

for projects, so that our teams are well-equipped to drive
improvements. We also increased awareness throughIn 2023
Certified 29 Lean Six Sigma Champions.
Trained 18 Green Belts, 154 White Belts,
and 5 in Lean Foundations.
Kicked off 11 Lean Six Sigma projects.

company-wide meetings, internal websites and various

communications campaigns, embedding these concepts
into our corporate culture.

and working smarter, not harder.

© 2024 Ultragenyx Pharmaceutical Inc

53

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Career Development
We strive to not only be a place where
employees can do the best work of

their career but also where they canexperience profound professional andpersonal growth. In addition to our setof employee development offerings,
we have career development tools

and programs designed to support

employees in growing in their careers.

Ultra-Orbit
The Ultra-Orbit program aims to provide an opportunity for

High-Potential Program: LEAD (Leadership
Expansion And Development)

2023 LEAD Cohort Highlights

employees at all levels who are interested in developing
or enhancing their program and project management

We invest in various training and development programs

43% were womenskills while remaining in their current role. Each participantthat are designed to build and strengthen our employees’
leadership and professional skills. The goal of our LEAD

13% were based outside the U.S.

of leaders that will help us evolve our company and36% self-reported as membersof ethnic/racial minorities

engages with a program manager mentor, focusing on

measurable outcomes and timelines. Since the program’spilot in 2022, 15 employees have benefited from participatingin Ultra-Orbit rotations, and a significant number of employees
have inquired about these rotations. In 2024, we plan toexpand the program more broadly within the company.

Career Coaching

program, which started in 2018, is to build the next levelsupport our culture as we grow.

Each of the five LEAD cohorts since the program began

have been cross-functional and diverse as well as balanced
geographically, with the aim of being inclusive of remote
employees. The program provides workshops, businesssimulations, mentorship and executive leadership talks to

To enhance career growth and development, we offer

help equip participants with the skills to activate change

network of professional executive coaches. Coaching session

cohorts, 72% of participants were promoted within one

on-demand career coaching services through an external

throughout the company. Among the first four LEAD

topics include working relationships, performance and role,year of completing the program.

In 2023
More than 10% of total open positions
were filled by internal candidates

growth and development, and general stress.

In 2023, nearly 22% of our global workforce participatedin the coaching program. Participants rated the qualityof sessions 4.9 out of 5 stars. Initial feedback showed:© 2024 Ultragenyx Pharmaceutical Inc97%

of participants feel more positive about their issue.

98%of participants are more likely to address
their situation.

94%

of participants learned a new skill or strategy.
54

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Emerging Talent Programs
Ultragenyx is committed to providing
emerging talent with a range ofopportunities to learn from a diverse

team of world-leading experts at the
cutting edge of research.

Through mentorship, hands-on experiences and exposure

to cutting-edge methodologies, Ultragenyx aims to empower
the next generation while building a sustainable pipeline ofskilled professionals poised to make significant contributions
to the field.

In 2023, Ultragenyx focused on strengthening relationships

with local schools and universities. We have invested in highschool outreach through biotech partners and continued
partnership with the Japanese Community Youth Council(JCYC) where we support professional and technicaldevelopment with juniors and seniors at Oakland Tech in

Oakland and Galileo High School in San Francisco. Further,

Internships and seasonal co-ops

Hosted lab tours experience

San Francisco, to provide advanced pharmacy practicebiotechnology, integrating them into meaningful projects

hands-on lab experiences and interactions with scientists to

we have relationships with the University of California,

experience (APPE) rotations to prepare students for careers
in medical affairs and with Middlesex Community College

in Massachusetts as a pathway for careers in manufacturing.
Ultragenyx Emerging Talent programs include high schoolinternships and outreach, college internships, co-ops,

Advanced Pharmacy Practice Experiences (APPE) rotations
and fellowships. In 2023, we hosted 24 interns.

Offers students practical training in life science/at Ultragenyx to enhance their professional development.
Fellowships and post-doctoral programs

Offers a chance to explore key scientific questions affecting

the rare disease community, develop investigative skills, andgrow in a collaborative environment under expert mentorshipin life sciences/biotechnology. In 2024, we plan to expand our
fellowship offering to engage with underrepresented groups
in the sciences, aiming to enhance physician diversity andintegrate various perspectives in rare disease research.

© 2024 Ultragenyx Pharmaceutical Inc

Immerses students in a biopharma environment, offering

inspire Science, Technology, Engineering, and Mathematics(STEM) careers. In 2023, we hosted more than 60 students
and their instructors from San Marin High School and the
University of California, Davis, Graduate School ofManagement. Additionally, Ultragenyx summer interns, alongwith juniors and seniors from six local schools – including

Balboa and Galileo high schools in San Francisco, and San

Marin, Novato, Terra Linda and Tomales high schools in thegreater Bay Area – visited our Novato campus for a lab tour
and various STEM-related events and talks.

55

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Employee Engagement
We believe active listening andemployee engagement are essential

to maintaining a healthy and thriving
workplace environment.

We use the results of the “Your Voice” employee

We use our survey consistently to measure three people-

take action on opportunities that will improve employee

of four to six survey questions for each goal. The areas weengagement survey to pinpoint key areas of focus and to
experience and engagement. To implement feedback more
effectively and hear from our employees more frequently,

we restructured our approach so our most robust “Census”

related corporate goals that are assessed using an index
measure are focused on:• Enhancing employee engagement at Ultragenyx

survey with 65 questions happens every 18 months instead• Strengthening how our managers and leaders support
DEIB in the workplace

within those 18 months that focus on a limited set of topics,• Improving Health & Well-being in the workplace toreduce stress and balance work and personal lifeof every 12 months, and we added two new “Pulse” surveys
including our corporate goals and key strategic initiatives

and efforts. We made this change to create a timely listening
platform so we can more quickly develop and deploy
solutions that address opportunity areas.

“YourVoice” results continue to be discussed with ourboard of directors as part of its oversight of the generalorganizational health of the company. In addition, theresults are routinely shared in a timely manner with oursenior leaders, managers and employees through team andcompany-wide meetings to increase shared accountability
for the health of the culture and levels of engagement.

© 2024 Ultragenyx Pharmaceutical Inc

56

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Employee Engagement (cont.)
Results from our 2023 Survey

External Recognition

We conducted our most recent 18-month survey in September 2023 and included a set of 65 scaled items and four open-ended questions.

In addition to strong employee engagement results, our achievements over the past year havein our surveys with 90% of eligible employees participating.

Globe for the second consecutive year and also received the Cultural Excellence Awards from Top Work

Employees who have worked for Ultragenyx for at least 90 days were eligible to participate in the survey. We continued to have strong participation
In our most recent survey, we achieved or outperformed on all three of our Thriving Culture 2023 corporate goals, including Employee Engagement,
DEIB and Health & Well-being.

The following highlights some of our company-wide strengths:• Employees continue to have a strong sense of belonging and pride
in working for Ultragenyx, suggesting that they feel connected tosomething bigger than themselves.
• Manager effectiveness is a key strength for us as a company as
demonstrated by employees having a positive perception of theirdirect manager’s support of inclusion and diversity, and managers
creating an environment where people can grow and develop.
• Our rewards and recognition category continues

to be a strength, and we outperform benchmark data.

Company-wide actions on improvement opportunities identified
from the most recent employee engagement survey include:• Continue to embed principles and methodologies ofContinuous Improvement including Lean Six Sigma into
the way we work at Ultragenyx.
• Continue to increase connectivity and collaboration acrossdifferent functions, teams and the broader company.
• Continue to empower leaders and employees to improvethe effectiveness of their meetings* and embrace ourshared best practices.

consistently been recognized. Notably, we were named one of the Top Places to Work by the Boston

Places for Diversity, Equity & Inclusion Practices, Appreciation, Employee Well-being, and Professional

Development. Furthermore, U.S. News & World Report named us as one of Healthcare’s Best Companies
to Work For, and we were recognized as one of the Best Places for Working Families®.

Employee Recognition
Our recognition programs are designed to contribute to fostering a supportive, dynamic and inclusive

workplace, celebrating the exceptional contributions of our team members who embody our mission,

vision and values. The Ultra Leadership Recognition Program, Ultra Sharp Award Program, and theRare Pearl Spotlight Award – presented by the Empower Women in Biotech employee resource group(ERG) – honor individuals who demonstrate exemplary leadership, enrich our culture of inclusion, andgo above and beyond in their roles. Additionally, our “U Earned It” program and UltraRare Awardcelebrate achievements and everyday successes, fostering a culture of appreciation and excellence.

* This year, we added a set of questions to better understand the effectivenessof our meeting practices as this has been a hot topic for our leaders.

Employee Engagement Score
Enhancing employee engagement is one of our corporate goals. We measure this with a set of questions focused on employee pride in Ultragenyx,
whether an employee would recommend Ultragenyx as a good place to work, if employees feel a personal sense of accomplishment from theirwork, and their intent to remain with us. Over the last five years, we have continued to maintain a high employee engagement score, scoring 86%
overall in 2023, with 92% of our employees responding that they are proud to work at Ultragenyx, 88% feel a personal sense of accomplishmentfrom their work, and 88% feel like they can build and maintain strong working relationships with their colleagues no matter where they are working.

© 2024 Ultragenyx Pharmaceutical Inc

57

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

2023 Corporate Responsibility ReportGRI Index

Diversity, Equity, Inclusion and Belonging (DEIB)
We are committed to fostering ahealthy, inclusive environment while

nurturing a culture of belonging where

all employees have equal opportunities.
We strive to create an environment

where everyone we work with, serve andengage with feels valued, respected
and empowered.

Our evolution from Inclusion & Diversity (I&D) to Diversity,

Equity, Inclusion & Belonging (DEIB) broadens our mission.

This change, initiated by our DEIB leaders during a focused

workshop, reflects lessons learned from our ongoing journey
and a commitment to addressing challenges, uplifting thevoices of all groups, and driving meaningful change.

Our multi-year vision for DEIB aims at going beyond words,
with meaningful, tangible actions and results that reflect
our values. To realize this vision, we have identified four
strategic DEIB pillars:Highlights of our recent DEIB accomplishments include the following:
Hosted Pivotal DEIB Leaders’ Workshops

Interview Process Enhancement

members of our DEIB Action Team and leaders of our ERGs.

consistency and raise awareness of unconscious bias andOur offsite DEIB workshops/retreats brought together

These sessions were crucial for team building, developing ourleadership skills, critically assessing and enhancing our 20202025 DEIB Roadmap, aligning our efforts, and executing theroadmap’s action items. Participants reported that theseworkshops ended with a renewed commitment and focusedenergy toward our DEIB initiatives.

• People and Communities
We prioritize creating a safe, inclusive culture
for our people and the communities we serve.

UltraTalks 2023 – Engaging in Neuroaesthetics• Inclusive Leadership
We lead by example, aimed at integrating DEIB
into our activities.

discussing the science of neuroaesthetics, and the CIME team• Organizational Diversity
We focus on attracting, developing and promoting
diverse talent.

and Intersectional MediaThe 2023 UltraTalks featured Susan Magsamen and Ivy Ross

(Joy Donnell, Munika Lay and Nicole Haggard) sharing insights
on intersectional representation in movies and media. Thesetalks provided unique perspectives on how art and media

We revamped our Interview Guide and tools to increase
how it can show up in the interview process.
Launched Internal DEIB webpage

Our internal DEIB webpage centralizes our dedication
to diversity, equity, inclusion, and belonging. It serves

as a comprehensive resource detailing our DEIB strategy,
showcasing DEI metrics and highlighting our ERGs andcommunity group initiatives, along with engagement
opportunities.

Conducted Representation Audit

We shared department DEIB demographic data with eachfunctional area leader, highlighting potential gaps andopportunities to improve diversity and representation.

influence community strength and individual well-being.

• Accountability and Transparency
We commit to working to drive DEIB progress, integrate it
into our operations, and transparently share our progress.

© 2024 Ultragenyx Pharmaceutical Inc

58

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Workforce Data
All data as of December 31, 2023*
Number of Total Employees

1,276

Number of U.S. Employees

1,089% of employees who are women

56.2%% of U.S. employees who are women

56.6%% of employees with a disability

3.8%% of U.S. employees who self-reported as veterans

1.0%% of U.S. employees who self-reported and identified
as racially or ethnically diverse

45.8%***

Age Breakdown (Total Employees)

Asian

27.8%

Black/African American

5.6%

Hispanic or Latino

8.5%

White

54.0%

<30 years of age

6.3%

30-50 years of age

63.0%>50 years of age

30.6%

Number of Employees on the Executive Leadership
Team (XLT)

9

Number of XLT members who are women

1

Number of XLT members who self-reported and identified
as racially or ethnically diverse

2

Asian

25.6%

Black/African American

3.3%

Number of Women in Management Positions

220

Hispanic or Latino

6.2%% of women in all management positions(as % of total management positions**)

50.1%

White

% of women VP and above (as % of total management
positions**)

American Indian/Alaskan Native, Native Hawaiian
or Other Pacific Islander, Two or More Races

62.8%

43.9%

*U.S. employee data is consistent with the company’s submission on the U.S. Federal Employer
Information Report Equal Opportunity Form (EEO-1).

% of women in STEM-related positions(as % of total STEM positions)55.5%**Total management positions include all management positions at Ultragenyx,
including employees in manager roles.

© 2024 Ultragenyx Pharmaceutical Inc

American Indian/Alaskan Native, Native Hawaiian
or Other Pacific Islander, Two or More Races
Not Specified

Number of U.S. employees in management positions

3.7%

According to our 2023 internal employee* engagement survey, theperceptions of our managers’ commitment to creating an inclusiveand diverse environment improved compared to the 2022 results.

92% of our employees* feel that their managers support I&D in theworkplace, up two percentage points from 2022.

90% feel that their managers model inclusive behaviors, up two
percentage points from 2022.

87% feel that their managers create an environment where people
feel they belong, consistent with 2022.

*Refers to employees that participated in the survey.

0.4%

390

2.1%

***Due to rounding, the percent breakdowns for racial/ethnic minorities add up to <100%.

For information on diversity in our board of directors, see Corporate Governance.

59

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Employee Resource and Community Groups
Employee Resource Groups (ERGs)
and Community Groups (CGs) are

ERGs are created with the purpose of building inclusive spaces for individuals who share common aspects of identity.

CGs are designed to foster connections among employees

our business’s DEIB goals.

like-minded individuals to engage in activities and

ERGs are an integral part of our DEIB strategy, as they are structured with the objective of influencing and advancing

discussions related to their common interests.

voluntary, employee-led groups thathave a distinct impact on the culture
at Ultragenyx and are equipped witha charter, mission and support team.

The UltraProud ERG hosted its third
Annual Coming Out Day, featuring Alicia
Roth Weigel and Hope Giselle, who led
discussions on allyship.

© 2024 Ultragenyx Pharmaceutical Inc

Seeks to empower the LatinX community at Ultragenyx
to realize its fullest potential and encourages the next
generation of LatinX talent to pursue careers in scienceand biotechnology

with shared interests. These groups offer a platform forWorks to promotes a culture of equality and belonging
to enable LGBTQIA+ employees to thrive in their careers
and lives and achieve greater impact in the world

Creates a forum that celebrates the diverse cultures ofAsian and Pacific Islanders (APAC), promotes dialogue about
the APAC experience, and supports the career growth anddevelopment of APAC employees at Ultragenyx

Seeks to create a supportive culture to encourage
women, diversity and equity in leadership and science

A mix of ethnic groups and cultures that coexist withsociety and here at Ultragenyx, with the aim of recognizing,celebrating and using our gifts, abilities and resources tosupport one another and the company

Seeks to enhance Ultragenyx’s remote working
experience to provide employees with a senseof belonging even while working from home

Connects employees with
opportunities to support
nonprofit organizations assistingunderserved communities

Supports employees in taking aholistic approach to their wellbeing and work-life balancethrough a variety of events,
initiatives and classes

Plans events, including fun popups, happy hours, watch parties
and annual company parties

60

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

DEIB in Recruitment
We have an intentional approach torecruitment that focuses on creating

a welcoming and positive experiencefor each candidate. We aim to create asense of belonging from the very start
to foster an inclusive workforce.

Our intention is to create a diverse pipeline of candidates

In 2023

applying for jobs at Ultragenyx by using multiple strategies,
such as diverse interview teams to “screen in” instead ofMore than 35% of new hires camethrough our employee referral program.

“out” to mitigate bias, and hosting virtual open houses andcareer fairs to support our broad outreach efforts. We also

provide interview skills training to our employees to support

More than half of our 120 new U.S.
hires self-reported as membersof diverse populations.

inclusive interviewing and a deepened understanding of how
unconscious bias shows up in the interview process and cancreate barriers for those who are underrepresented in theworkforce. This training supports our ultimate company goalsof being an employer of choice for diverse talent and to havecandidates and new employees feel a sense of belonging
from the very start of their relationship with Ultragenyx.

Additionally, we continued our partnership with Disability

Solutions (DS) on outcomes for talent with disabilities, andUltragenyx’s jobs became live on the Disability Solutions’

Career Center. The DS Career Center works with Ultragenyx
to drive traffic to our job postings by actively recruiting

candidates with disabilities from their large network of local
and national workforce community partners.

© 2024 Ultragenyx Pharmaceutical Inc

Through our intentional approach, we have seen an increasein the number of diverse candidates interviewing forpotential employment as well as the number of new hires
who self-report as members of diverse populations.

61

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Occupational Health, Safety and Wellness
The health and safety of ourworkforce is a key priority.

Our health and safety management system is vital to a safe and healthy work environment and includes several elements, such as Global Environmental, Health, Safety and Sustainability(EHSS) standards, site-specific standard operating procedures, incident and safety observation reporting, hazard identification and risk assessments, job safety analyses, ergonomic assessments

and industrial hygiene evaluations. The system is based on the principles of ISO 45001:2018, the International Organization for Standardization (ISO) standard for occupational health and safety
management, and is designed to help us comply with applicable statutory and regulatory requirements and Ultragenyx policies, proactively identify and prioritize occupational health andsafety risks and potential mitigation options for our operations, and drive continued improvement across our global operations. We continue to search for opportunities to improve. In 2023,

as part of our efforts to advance health and safety management across our business, we focused on the ongoing development, improvement and effective implementation of our health andsafety management system and reporting processes. In 2023, we rolled out the high hazard work standard and developed a contractor safety management standard, which is expected to berolled out in 2024.
2023 Highlights:
Developed and published monthly

Implemented a New Hire EHS Checklist

warehouse safety and lockout tagout

Completed more than 140 ergonomic

an enterprise-wide view on incidents,

with site-specific safety procedures suchtraining courses to 860 employees in 2023.

identify and mitigate potential muscular

EHSS Performance Reports to provide
compliance, targets and training tostakeholders and the executive sponsor.
Strengthened key Global EHSS Standards

as evacuation routes and emergency
equipment. A total of 315 new hire

checklists were completed in 2023.

for Waste Management, Occupational

Rolled out laboratory safety refresher

and High Hazard Work Activities.

reinforce hazard identification and safe

Health, Office Safety, Contractor Safety
Enhanced our safety culture by establishing

training to more than 200 employees towork practices in our laboratories.

and communicating targets to increase

Rolled out comprehensive EHS training

preventative ergonomic assessments, andGTMF, including such topics as site-specificsafety observation reporting, expand

complete hazard assessments for our top
chemicals of concern.
© 2024 Ultragenyx Pharmaceutical Inc

to familiarize employees and contractors

for manufacturing employees at the new
EHS training, biological safety, hazardous

waste management, respiratory protection,

procedures. We provided more than 50

Established a safety observation system

to engage our front-line employees in thesafety improvement process, which resulted
in the proactive reporting and addressingof over 300 safety hazards.

Global EHSS conducted industrial hygiene

surveys at our manufacturing and R&D sites,
collecting 70 samples for chemicals suchas formaldehyde, potent pharmaceuticalcompounds, anesthetic gases, disinfectants

and solvents. We also conducted surveys fornoise and indoor air quality.

hazard assessments for employees toskeletal injuries. In 2023, we emphasizedpromoting ergonomic assessments, withthe majority being proactive rather thandue to discomfort.

Improved our internal hazardous waste
management program by establishingmembership with CHWMEG, a non-profit

association that promotes responsible waste
stewardship, and performing due diligence

reviews of 6 treatment, storage and disposal
facility audit reports to proactively assess
and mitigate environmental liability.

62

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Occupational Health, Safety and Wellness (cont.)
Wellness
In addition to formalizing our new flexible and remote work models, we continue to provide our employees with wellness offerings to support their physical and mental health.
Spring Health offers mental wellness screening, stress management, coaching

Caring for U is a global reimbursement program offering employees up to

year to employees globally and covered dependents.

includes fitness classes, gym memberships, childcare, eldercare, meal delivery

and up to four free virtual therapy sessions and two free psychiatry sessions each

$1,200 annually (in local currency) for wellness and caregiving activities. This

2023 Safety Data*
Lost Time Incident Frequency Rate (LTIFR)**

0.83

Total Recordable Incident Rate (TRIR)***

0.58

Number of Fatalities – Employees

0

Number of Fatalities – Contractors

0

Mindfulness and Meditation programs as well as informal support groups arefor dependents, and pet walking, supporting a well-rounded, healthy lifestyleand caregiving responsibilities.

* Data covers employees and contractors that are directly supervised on a day-to-day basis

Annual Flu Shot Clinics are available in offices. Flu shots are covered for free inAccess to a Personal Health Advocate is available to assist our U.S. employees** LTIFR = (Number of lost-time injuries) / (Total hours worked) x 1,000,000

their family members outside the U.S. Additionally, COVID-19 rapid antigen tests

Ergonomics Assessments are offered to help employees adjust their workstations

available to employees struggling with caregiving, isolation and stress.

the U.S. for employees plus family members and are reimbursed for employees andand their families with navigating the healthcare system and maximizing benefits.

are available globally in offices at no charge.

and equipment for long-term comfort and physical health.

Emergency Response and Preparedness*** TRIR = (Number of recordable incidents) / (Total number of hours worked) x 200,000

Return to Office Update
In 2023, as COVID-19 transitioned to an endemic state and more

Our Global EHSS standard establishes the requirements for emergency preparedness and response planning and our site-specific Emergency Response and Preparedness

treatments became readily available, we sunsetted our vaccine

chain of command, identification of resources to provide rescue and medical services, and evacuation and shelter-in-place procedures.

relaxed our COVID-19 Health and Safety guidelines. These decisions

Plans (ERPP) are implemented to protect our workforce in the event of an emergency. Site-specific ERPPs include procedures for reporting emergencies, a clearly definedrequirement for employees, contractors and temporary workers andWe conduct emergency evacuation drills at least annually, designed to familiarize our workforce with site emergency procedures, establish proficiency in executing

were made after careful consideration of the most recent medical,

for ERPP improvement.

and senior leadership.

an orderly evacuation, and determine accurate occupant accountability. Reviews are carried out after each drill to identify the effectiveness and opportunities

scientific and government data, analyzed by the COVID-19 Task Force

In 2023, we implemented a mass notification system to rapidly alert our workforce of emergency situations or potential threats and direct them on how to respond.

© 2024 Ultragenyx Pharmaceutical Inc

63

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

2023 Corporate Responsibility ReportGRI Index

Employee Compensation and Benefits
We recognize that our employees
are our greatest asset, and their

dedication and talent are the driving
forces behind our success. To honor

this commitment, we offer competitivecompensation and benefits packages
aimed at attracting, retaining andmotivating top industry talent.

Our approach to compensation is rooted in fairness andin similar roles. To strengthen our endeavors, we leveragedepartments and demographic groups. With real-timeThis strategy aligns our organizational culture and mission

monitor and manage pay equity practices. This includes

principles throughout the hiring process and employment

equity, operating within a pay-for-performance framework.
with our goal of providing equitable, transparent andunbiased remuneration for our employees. Our compensation
and benefits package offers extensive support for health,family and financial well-being. It includes health, life anddisability insurance, 401(k) matching, cash bonuses, equity
awards, paid time off for volunteering, wellness programsand tuition reimbursement.

We base compensation decisions on multiple factors, includingrole, performance, location, relevant experience, external

and internal peer data, and professional contributions. These

decisions are thoroughly reviewed by senior leadership to help
promote fairness and consistency across the company, alignwith industry benchmarks and maintain competitiveness. Thisapproach not only helps us attract and retain top talent but
also fosters a culture of inclusivity and meritocracy.

We are firmly committed to maintaining pay equity for

all our employees worldwide, an integral component ofour broader focus on DEIB. As part of this commitment,
we regularly conduct thorough pay analyses designed

to assess and uphold equitable compensation practices.

These assessments take into account various factors suchas performance, experience, level, tenure and location to
identify any potential pay disparities among employees© 2024 Ultragenyx Pharmaceutical Inc

advanced software that enhances our capacity to effectively
utilizing sophisticated data analytics, which enable acomprehensive examination of compensation across roles,

monitoring capabilities, we track adherence to our pay equity
lifecycle, facilitating prompt resolution of any emerging issues.

Key compensation practices include:• Benchmarking process: We review each position
for an appropriate pay range by referencing externaltalent markets, utilizing third-party benchmark
data, and considering internal equity. Our aim is tomaintain equitable and competitive compensation
within these established ranges.
• Transparency: We embrace transparent pay practices
by clearly defining salary ranges for every role andpromptly share this information with employees uponrequest. We offer extensive training and resources tohelp employees understand how their compensation
is assessed and calculated.
• Regular reviews: Regular reviews of ourcompensation policies and practices are integral
to our strategy. These reviews help in addressing
any disparities or inequities that may emerge.

• Customized compensation structures: Employees
commuting to an office in higher cost of living
areas receive higher salary increase budgets and arebenchmarked to salary ranges tailored to enable
them to support themselves and their families in theirlocale. Additionally, employees in our manufacturing
facilities are eligible for competitive shift differentials
and overtime to supplement base earnings. These
practices are aimed to provide that our workforce
is paid with fair and livable wages, incorporating
considerations of local economic landscapes, costof-living/labor metrics and industry standards.
• Performance-based rewards: Our compensation
structure includes performance-based cash andstock-based elements to recognize and appropriately
reward exceptional performance.

For information on median compensation and CEO pay ratio, see the Proxy.

64Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Employee Compensation and Benefits (cont.)
Our Benefit Programs2023 Compensation and
Benefits Enhancements

Our benefit programs provide employees and their families
with access to a suite of innovative programs that aredesigned to enhance their physical, emotional, familial,
financial and social well-being, plus additional perks tosupport employees both in and outside the office. Our

A flexible work model

programs include a comprehensive selection of medical,

12 weeks of paid familycare leave with no waiting
period$1,200 per year
in wellness credits

Paid holiday weeks inAugust and December

dental and vision plans; retirement savings options;competitive paid time off; and other initiatives that support

Improved our company-paid disability
coverage, providing additional income
protection.

balancing work with life. Eligible employees participate
in our annual short-term and long-term, equity-based

incentive programs, which provide opportunities to sharein our company’s success.

Paid volunteer time:
up to 16 hours
per year

Employee stock purchaseplan with sharesdiscounted 15%

More information can be found on our Career webpage.

© 2024 Ultragenyx Pharmaceutical Inc

Increased the employer contribution
to the Health Savings Account (HSA)
for employees enrolled in the High
Deductible Health Plan.

Regularly hosted
UltraTalks from a variety ofguest speakers, designed
to spark new ways ofthinking

Robust employee inclusionand development
programs

Launched a voluntary Long Term Careinsurance program for U.S. employees.
Introduced a new performance andbonus evaluation framework in 2023,
focusing on goal achievement, job
performance, and value alignment
to enhance clarity, alignment,
empowerment, and transparency.

65

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Communities
Aligning our corporate philanthropic efforts
with our mission and purpose

We are committed to supporting initiatives that provide
meaningful impact for the rare disease community;
public health and access to care; Science, Technology,
Engineering, Arts and Mathematics (STEAM) education;and local, at-risk communities.

© 2024 Ultragenyx Pharmaceutical Inc

66

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Aspiration
To make a positive impact

Our Objectives

2023 Progress

we operate and beyond.

Support charitable organizations that contributemeaningfully to the health and well-being of thecommunities where we operate and beyond• Approved more than $2.5 million in charitable donations, independent medical education and health-related grants
across 21 countries

Expand opportunities for employee volunteerism• Partnered with Life Science Cares and other organizations to launch the inaugural Ultragenyx Days of Service in the
U.S., Canada, EMEA (Europe, Middle East and Africa) and LatAm (Latin America) regions, with employees completing
more than 30 service projects

in the communities where• Provided support to organizations focused on emergency and humanitarian relief, including contributions toearthquake efforts in Turkey and Syria, storm relief in Pajaro, California, wildfire assistance in Maui, Hawaii, andhurricane relief in Acapulco, Mexico• More than 50% of employees signed up to participate in Days of Service projects© 2024 Ultragenyx Pharmaceutical Inc

67

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Charitable Giving
Our charitable giving mission builds
on Ultragenyx’s broader mission totransform the lives of people livingwith rare disease.

We direct our charitable giving to our local communities and beyond, and we recognize that healthy lives are supported byhealthy communities and environments, including education and access to healthcare, and emergency relief aid during timesof crisis. Our corporate philanthropy is focused on the following priorities:• Rare disease community support• Local, at-risk communities• Equitable healthcare• Emergency relief aid• STEAM education
We are committed to supporting initiatives that we believe provide impactful resources for each of these priorities.

In 2023
approved more than $2.5 million incharitable donations, independent
medical education and health-related
grants across 21 countries.

Employee Giving and Volunteering
Ultragenyx offers employees the opportunity to take two(ERG) called UltraGiving that is committed to connecting

time giving back to our communities and contribute to localorganizations assisting underserved communities. Once

paid volunteer days each year (16 hours), so they can spend
initiatives. In 2023, more than 450 full-time and part-time
employees recorded donating more than 2,900 hours fornumerous volunteer activities, including supporting STEAM
events, raising money for unhoused communities, working
at local food banks, participating in holiday gift drives,

building hygiene kits, and working with family-to-family

programs. Additionally, we have an employee resource group© 2024 Ultragenyx Pharmaceutical Inc

employees with opportunities to support nonprofit

the connections are made, employees can choose to use

their paid volunteer time, volunteer on their own, or makedonations. In some cases, the organizations are the same as
those supported by corporate giving; in others, employees
identify different organizations they wish to support.

UltraGiving has chapters in the San Francisco Bay andGreater Boston areas, Utah and Latin America.

68

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility ReportInaugural Days of Service
During our inaugural Days of Service, more than 50% of our employees worldwide registered to participate in numerous impactful volunteer activities,

embodying our commitment to social impact and underscoring the power of collective action in making a tangible difference in our local communities.
Throughout the campaign, employees completed more than 30 service projects around the world.
KEY HIGHLIGHTS FROM THIS INITIATIVE INCLUDE:
Enjoyable and Rewarding Experiences

Employees across various locations actively participated in diverse

volunteer activities, finding joy and fulfillment in stepping away fromroutine work to serve others.

Strengthening Community Ties

Whether volunteering at food banks, schools or nature preserves, our teammembers deepened their connections to local communities.
Significant Impact and Outreach• 5,100 care packages assembled in California, including STEM
and hygiene kits, benefiting various nonprofit organizations• 800 humanitarian kits provided for Syrian war refugees by our
Canada team

• 880 care packages assembled in Massachusetts, including STEM kits
and school supplies, supporting local education and foster care
• 750 emergency necessity kits distributed by our Miami LATAM team,aiding earthquake and war victims• Environmental conservation efforts across LATAM, EMEA
and the U.S., including:-Cleaning up the Xochimilco Lake area in Mexico as partof a nature conservation effort-

Planting 30 native species in Colombia-

Removing more than 300 lbs (138 kg) of waste fromthe Llobregat River in Spain• Over 120 to-go meals donated in Salt Lake City to aid
the unhoused community• Many additional hours volunteered across various programs,
focusing on education, health, environmental conservation,and emergency relief

Employees completed more than 30 service

projects around the world during our inauguralDays of Service campaign.

© 2024 Ultragenyx Pharmaceutical Inc

69

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

2023 Corporate Responsibility ReportGRI Index

Rare Disease Community Support
We partner with organizations thatIn 2023, Ultragenyx proudly supported:share in our mission to transform thelives of people living with rare disease.
Our rare disease community support

includes patient and sibling programs,
respite care and support programs forcare partners, and awareness initiatives.

Camp Korey is a medically safe camp experience that offers

Ultragenyx made a contribution to Parent to Parent USA, atreatments that often overshadow childhood. The objective

informational support to families with children who havechildren and families an escape from the endless medical

is to create empowering and adaptive year-round programsfor children and their families living with life-altering medical
conditions, free of charge. Ultragenyx funded the overnightsummer program where more than 270 children aged 7 to 17
with complex medical conditions and their family members
spent four to five nights at camp enjoying activities suchas Archery, Arts & Crafts, Silly Olympics, Fishing & Boating,
Swimming, Pet Therapy and Stage Night.

© 2024 Ultragenyx Pharmaceutical Inc

nonprofit organization dedicated to offering emotional andspecial needs. The donation was directed toward supporting
the P2P USA’s Leadership Institute, a live online event thatbrought together nearly 200 parents and staff for three anda half days of professional development and topics that
affect parents of individuals with disabilities.

A Rare Affair
Ultragenyx hosted the 2023 Rare Affair fundraising
event, now in its 11th year. Each year the Rare

Affair has focused on raising money and visibility

for a different rare disease advocacy organization.
The 2023 event gathered more than 250 biotech
executives, venture capitalists and rare diseaseadvocates and successfully generated donations
to the Osteogenesis Imperfecta Foundation(OI Foundation) to support its mission in OI
research and advocacy.

70Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

GrantsIn addition to our philanthropic

efforts, we support the rare diseasecommunity through educational

initiatives, patient advocacy, research
and access to information.

Our grant support aims to enhance awareness, advance the

To promote compliance with applicable standards anddiseases, and empower healthcare professionals to bridgethat is based on:

medical and scientific understanding of rare and ultrarareclinical, research and practice gaps. Our specific focus is on
patient and professional organizations that help improveawareness, care and access, and provide vital support to therare disease community.

OUR GRANT SUPPORT INCLUDES:

guidelines, our funding undergoes rigorous evaluation,• The Accreditation Council for Continuing Medical
Education (ACCME) Standards for Commercial
Support (SCS)• The American Medical Association (AMA) Ethical
Guidelines for Gifts to Physicians from Industry

Independent Medical Education• The FDA Guidance for Industry: Industry Supported
Scientific and Educational Activities

technical and scientific programs, as well as continuing• PhRMA Code on Interactions with
Healthcare Professionals

Supporting both accredited and nonaccredited clinical,

medical education (CME) activities focused on rare diseases
for healthcare providers.

Health-Related Grant Funding

Sponsorships and grants for nonprofit patient organizations

and for-profit health institutions for multiple initiatives. These

Please see our Grants webpage for the latest list of areas
currently being considered for funding.

In 2023
More than 100 organizations focusedon medical research and treatment,
and rare disease awareness, education
and advocacy located in 20 countries
received health-related grants totalingnearly $2 million
More than 20 organizations in 10
countries received independent medical
grants totaling approximately $0.4
million, with over 2,000 healthcare
professionals educated through these
supported independent medical
education programs

include patient advocacy-focused initiatives, non-accreditedscientific meetings such as conferences, summits andforums, fundraising and disease awareness events, researchand educational programs, strategic partnerships, medical
publications in Europe and Latin America, and fellowships.

© 2024 Ultragenyx Pharmaceutical Inc

71

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Empowering Patient Advocates
Elsie Evans has had a profound journey living with homozygous familial

Elsie’s story also underscores the significance of community and connection forfor patient education and support. Diagnosed with HoFH when she was 3.5 years old, Elsie

fostered a network of support and shared knowledge, strengthening the bond among

hypercholesterolemia (HoFH), a rare lipid condition, and becoming a passionate advocate
faced numerous health challenges, including heart attacks in her early 20s and the needfor regular apheresis treatment.

individuals battling rare diseases. By sharing her experiences and challenges, Elsie haspatients facing similar struggles. This sense of community is vital for emotional support
and collective growth, helping patients navigate their journeys with greater confidence

Her transition from a career in education to a role in patient advocacy is a testament toand understanding.

Europe Foundation and Heart UK, focusing on educating and supporting patients living

groups that play a crucial role in enhancing the lives of those affected by rare diseases.

her dedication to making a difference. Elsie now contributes significantly to the EFFORTWe are committed to supporting remarkable individuals such as Elsie and patient advocacy

with rare conditions such as HoFH. Her work emphasizes the importance of informed

We strive to bridge the gaps between patients, healthcare providers and researchers,

treatment choices and a deeper understanding of rare conditions. Elsie’s experience withtreatments, particularly apheresis, highlights the varied and complex nature of managing
HoFH and underscores the crucial need for treatments that do not adversely impact thepatient’s quality of life.

raising awareness about rare conditions and advocating for equitable healthcare access.
Elsie’s journey and her advocacy work shed light on the disparities in treatment accessand healthcare, reinforcing our dedication to creating a more inclusive and supportive
healthcare environment for all.

Elsie embodies the resilience and transformative power of patient advocacy in the realmof rare diseases. Her dedication to education and advocacy has not only improved her own
life but also promises to make a lasting impact on the rare disease community worldwide.

Elsie’s story underscores the significance of communityand connection for individuals battling rare diseases.

© 2024 Ultragenyx Pharmaceutical Inc

72

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Equitable Healthcare
We are committed to working tofoster a society where healthcare

is accessible to all, recognizing thisas a fundamental element of a

thriving and sustainable community.
Our efforts are focused on reducing
healthcare disparities, with the goalof everyone, regardless of background,gaining equal access to the healthservices they need.

We support organizations working to enhance publichealth and expand access to care for local and underserved
communities. Our equitable healthcare contributions focus
on public health initiatives, improving access to services,and promoting wellness in these communities, all with thegoal of reducing healthcare disparities.
2023 HIGHLIGHTS

Inaugural Days of Service• In California, team members assembled 1,600 hygienekits benefiting California’s underserved populations.
This effort benefitted a variety of Bay Area nonprofit
organizations and programs, including 650 hygienekits for Ritter Center, 150 for North Marin Community
Services, 300 for HealthRIGHT360, and 500 for Ayudando
Latinos A Soñar (ALAS).

• The Brazil LATAM team members volunteered at Lar da
Criança Ninho de Paz in São Paulo, an organization caring
for individuals aged 15-41 with severe cerebral palsy. Our
team supported the 13 residents of Ninho de Paz withboth in-kind and monetary donations.

Transforming Lives in Colombia

Donated to Fundacion Operacion Sonrisa that provide

more than 23,000 surgeries for children with cleft conditions,leveraging a network of 200 specialist volunteers forcomprehensive care.

Promoting Public Health and Wellness

Partnered with Life Science Cares, offering Ultragenyx
employees volunteer opportunities to support healthinitiatives in the San Francisco Bay and Greater Boston areas.
Supporting Pediatric Healthcare

Our UltraGiving team joined the Intermountain Foundation at
The Festival of Trees fundraiser, benefiting Primary Children’s

Hospital through a silent auction of holiday decorations to aid
needy families and enhance pediatric care.

Employees in California packing hygiene kits for Ritter Center during Ultragenyx’s inaugural Days of Service.

© 2024 Ultragenyx Pharmaceutical Inc

73

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

STEAM Education
We support locally-implemented
initiatives and organizations thatinspire and advance the developmentof the next generation of leaders inScience, Technology, Engineering,

the Arts, and Mathematics (STEAM),
thereby empowering young minds
irrespective of their backgrounds.

Our STEAM education giving targets include diversity, equity and inclusion (DEI) initiatives, scholarships and science events or programs.
2023 HIGHLIGHTS
Partnership with Biotech Partners

Back-to-School Support

opportunities for students pursuing careers in STEAM fields.

community organizations such as East End House and Homeward Bound. Our

We provided financial support to Biotech Partners, enhancing educational
Empowering Young Women

employees generously donated approximately 150 filled backpacks and volunteered

and nearby areas, enabling their participation in 10 mini workshops. These

UltraMosaic ERG’s Contribution

Your Horizons to inspire young women to explore STEAM careers.

the UltraMosaic ERG donated $5,000 to the scholarship fund at Alabama A&M

We sponsored more than 50 seventh and eighth-grade girls from Sonoma County

their time to assemble them.

workshops, led by female scientists and engineers, were organized by Expanding

Demonstrating our commitment to higher education and diversity in STEAM,

Promoting Creativity in STEAM

University, supporting students in their academic pursuits.

In Massachusetts, we supported students chosen by Bedford Creativity to compete

in Destination Imagination’s global finals. This competition encourages students to

Inaugural Days of Service

apply their STEAM knowledge creatively to solve complex, real-world problems.

• In Massachusetts, team members assembled 880 care packages, including
STEM kits, school backpacks and duffle bag care kits. This effort benefited
the School on Wheels and Foster Love organizations.

Investment in Scientific Adventure for Girls

Our donation of $25,000 to Scientific Adventure for Girls supported engaging

and educational afterschool STEM programs for young girls in the San Francisco
East Bay Area. Additionally, we extended our support to the Buck Institute andparticipated in the North Bay Science Discovery Day, hosting a student-centeredexhibit that was visited by more than 8,000 visitors, mostly families with children
under the age of 10.

© 2024 Ultragenyx Pharmaceutical Inc

Our teams in Boston and San Francisco organized supply drives to assist local• In California, team members assembled 3,500 STEM and final exam care kits
and school backpacks. This effort benefitted a variety of Bay Area nonprofit
organizations and programs, including 1,000 STEM kits for WEAREPIEFEST,
1,000 STEM kits for Scientific Adventures for Girls, 500 backpacks and supplies
for Ritter Center and North Marin Community Services. Upward Scholars,
Breakthrough Collaborative SF, Guardian Scholar Program, and HARTS Program
together received 1,000 final exam care kits.

74Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

2023 Corporate Responsibility ReportGRI Index

Local and At-Risk Communities
We are committed to helping
to nurture and improve local

communities, foster closer-knit bonds,
and create a positive impact thatprovides all individuals, especially thosein challenging circumstances, access toessential resources and opportunities.

Beyond corporate giving, our employees also donate

through the UltraGiving ERG to advance philanthropy

through volunteer events, fundraising and other efforts that
aim to benefit local and at-risk communities. Additionally,

Ultragenyx employees volunteer their time to serve on theboards of directors for local nonprofits, such as North Marin

Community Services and the Novato Chamber of Commerce.
Heroes and Helpers Grant

Ultragenyx provided a grant to the Heroes and Helpers

Program, run by North Marin Community Services (NMCS) in

partnership with the Novato Police Department and the Novato
Fire Protection District. The grant allowed 10 children fromthe NMCS Child Development or Case Management Programs
to shop for holiday gifts for their family members, assisted bya police officer or a firefighter, the heroes and helpers.
Support for Hunger Relief

Ultragenyx supported the Alameda County Food Bank in its

hunger-relief efforts to provide meals to at-risk communities.
Spreading Joy with Toys

Our UltraGiving team in Salt Lake City collaborated with Tiny
Tim’s Toys to build wooden toy cars for children in several
countries, including the U.S., Zimbabwe, Iraq, Afghanistan

and others. In the U.S., the toys were distributed to hospitals,
including Primary Children’s and Shriner’s, for patientenjoyment. Additionally, our UltraGiving team in EMEA donatedwrapped gifts for 30 refugee children aged 2 to 18 years.
© 2024 Ultragenyx Pharmaceutical Inc

Inaugural Days of Service• In Salt Lake City, Ultragenyx collaborated with The Road
Home to support unhoused individuals and families. Morethan 120 to-go meals were donated and delivered, andteam members spent two days serving hot lunches at theorganization’s downtown facility.
• In California, employees volunteered to bag fruits andvegetables at Redwood Empire Foodbank and SF-Marin
Food Bank and serve meals at Family House.
• Employees also took part in environmental conservation
efforts across LATAM, EMEA and the U.S., including:– In Massachusetts, employees volunteered to remove
trash from parks and local waterways via a partnershipwith the Mystic River Watershed Association and theCharles River Conservancy, mulch and weed at The
Food Project farm, and assemble meals with theCommunity Servings organization.
– In California, employees volunteered at Marin County
Parks to plant monarch butterfly habitats in green
space, and to mulch and weed at Florence Fang
Community Farm.
– The Miami LATAM team actively participated in tree
planting and the cleanup of local lakes and rivers.

– The Argentina LATAM team partnered with Fundación
Manos Verdes for a river cleanup and collected 15
garden-sized bags of trash.

– The Colombia LATAM team partnered with Fundación
Red de Arboles, planting 30 different native species andeliminating invasive ones, as well as cleaning the area.

– The Mexico LATAM team collaborated with Voluntarios
México in cleaning up the Xochimilco Lake area aspart of a nature conservation effort.
– EMEA team members, in collaboration with
Ecoherencia, volunteered by cleaning the banks of the
Llobregat River in Barcelona, Spain, and successfully
removed more than 300 lbs (138 kg) of trash.
Additionally, they constructed 25 birdhouses to support
local bird species in the river’s man-made areas.
Cross-ERG Gift Drives

The EmpowerX Women in Biotech ERG partnered with

UltraGiving team members to organize year-end gift drives:• Supported the work of Wonderfund, an organizationthat serves children engaged with the Massachusetts
Department of Children and Families (DCF). Wonderfund
works directly with social workers to meet the individual
needs of children and helps create magical holiday
moments for children throughout the state. Ultragenyx
employees provided holiday gifts and clothing for 125
local families in Massachusetts.
• Sponsored 66 children supported by the Marin Foster
Care Association. Each child provided a wishlist of three
items. Ultragenyx employees fulfilled all of these wishes
in addition to providing stocking stuffer gifts.

75

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Emergency Aid Relief
We prioritize emergency relief aid

as part of our mission, offering rapid

assistance during humanitarian crises,
especially in the realms of publichealth and medical services.

In 2023, we directed our support to organizations dedicatedto offering emergency and humanitarian relief in theaftermath of several natural disasters:• Contributed to the Turkey and Syria earthquake relief
efforts through Direct Relief & AHBAP.
• Supported the Pajaro levee storm relief via theCommunity Foundation for Monterey County.
• Participated in wildfire relief efforts through the Maui
Strong Fund of the Hawai‘i Community Foundation.
• Assisted in hurricane relief in Acapulco, Mexico, throughCruz Roja Mexicana.
• Our UltraGiving team in Canada partnered with
Global Medic and assembled 800 humanitarian kits
to be delivered to victims of the Syrian civil war.
• Inaugural Days of Service:– Our Miami LATAM team assembled 750 emergency
necessity kits for earthquake victims in Turkey andthose impacted by the war in Ukraine.
– In Canada, team members assembled 800
humanitarian kits for Syrian war refugees.
During Ultragenyx’s Inaugural Days of Service, employees in Miami, FL packed humanitarian aid kits to support those impacted
by the Turkey earthquake and the war in Ukraine.

© 2024 Ultragenyx Pharmaceutical Inc

76

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

PlanetReducing our environmental impact
and promoting sustainability

We are committed to developing an environmental
strategy that minimizes our environmental footprint
across our business.

© 2024 Ultragenyx Pharmaceutical Inc

77Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Aspiration
To conduct business in anenvironmentally responsible

manner and strive to continuously

Our Objectives

2023 ProgressContinue to implement improvements to reduce
our environmental footprint• Purchased 1,955+ megawatt-hours (MWh) of renewable electricity, which avoided an estimated 473 metric tons (MT)of carbon dioxide equivalent (CO2e); installed additional electric vehicle charging ports, bringing our total to 38• Strengthened our waste vendor partnerships to expand diversion and circularization programs

improve our performance tobenefit our employees, customers,

communities and the environment.

© 2024 Ultragenyx Pharmaceutical Inc• Launched My Green Lab certification pilot program at our Translational Sciences lab in Novato, California
Develop an environmental strategy• Continued and expanded the collection of environmental data across our facilities using our newly developed
procedures and policies; enhanced our global EHS&S policy to cover additional health, safety and sustainability topics• Considered sustainability impacts and opportunities across our supply chain78

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Reducing Environmental Impacts
Ultragenyx is committed to working

to reduce the environmental impactof our operations by enhancing andpromoting sustainable practices

throughout our office, laboratory
and manufacturing spaces, bothleased and owned. We design ourfacilities with sustainability in mind.

Over the past two years, we have developed policies

and procedures for data collection in alignment with the

New Space with Sustainability in Mindfor tracking and reporting environmental information,

During 2023, we relocated our team in Cambridge, Massachusetts to a brand-new space in Somerville, Massachusetts,

by location. Additionally, the established procedures areby this team. The Real Estate and Capital Projects team carefully selected this new Somerville location, noting the

key performance indicators. In 2023, we made progress in

The building, located in the Brickbottom district of Somerville, is expected to achieve Platinum status under the U.S.

designed to assist in monitoring our performance. Alongside(BD+C) Core and Shell standard.

Greenhouse Gas (GHG) Protocol. The policies set standards
facilitating the analysis of our environmental impacts

to accommodate our need for additional space and modern facilities essential for the vital work being carried out

designed to support the comparability and reliability of ourenergy efficiency and accessibility to the MBTA Green Line East Somerville train stop.

automating data collection and in implementing a dashboard

Green Building Council’s LEED (Leadership in Energy and Environmental Design) for Building Design + Construction

our efforts in benchmarking and analytics, we anticipate

these programmatic improvements will enable our teams

to develop environmental initiatives with greater efficiency.
Ultragenyx acknowledges the impacts and risks posed by

Designed with a commitment to environmental sustainability, the building emphasizes clean indoor air quality andample natural lighting. It also features a range of high-performance technologies aimed at reducing energy and water
consumption, thereby lessening our impact on the city’s resources.

climate change on our business operations and stakeholders.

Our strategy incorporates senior management level oversight
to comply with environmental regulations as well as manage
potential climate-related risks and opportunities to support
business continuity. We are taking steps to implement

governance processes designed to manage and mitigate
climate-related risks and impacts.

© 2024 Ultragenyx Pharmaceutical Inc

79

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Reducing Environmental Impacts (cont.)
We have strategically implemented a series of initiatives aimed at reducing our environmental footprint:
Renewable Energy

Sustainable Transportation

Eco-Efficient Facility Upgrades

Green program for our corporate headquarters campus in Novato, California.

we offer 38 electric vehicle (EV) charging stations across our California and

HVAC systems with more energy-efficient models and programmed these systems

We purchase 100% renewable electricity through Marin Clean Energy’s Deep
Through the program, we are purchasing Green-e® certified renewable electricity
from solar and wind sources and avoiding GHG emissions associated with ourelectricity use. Purchasing renewable electricity supports the City of Novato’s

Climate Change Action Plan, which outlines strategies for the city to achieve a
GHG reduction target of 40% below 2005 levels by 2035. In 2023, Ultragenyx
purchased 1,955+ MWh of renewable electricity through this initiative, which
avoided an estimated 473 MT CO2e.

To reduce commuting emissions and encourage sustainable urban mobility,
Massachusetts facilities, including 4 ADA-compliant connections, to support

employees who drive electric vehicles. Additionally, we promote the use of public
transportation through comprehensive commuter reimbursement benefits. Our

new Somerville facility in Massachusetts was strategically selected for its proximity
to the MBTA Green Line East Somerville stop.
Sustainability in Lab Operations

In 2022, the Laboratory Operations team implemented a new management

system designed to enhance the tracking and review of frozen sample inventory,

Our facilities team has converted to LED lighting in all common spaces; replaced
for setbacks during off hours; installed building management systems that have
improved efficiency in use of lighting, heating and cooling; and replaced main
passenger elevators with higher-efficiency models.
Improved Space Utilization

In 2023, our global facilities and engineering teams conducted a space utilizationassessment. This led to the consolidation of office buildings on our Novato,
California, campus and is expected to reduce energy consumption.

which improved cross-functional collaboration with scientists and study leads
and increased space efficiency, reclaiming nearly an entire freezer’s worth ofadditional storage. Concurrently, the team embarked on replacing outdated sample
freezers with newer, more energy-efficient models, a move expected to reduceenergy consumption over time. Building on these sustainable practices, in 2023

we launched a My Green Lab certification pilot at our Novato, California lab, withsignificant support from lab users and leadership. A cross-functional team is set
to implement eco-friendly changes in 2024 and aims to eventually extend thecertification to more labs.

© 2024 Ultragenyx Pharmaceutical Inc

80Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Engaging Our Workforce in Sustainability
Engaging our workforce inenvironmental sustainability is criticalto our objective to continue to improve
our performance and reduce ourenvironmental footprint. We recognize
the grassroots efforts to reduce ourenvironmental impacts and are proudof our workforce, who we believe areenvironmentally conscious.

In 2023, we launched a My Green Lab certification pilot

program at our Translational Sciences laboratory in Novato,
California. The My Green Lab Certification is a benchmark
for best practices in laboratory sustainability.

This initiative has gained strong support from both lab users
and executive leadership, with more than 60% of lab users
completing the baseline survey assessment. A dedicatedcross-functional working group plans to implement changes
based on the assessment findings throughout 2024.

Additionally, the group aims to extend the My Green Lab
Certification to more labs in the coming years.

During Ultragenyx’s inaugural Days of Service in May 2023,
employees in California and Massachusetts supported
several environment related volunteer projects. These

included gardening, mulching and weeding at Florence

Fang Community Farm in San Francisco, as well as waterway
cleanup and park beautification in Cambridge and Woburn,

GTMF Slash the Trash
In April 2023, in honor of Earth Day, members of our workforce at the Gene Therapy Manufacturing Facility (GTMF)
hosted a trash cleanup volunteer project in the industrial park surrounding the building.

Massachusetts.

© 2024 Ultragenyx Pharmaceutical Inc

81

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility ReportManaging Waste and Water
We work to comply with applicable

federal, state and local requirements
for the management of water andhazardous and non-hazardous wastes.

We have procedures in place, along with training andcompliance audits, to promote appropriate handling anddisposal of our waste streams. We partner with Polycarbin,a company dedicated to circular solutions designed

to transform today’s laboratory waste into tomorrow’slaboratory products, to recycle pipette tip boxes. Polycarbin’s
mailbox program follows a closed-loop recycling approach,where it receives lab plastics that might otherwise go tolandfill or downcycling and remanufactures the waste into
new lab products. Through this partnership, Ultragenyx

Managing Water
We continue to be conscious of our water footprint.
We have upgraded common area toilets and sinks

to low-flow models in an effort to conserve water. In

the future, we plan to continue to enhance our water
stewardship, particularly at facilities located in waterscarce areas in the U.S.

diverted more than 1,300 pounds of plastic waste from

Advancing Sustainability in Our
Wastewater Treatmenta single-stream recycling program across our sites.

In 2023, our global Facilities, Engineering and Capital

landfill since 2022. Furthermore, we have implemented

Since 2022
More than 1,300 pounds of laboratory
plastic waste diverted from landfill
through our Polycarbin partnership.

Projects teams, alongside external consultants andindustry experts, conducted a review of our wastewater

treatment at select East Coast facilities. As a result of thestudy, we introduced a new chemical decontamination

method to treat process and laboratory waste effluent at our lab in Woburn, Massachusetts, and GTMF in Bedford,

Massachusetts. These innovative processes replace thermal sanitization, an industry standard practice that is energy
intensive. Choosing chemical decontamination allows us to bypass the additional energy consumption and carbon
emissions associated with traditional lab practices.

Additionally, our wastewater pretreatment systems in Woburn and Bedford now use sodium hydroxide instead of

bleach for decontamination. This switch mitigates the potential for harmful byproducts associated with bleach use

and reduces the risk of adverse environmental impacts. We determined that lower volumes of a concentrated sodiumhydroxide solution are as effective as bleach in wastewater treatment, thereby minimizing chemical volumes required
for the process.
© 2024 Ultragenyx Pharmaceutical Inc

82

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Supply Chain Sustainability
Our commitment to operating in anenvironmentally responsible mannerextends to our supply chain.

We expect our suppliers to follow applicable environmental

Key examples of integrating sustainability within our supply

chemical and waste management, recycling, industrial• Consolidating small volume orders to help reduce thenumber of shipments and prioritizing direct-to-patient
shipments whenever feasible to eliminate the need forextra transportation and handling.

laws, regulations and standards, such as those concerning

wastewater treatment and discharge, air emissions controls,
environmental permits and environmental reporting. We

expect and encourage our suppliers, wherever possible, tosupport a proactive approach to environmental matters,
undertake initiatives to promote greater environmental

responsibility, and encourage environmentally preferable
technologies and sound life-cycle practices. These

expectations are laid out in our Global Standard for

Suppliers. Our teams work closely with internal and external

stakeholders to evaluate whether best practices are followed

chain include:• Conducting long-term stability studies for our productswith the goal to maximize shelf life for our products toprovide a reliable supply for patients and reduce disposal
needs and associated environmental impacts.
• Balancing shipping and packaging sustainability withpatients’ needs and industry requirements, such asutilizing sea freight in lieu of air freight whenever possible.

across various steps of our supply chain and whether vendors
are able to support our objectives.

© 2024 Ultragenyx Pharmaceutical Inc

83Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Governance

Maintaining robust corporate governance and risk management
and upholding high standards of honest and ethical business conduct

The foundation of our purpose to lead the future of raredisease medicine is built upon our commitments to strong
corporate governance, ethics and integrity, compliance,data protection and security, and responsible procurement.

© 2024 Ultragenyx Pharmaceutical Inc

84

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Aspiration
Through strong corporate

Our Objectives

2023 Progress

integrity, we seek to prevent

Act responsibly and with integrity and provide annual,targeted training to our workforce on Ultragenyx’s
ethical standards• ~90% of survey responders to the annual compliance culture survey stated that the annual compliance training
is adequate for them to confidently execute their responsibilities

Maintain a high compliance culture and adherence
to all applicable legal requirements• All reported complaints related to potential breaches to our code of conduct and incidents of discriminationor harassment were investigated and promptly addressed, as appropriate

Maintain a high rate of third-party due diligenceof our suppliers• Published a formal Global Human Rights policy

governance and a culture ofsignificant issues before they occur
and foster an environment where
issues can be disclosed without
the threat of retaliation.

© 2024 Ultragenyx Pharmaceutical Inc• Continued efforts to comply with the U.S. Department of Treasury’s OFAC regulations

85

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

2023 Corporate Responsibility ReportGRI Index

Corporate GovernanceWe believe that good corporate

governance promotes the long-term
interests of our stockholders andother stakeholders. We are committed
to maintaining good corporate

governance practices and periodicallyreviewing our practices.

Our Global Code of Conduct establishes principles andexpectations that apply globally to all employees, officers

and directors regardless of position or tenure. Our corporate
governance guidelines serve as a framework for conducting
the board’s business and assist the board in the exercise of

Board Diversity• Three are women• Average age of 60.4 years• Three self-identify as a racial or ethnic minority• Average tenure of 7.4 years• One self-identifies as LGBTQ+

its duties and responsibilities to serve the best interests of
Ultragenyx and its stockholders.

Board of Directors
Our board of directors provides us with strategic guidanceas we work to advance our mission to transform the lives ofpeople with rare disease. Our board comprises experienced
leaders who represent a diversity of talents, skills,
backgrounds and expertise.

Our board currently has a standing Audit Committee,

Compensation Committee, Nominating and GovernanceCommittee, and Research and Development Committee.

Each of these committees operates under a written chartersetting forth the functions and responsibilities of thecommittee, a copy of which is available on our website.

Examples of our commitment to good corporate governance include the applicationof the following:
Best Practices

Independence• Diverse board, including
adoption of “Rooney Rule” fordirector search process• All directors are independent
except our president and CEO• Ongoing shareholder
engagement program• Board oversight of sustainability
and corporate responsibility
matters• Minimum stock ownership
requirements for directors andnamed executive officers

directors, seven of whom are independent.

• More than 90% attendance ofboard and committee meetingsin 2023 by our current directors

For more information on our directors and corporate• Director Overboarding Policy

As of January 31, 2023, our board consisted of eight

governance, please see our Proxy Statement.

• Active independent board
chairman• 100% independent directors on
Audit Committee, Nominating
and Corporate GovernanceCommittee, and Compensation
Committee

Accountability• Director Resignation Policy fordirectors who receive less thanmajority support in uncontested
elections• Clawback Policy withdiscretionary recoupment
provisions beyond basic legal
requirements• Annual board and committee
self-evaluations• Prohibition against hedging
transactions

See also Corporate Responsibility Oversight.
© 2024 Ultragenyx Pharmaceutical Inc

86Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Corporate Responsibility Oversight
We are committed to integrating corporate

responsibility with our overall corporate strategy.

This commitment starts at the board of directors’level. The Nominating and Corporate GovernanceCommittee of our board regularly reviews andmakes recommendations on sustainability andcorporate responsibility matters including policies
and initiatives. Furthermore, the Corporate

Responsibility Working Group, an executive-

sponsored, cross-functionally represented grouptasked with advancing our progress, reports tothe executive leadership team on our progress.

© 2024 Ultragenyx Pharmaceutical Inc

87

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Risk Management
Ultragenyx’s board has overall

responsibility for the oversight ofthe company’s risk management

process, which is designed to support

the achievement of organizational andstrategic objectives to improve long-term organizational performance andenhance stockholder value.

The board periodically reviews our business strategy andmanagement’s assessment of the related key and emerging
risks and discusses with management the appropriate

level of risk for the company. In 2023, the board and thecommittees reviewed with management various risks
and mitigation strategies, including those related to:• Implications from the macroeconomic climate• The company’s initiatives related to corporate
responsibility and sustainability matters• Cybersecurity and security programs related
to our information technology systems• Human capital management, such as employee
retention and recruitment

Business Continuity Management and Disaster Recovery
Ultragenyx has established the fundamentals of a Business Continuity Management (BCM) and Disaster Recovery (DR)program, guided by best practices from organizations such as the International Organization for Standardization (ISO),
including ISO 22301. This program is aimed at supporting organizational resilience against disruptions in business
operations. The overarching goal is to continually improve practices and foster the uninterrupted continuation ofcritical business functions in the face of disruptions.

This program integrates several key elements: program governance for executive oversight and decision-making,business impact and risk assessments for strategy development, and crisis management and communications for

coordinating company-level responses. Responsibilities within Ultragenyx are clearly delineated. Leadership roles,
including Functional Leaders and Critical Process Owners, are tasked with identifying and safeguarding criticalprocesses under Business Continuity Plans (BCPs).

• The continued appropriateness of the company’sclassified board and other structural elementsof the company

BCPs are annually reviewed and regularly updated to reflect the evolving business environment. The implementation• The company’s approach to evaluating our clinicaland preclinical programs

maintaining uninterrupted business processes. To validate their effectiveness, we regularly perform and document

The board delegates oversight of certain risks to each board
committee, and each member of the executive leadershipteam is responsible for certain risk areas. Executive leadershipis responsible for establishing our business strategy, identifyingof BCPs is centrally managed through cloud-based software.

Additionally, we have IT Disaster Recovery plans that focus on restoring critical networks and systems essential forvarious recovery tests.

In 2023, we undertook a third-party assessment designed to enhance the maturity of the program. Future potential

enhancements under consideration include refining the company’s impact analysis, further enhancing the company’s
recovery and crisis plans, and expanding testing activities.

and assessing the related risks, and implementing appropriate
risk management practices. For a summary of risks anduncertainties related to our business and operations, see
Item 1A Risk Factors in our 2023 Annual Report.
© 2024 Ultragenyx Pharmaceutical Inc

88Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Risk Management (cont.)
The board of directors, Its committees and Ultragenyx’s executive leadership team oversee the company’s risk management program, which Includes periodic reportingand open lines of communication.

© 2024 Ultragenyx Pharmaceutical Inc

89

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Ethics and Integrity
Ultragenyx is committed to upholding
high standards of honest and ethicalbusiness conduct as the foundation

upon which we build our reputation.

We expect every director, officer, employee and supplier to

We work to uphold a high compliance culture by requiring ethical behavior, holding each individual accountable forboth within our company and with our customers, businessapplicable laws and regulations while maintaining patient safety and leadership accountability. We developed our complianceadhere to high ethical standards in all business interactions,partners, competitors and the communities we partnerwith and where we operate. We have cultivated a culture

and established policies aimed at guiding our employees
to do the right thing. The policies are specifically crafted
to prevent, deter and identify instances of bribery, fraud
and other unethical business practices.

Our code of conduct sets expectations on ethical decisionmaking and covers a variety of topics, such as equal

employment opportunity, anti-discrimination and anti-

harassment, anti-bribery and anti-corruption, and anti-trustand competition laws. It also makes clear when and how

compliance, fostering effective communication and working together to make good decisions. We work to comply with

program in accordance with the laws applicable to our industry, the Program Guidance for Pharmaceutical Manufacturers

published by the Office of the Inspector General of the U.S. Department of Health and Human Services, and the PhRMA Codeof Interactions with Healthcare Professionals.

Ultragenyx’s compliance program includes:• A compliance officer and compliance committee
responsible for developing, operating and monitoring thecompliance program and with authority to report directly
to the board and our CEO• Written standards of conduct, policies and practices that
document the company’s commitment to complianceand requirements to strictly follow fraud and abuse laws

individuals should raise concerns and documents our no-• Easy to understand, effective and readily available
education and training programs for all employeeswith appropriate disciplinary actions, when necessary,• Open lines of communication and partnership between
the compliance officer and workforce to help us ethicallyachieve our company mission and goals

retaliation policy. We enforce our policies and requirements
and we take a zero-tolerance approach to any violationof law or policy.

• Continued enhancement of an audit and monitoring
program to identify and address risks• Enforcement of compliance obligations throughguidelines that include disciplinary action fornoncompliance• Mechanisms to investigate and respond promptly
and properly to reports of noncompliance, including
processes to initiate corrective measures

In 2023
Ultragenyx did not have any material
monetary losses as a resultof legal proceedings associated withcorruption and bribery(please refer to our 2023 Annual Report).

© 2024 Ultragenyx Pharmaceutical Inc

90Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Ethics and Integrity (cont.)
We consistently perform a range of monitoring and auditing

day responsibilities. All full-time, U.S.-based employees must

Employees have an obligation to report any conduct thatinternal audit, finance and legal teams. We use technology

policy and complete the company-offered training within

and/or the code of conduct. Various avenues are available

activities across business operations, in collaboration with ourand automated tools to monitor and report on compliancematters. An annual survey is also distributed to assess ourcompliance culture. In 2023, approximately 90% of survey

respondents indicated that the annual compliance training
sufficiently prepares them to confidently execute their
responsibilities.

acknowledge our anti-harassment and anti-discrimination

the first several months of employment. All U.S. employees,
including part-time and temporary employees, musttake a harassment prevention training course every year.

Additionally, on an annual basis, employees are expected

to receive and acknowledge understanding of the code of

to seek advice on ethical behavior and report concerns

related to violations of such behavior, and we have a strict
no-retaliation policy for individuals who raise concerns
in good faith.

conduct. Every year, an annual compliance culture survey is

The company reinforces adherence to the code of conduct’ssent to all employees to gauge our compliance culture. In

bribery and anti-corruption, conflicts of interest, insider

the code of conduct and other ethical standards within theamong other areas. These trainings, along with our policies

written or digital acknowledgment of the code of conduct

expectations by providing employees with training on anti-

2023, 99% of full-time employees have received training on

training, anti-harassment, and data protection and privacy,

last three years and 97% of full-time employees provided

and procedures, outline the expected conduct for day-to-in 2023.

© 2024 Ultragenyx Pharmaceutical Inc

they, in good faith, believe violates laws, corporate policies,

91Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Ethics and Integrity (cont.)
Raising A Concern

Ethical Treatment of Animals

Ultragenyx’s compliance hotline, which also serves as theare required to be promptly handled, and any identified

Ultragenyx is committed to the ethical treatment of animals

We expect external service providers to meet or exceed all

allows employees or anyone else to report any potential or

a complaint of discrimination or harassment, regardlesstherapies for patients with rare disease.

and national laws and regulations. Our external service

Confidential and Anonymous Financial Concern Hotline,

actual violation of our code of conduct, company policies
and procedures, and applicable laws and regulations.

Any individual can provide comments using the hotline.

Messages can be submitted anonymously using a secure webform, email or telephone. Additionally, a strict non-retaliation
policy ensures individuals can report concerns without fearof reprisal or discrimination, promoting an environmentof openness and trust.

issues are required to be addressed. If an employee makesof where the complaint is made, Ultragenyx’s policy is

to conduct a timely and thorough investigation and takeappropriate action. Investigations are required to be assessedand conducted based on Ultragenyx’s internal investigations

protocol and are required to be conducted by the appropriate
personnel depending on the issue. If and when issues arise,
we are required to identify root causes and, in a timely

and efficient manner, implement measures to stop repeat

Complaints or other messages left on the complianceoccurrences. In 2023, all reported complaints related toof compliance and the chairperson of our Audit Committee,

discrimination or harassment were investigated and promptly

hotline are anonymously sent to our chief legal officer, headpotential breaches to our code of conduct and incidents of

who then take the necessary next steps. All hotline reports

addressed as appropriate.

© 2024 Ultragenyx Pharmaceutical Inc

used in the development of potential new and life-changing
Our company is committed to the “3Rs:”

Replace: Use non-animal methods for experiments wheneverpossible, such as simulations and computational tools
or in vitro systems

Reduce: Use the minimum number of animals in each study
in order to achieve valid results and objectives

Refine: Use procedures that decrease the potential for pain
and limit distress for animals

animal care and use standards that are applicable to local
providers are accredited by the American Association for

Accreditation of Laboratory Animal Care (AAALAC), a private,nonprofit organization that promotes the humane treatmentof animals in science through voluntary accreditation andassessment programs. Additionally, animal facilities aregoverned by an Institutional Animal Care and Use Committee(IACUC), which oversees all aspects of animal care, welfare

and scientific programs for research. The IACUC reviews all
animal use protocols, oversees compliance with federal

regulations, inspects animal facilities, and manages animal
handling/training and educational programs.

92

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Interactions with Patients, Caregivers and Healthcare Professionals
We respect the doctor-patient

relationship and the privacy rightsof patients. We strive to interact

with patients and caregivers in an

appropriate manner and in compliancewith applicable laws, regulations
and our internal Healthcare
Compliance Manual.

Ultragenyx has adopted policies and practices consistent with the PhRMA Code and other applicable industry standards that

Our Healthcare Compliance Manual is available inwith healthcare professionals who provide services to our company as researchers, consultants and speakers. Additionally,

Our compliance program is overseen by a management-

govern interactions with healthcare professionals. These policies encompass support for medical education and collaboration
the policies include provisions for business courtesies, grants and charitable contributions that state that such funds are

not conditioned, either expressly or implicitly, on any agreements to prescribe, purchase, recommend, influence or provide
favorable formulary status for any Ultragenyx medicine. The policies also cover provisions related to the promotion of

Ultragenyx medicines in compliance with the FDA’s regulatory framework, as well as with regulatory requirements in otherjurisdictions regarding the promotion of pharmaceutical products.

Our Healthcare Compliance Manual outlines key principles related to our ethical marketing and sales practices:• Interactions with customers focus on education about
the benefits and risks of our products to promote theirappropriate use. These interactions must occur in venues
conducive to education, and Ultragenyx prohibits theprovision of entertainment to its customers.
• Promotional communications must be truthful, notmisleading, and fairly balanced with appropriate safety
information. They must also be consistent with the
medicine’s label.

© 2024 Ultragenyx Pharmaceutical Inc• Employees are prohibited from using items of value
or in-kind services to reward or induce a healthcare
professional to utilize, prescribe, purchase or recommend
our medicines.
• The hiring of healthcare professionals as speakers or
consultants must be based on a legitimate business
need, free from inappropriate influences. Any fees paid tohealthcare professionals must not exceed the fair market
value of the service provided.

English, Spanish, Portuguese, French and German.

level compliance committee that provides guidance withrespect to healthcare law compliance. The committee
meets at least quarterly.

Ultragenyx is committed to meeting all U.S. state andfederal reporting requirements including the OpenPayments Report, commonly known as the Sunshine Act,as well as other applicable global transparency reporting

requirements. Our disclosure of payments to the Sunshine

Act can be found on the Centers for Medicare & Medicaid
Services (CMS) open payments website.

93Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Data Privacy
The rightful collection and utilizationof personal information from diverse
sources – including patients, clinical

trial participants, customers, healthcare
providers and our employees – areintegral to our operations.

We are dedicated to protecting the privacy and integrity of

We follow all applicable data protection laws, regulations and best practices, including the General Data Protection Regulation

privacy laws. This commitment is upheld through our data

• Our privacy policies set forth the practices of
Ultragenyx regarding the collection, use, retention
and disclosure of personal information in connection
with all corporate activities.

• We have a standardized process for responding to data
subject requests regarding their data that Ultragenyx
processes (see Data Protection, Anonymization and
Security in this report for more information).

• Our DPO collaborates across the business with the goalof ensuring that any data shared internally is with theright functions and for the right reasons.

• We expect our workforce to be accountable, to protect
personal data – which we may have access to during
the ordinary course of our business operations – andto process such data responsibly in accordance withcompany policies and any applicable laws. All employees
receive periodic training on privacy as part of our annual
code of conduct training.

this information and related holders by adhering to global

privacy program, which encompasses global privacy policies,
comprehensive training, and system operating proceduresand programmatic controls. Our proactive approach toprivacy demonstrates our unwavering commitment tohigh standards of data security and compliance, thereby

safeguarding the trust of our stakeholders and maintaining
the integrity of our business practices.

Our chief information officer (CIO), in partnership with

our data privacy officer (DPO) and the rest of our Legaland Compliance departments, oversee and manage ourapproach to privacy-related matters, as they relate to

Ultragenyx, third party data and technological and cybersecurity platforms.

© 2024 Ultragenyx Pharmaceutical Inc(GDPR), California Consumer Privacy Act (CCPA) and Lei Geral de Proteção de Dados (LGPD), among others.

• Our information technology team, in collaboration
with the DPO, and their respective teams are responsible
for evaluating the company’s software programs andapplications for data privacy compliance. This review
includes, among other considerations, a determinationof whether personal information will be transmittedwith the goal of ensuring that all new and existing
critical applications are managed in accordance withapplicable laws.

In 2023
Ultragenyx did not have any material
data privacy breaches.

94Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Cybersecurity
Ultragenyx has an information
security program with policies

and procedures designed to guideour security and data protection
decision-making process.

In 2023
98% of employees
received IT-related training

GovernanceTraining and AwarenessBusiness Resilience
and Compliance

Identifying and Mitigating
Cybersecurity Risks• Board-level oversight is assigned to our Audit
Committee. The CIO regularly reports to the
Audit Committee to provide an overview of therisks, processes, procedures, recommendations
and an overall assessment of our cybersecurity
program.

• We provide information security training toemployees at a minimum of twice per year.
The same training is provided to members of
our contingent workforce who have access to
our internal systems and can be a risk to our
information technology infrastructure. We
implement additional training, as needed.

• We have documented the appropriate disasterrecovery plans for all Ultragenyx critical systems.

• We have ongoing managed vulnerability
scanning and patching through our vulnerability
management program.

• Strategic and tactical oversight and direction forcybersecurity-related matters (cyber program
strategy, program deficiencies and maturation,
program and process review and testing, training
and awareness, and reporting) are led by ourhead of information security, who reports
directly to the CIO.
• Our cybersecurity approach is informed by
the NIST 800-53 standard and complianceframework.
• Our team includes members who are certified
with qualifications including CISSP, CRISC and
CISA, and are proficient in the managementof cybersecurity protocols and practices.
• We have established a partnership between
the DPO and CIO to implement proper controls
for data protection, use, storage and retention
that are designed to confirm our technologicalsolutions meet legal and regulatory
requirements.

© 2024 Ultragenyx Pharmaceutical Inc• We conduct phishing exercises at least
three times per year, focusing on users withrepeated simulation failures and implementing
corrective actions. We perform additional
testing, as needed.
• Our Acceptable Use Policy (AUP) was updated toinclude generative AI usage and included in our
training curriculum.
• We issue regular employee awareness
communications, including email newsletters.
These communications are intended toconsistently inform users about the escalation
process to follow if they notice anythingsuspicious and remind them to report any
security incidents to our security email address.

• We also have documented our incident response
plan’s process and have performed tabletop• We monitor internal and external cybersecurity
exercises annually.
threats and review and revise our cybersecurity
defenses on a regular cadence.
• We updated our critical vendors disasterrecovery plans for our information• Targeted audits and penetration tests aretechnology systems.
conducted throughout the year by internaland external entities. In 2023, we conducted• We purchase a fixed amount of cybersecurity
our annual third-party internal and externaland crime insurance coverage to help mitigate
penetration testing. The third-party also
some of the risk and potential liability fromperformed vulnerability testing and simulated
cybersecurity breaches, including claims related
various types of attacks to exploit any weakness
to data privacy regulatory matters, third-party
discovered. We document findings andlawsuits and the costs associated with data
execute remediation plans. Our security teambreach events.
also performs various vulnerability scanning• We require that new and existing systems
throughout the year.
are built to comply, and remain in compliance• In 2023, we continued to onboard andwith regulatory requirements, including SOX
inventoried all new systems in a central asset
and GxP programs.
management tool and monitor and update
operating procedures. We operationalized cloudsecurity, endpoint data loss prevention, andprivileged access management solutions.
• We onboarded a governance, risk andcompliance solution as well as a risk monitoring
service to monitor Ultragenyx security
performance, as well as that of our vendors.

95

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Responsible Supply Chain Management
We understand the critical role ofresponsible supply chain managementin achieving our mission.

In 2022, we introduced the Global Standard for Suppliers

Central to our success in developing therapies to treat rareassessments and audits designed to verify compliance with

Conduct. This standard sets forth expectations for our

long-term partnerships with suppliers that share our values.

Finally, our key contracts stipulate that suppliers be open

to complement and supplement the Global Code of

suppliers in areas including integrity, legal compliance, labor
standards, human rights and environmental stewardship. In
2023, we published a formal Global Human Rights policy,

which covers extensive areas, such as labor practices, clinical
trials, product governance and supply chain ethics, and aims
to address potential human rights issues.

Our supplier engagement process encompasses legal andcompliance risk screening, including screening for economic
sanctions and anti-bribery and anti-corruption activities,

and is designed to align with our requirements and company
policy. All of our strategic manufacturers are currently

located in North America, Western Europe and Japan. Part

Our approach to responsible procurement includes ourof our audit process involves the integration of corporate

Ultragenyx maintains a zero-tolerance stance on modern

party’s commitments in our evaluation of potential partners.

all relevant laws. We transparently communicate our efforts

role in our supply chain management. It allows us to more

expectation that suppliers uphold high ethical standards.

responsibility criteria, which allows us to consider the third-

slavery in any form. We expect all suppliers to comply with

Our supplier quality management system plays a pivotal

against modern slavery in our statement on Transparency

effectively monitor supplier quality and supports integrationin our Supply Chain and Modern Slavery.

© 2024 Ultragenyx Pharmaceutical Inc

and ultrarare diseases is the establishment of strategic,

applicable laws and regulations.

to audits assessing compliance. Post-audit, we endeavor to

engage collaboratively with suppliers to develop and follow

through on action plans. Additionally, we require compliancewith the U.S. Department of Treasury’s OFAC regulations

before financially transacting with any contracted third party.
These measures reflect Ultragenyx’s commitment to fosteringa supply chain that is aligned with our core values and theunique needs of the rare disease community.

with future partners. High-risk suppliers undergo formal

96

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

SASB Index
The following index lists the activity and accounting metrics from the Sustainability Accounting Standards Board (SASB) Biotechnology and Pharmaceuticals Industry
Standard (2018) with associated response, reference or report location.
Accounting Metric

Code

Response / Reference / Report Location

Number of patients treated

HC-BP-000.A

See our 2023 Corporate Responsibility Report, Access and Affordability.

Number of drugs 1) in portfolio and 2) in research and development (Phases 1-3)

HC-BP-000.B

See our current Pipeline.

Topic

Accounting Metric

Code

Response / Reference / Report Location

Safety ofClinical Trial
Participants

Discussion, by world region, of management process for ensuring quality
and patient safety during clinical trials

HC-BP-210a.1

See our 2023 Corporate Responsibility Report, Clinical Trials, Quality and Safety.

Number of FDA Sponsor Inspections related to clinical trial management
and pharmacovigilance that resulted in: (1) Voluntary Action Indicated(VAI) and (2) Official Action Indicated (OAI)

HC-BP-210a.2

There were no FDA Sponsor Inspections related to clinical trial management and pharmacovigilance that resulted in either Voluntary Action Indicated (VAI) or
Official Action Indicated (OAI) in 2023. See the FDA Compliance Dashboard for more information.

Total amount of monetary losses as a result of legal proceedings
associated with clinical trials in developing countries

HC-BP-210a.3

There have been no reported monetary losses as a result of legal proceedings associated with clinical trials in developing countries for the year 2023. See our2023 Annual Report, page 67 (Item 3. Legal Proceedings).

Description of actions and initiatives to promote access to health careproducts for priority diseases and in priority countries as defined by theAccess to Medicine Index

HC-BP-240a.1

Our products and clinical research primarily target rare and ultrarare diseases, which fall outside the 2023 Access to Medicine Index's main focus areas. Ourinvestments in R&D, strategic partnerships, and innovative technologies are all aimed at enhancing treatment accessibility and affordability. Additionally, throughpatient assistance programs and expanded use initiatives, we support patients globally, especially in economically disadvantaged countries. We also provide
health-related grants that support organizations focused on medical research and treatment, and rare disease awareness, education and advocacy globally. See
our 2023 Corporate Responsibility Report, Access and Affordability, Rare Disease Community Support and Grants.

List of products on the WHO List of Prequalified Medicinal Products aspart of its Prequalification of Medicines Programme (PQP)

HC-BP-240a.2

Ultragenyx products are not on the WHO list due to our focus on rare and ultra-rare diseases, while the WHO prioritizes conditions such as HIV/AIDS, tuberculosis,
malaria, and reproductive health. See the WHO Prequalification of Medical Products for more information.

Access to
Medicines© 2024 Ultragenyx Pharmaceutical Inc

97

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

SASB Index (cont.)
Topic

Accounting Metric

Code

Response / Reference / Report Location

Affordability& PricingNumber of settlements of Abbreviated New Drug Application (ANDA)
litigation that involved payments and/or provisions to delay bringing an
authorized generic product to market for a defined time period

HC-BP-240b.1

See our 2023 Annual Report, pages 19-23 (Item 1. Business - Patents and Proprietary Rights).

Percentage change in: (1) average list price and (2) average net priceacross U.S. product portfolio compared to previous year

HC-BP-240b.2

See our 2023 Corporate Responsibility Report, Access and Affordability.

Percentage change in: (1) list price and (2) net price of product with largest
increase compared to previous year

HC-BP-240b.3

See our 2023 Corporate Responsibility Report, Access and Affordability.

List of products listed in the Food and Drug Administration’s (FDA)
MedWatch Safety Alerts for Human Medical Products database

HC-BP-250a.1

Our products are not listed in the FDA MedWatch. See the FDA FAERS MedWatch website for more information.

Number of fatalities associated with products as reported in the FDA
Adverse Event Reporting System

HC-BP-250a.2

There no fatalities associated with Ultragenyx products in 2023. See the FDA FAERS MedWatch website for more information.

Number of recalls issued, total units recalled

HC-BP-250a.3

Ultragenyx did not issue any recalls. See the FDA Data Dashboard for more information.

Total amount of product accepted for takeback, reuse, or disposal

HC-BP-250a.4

Ultragenyx manufactures medicines on a schedule that is designed to avoid their expiration before patient use. In the event that medicines expire before use andare returned, are found to be unsuitable for release or are subject to a recall or withdrawal notice, Ultragenyx does not reintroduce them again for reuse. They getdisposed of using regulated and monitored incineration processes.

Number of FDA enforcement actions taken in response to violations ofcurrent Good Manufacturing Practices (cGMP), by type

HC-BP-250a.5

Ultragenyx was not involved in any FDA enforcement actions in response to violations of cGMP. See the FDA Data Dashboard for more information.

Drug Safety© 2024 Ultragenyx Pharmaceutical Inc

98Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

SASB Index (cont.)
Topic

Accounting Metric

Code

Response / Reference / Report Location

Counterfeit
Drugs

Description of methods and technologies used to maintain traceability of
products throughout the supply chain and prevent counterfeiting

HC-BP-260a.1

Ultragenyx has implemented a comprehensive process designed to address counterfeit product risks, including a Field Action procedure and security features liketamper-evident seals and serialization of product labeling. See our 2023 Corporate Responsibility Report, Quality in Supply Chain, section on Counterfeit Drugs.

Discussion of process for alerting customers and business partners ofpotential or known risks associated with counterfeit products

HC-BP-260a.2

Ultragenyx has implemented a comprehensive process designed to address counterfeit product risks, including a Field Action procedure and security features liketamper-evident seals and serialization of product labeling. See our 2023 Corporate Responsibility Report, Quality in Supply Chain, section on Counterfeit Drugs.

Number of actions that led to raids, seizure, arrests, and/or filing of
criminal charges related to counterfeit products

HC-BP-260a.3

Ultragenyx had no instances of actions related to counterfeit products in 2023. See our 2023 Corporate Responsibility Report, Quality in Supply Chain, section
on Counterfeit Drugs.

Total amount of monetary losses as a result of legal proceedings
associated with false marketing claims

HC-BP-270a.1

There have been no reported monetary losses as a result of legal proceedings associated with false marketing claims for the year 2023. See our 2023 Annual
Report, page 67 (Item 3. Legal Proceedings).

Description of code of ethics governing promotion of off-label use of
products

HC-BP-270a.2

See our Global Code of Conduct.

Employee
Recruitment,
Development &Retention

Discussion of talent recruitment and retention efforts for scientists andresearch and development personnel

HC-BP-330a.1

See our 2023 Corporate Responsibility Report, People chapter.

(1) Voluntary and (2) involuntary turnover rate for: (a) executives/senior
managers, (b) midlevel managers, (c) professionals, and (d) all others

HC-BP-330a.2

See our 2023 Corporate Responsibility Report, Human Capital Development.

Supply Chain
Management

Percentage of (1) entity's facilities and (2) Tier I suppliers' facilities
participating in the Rx-360 International Pharmaceutical Supply Chain
Consortium audit program or equivalent third-party audit programs for
integrity of supply chain and ingredients

HC-BP-430a.1

See our 2023 Corporate Responsibility Report, Quality in Supply Chain.

Business Ethics

Total amount of monetary losses as a result of legal proceedings
associated with corruption and bribery

HC-BP-510a.1

There have been no reported monetary losses as a result of legal proceedings associated with corruption and bribery for the year 2023. See our 2023 Annual
Report, page 67 (Item 3. Legal Proceedings).

Description of code of ethics governing interactions with health care
professionals

HC-BP-510a.2

See our 2023 Corporate Responsibility Report, Interactions with Patients, Caregivers and Healthcare Professionals.

EthicalMarketing*The SASB metrics are referenced above for informational purposes only with no claim of fulfillment to any given metric.

© 2024 Ultragenyx Pharmaceutical Inc

99

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility Report

Global Reporting Initiative (GRI) Index
This index is aligned with the Global Reporting Initiative’s Sustainability Reporting Standards. It provides easy access to Core reporting elements and, where available,
additional Comprehensive reporting level elements for the period January 1 through December 31, 2023, unless otherwise noted. GRI 1 used: GRI: Foundation 2021.
GRI Standard

Disclosure2023 Location

GRI 2: General Disclosures 2021

2-1 Organizational details

2023 Corporate Responsibility Report, About Us and 2023 Annual Report, Item 1. Business.

2-2 Entities included in the organization’ssustainability reporting

The 2023 Corporate Responsibility Report mainly covers information from the fiscal year ending December 31, 2023, unless otherwise indicated. Questions and inquirieson the reported information can be submitted to cr@ultragenyx.com. | 2023 Corporate Responsibility Report, About This Report.

2-3 Reporting period, frequency and contact
point

Ultragenyx publishes a corporate responsibility report annually.
2023 Corporate Responsibility Report, About This Report.

2-4 Restatements of information

No restatements of information were made.

2-5 External assurance

No external assurance was performed.

2-6 Activities, value chain and other business
relationships

2023 Annual Report, Item 1. Business.

2-7 Employees

2023 Corporate Responsibility Report, Workforce Data.

2-8 Workers who are not employees

Information unavailable.

2-9 Governance structure and composition

2023 Corporate Responsibility Report, Corporate Governance. | 2023 Corporate Responsibility Report, Corporate Responsibility Oversight.

2-10 Nomination and selection of the highest
governance body2024 Proxy, Nomination of Directors.

© 2024 Ultragenyx Pharmaceutical Inc

100Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility ReportGRI Index (cont.)
GRI Standard

Disclosure2023 Location

GRI 2: General Disclosures 2021 (cont.)

2-11 Chair of the highest governance body

Daniel G. Welch, Chairperson of the Board. | Ultragenyx’s Leadership.

2-12 Role of the highest governance body inoverseeing the management of impacts

2023 Corporate Responsibility Report, Corporate Responsibility Oversight. | Board Committees and Charters.

2-13 Delegation of responsibility for managing
impacts2023 Corporate Responsibility Report, Corporate Responsibility Oversight.

2-14 Role of the highest governance body insustainability reporting

2023 Corporate Responsibility Report, Corporate Responsibility Oversight.

2-15 Conflicts of interest

2023 Corporate Responsibility Report, Corporate Governance. | Corporate Governance Guidelines.

2-16 Communication of critical concerns

2023 Corporate Responsibility Report, Risk Management. | 2023 Annual Report, Item 1A. Risk Factors.

2-17 Collective knowledge of the highest
governance body2024 Proxy, Board Diversity, Skills and Experience.

2-18 Evaluation of the performance of thehighest governance body

Corporate Governance Guidelines.

2-19 Remuneration policies

2024 Proxy, Executive Compensation. | 2024 Proxy, Director Compensation.

2-20 Process to determine remuneration

2024 Proxy, Executive Compensation. | 2024 Proxy, Director Compensation.

2-21 Annual total compensation ratio

2024 Proxy, CEO Pay Ratio.

© 2024 Ultragenyx Pharmaceutical Inc

101

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility ReportGRI Index (cont.)
GRI Standard

Disclosure2023 Location

GRI 2: General Disclosures 2021 (cont.)

2-22 Statement on sustainable development
strategy2023 Corporate Responsibility Report, Letter From Our CEO. | 2023 Corporate Responsibility Report, Corporate Responsibility Approach.

2-23 Policy commitments

2023 Corporate Responsibility Report, Governance chapter | Global Code of Conduct. | Global Standard for Suppliers. | Human Rights Policy.

2-24 Embedding policy commitments

2023 Corporate Responsibility Report, Governance chapter | Global Code of Conduct. | Global Standard for Suppliers. | Human Rights Policy.

2-25 Processes to remediate negative impacts

2023 Corporate Responsibility Report, Governance chapter | Global Code of Conduct. | Global Standard for Suppliers. | Human Rights Policy.

2-26 Mechanisms for seeking advice and raisingconcerns2023 Corporate Responsibility Report, Raising A Concern | Global Code of Conduct. | Global Standard for Suppliers. | Human Rights Policy.

2-27 Compliance with laws and regulations

2023 Corporate Responsibility Report, Ethics and Integrity | 2023 Annual Report, Item 3 Legal Proceedings.

2-28 Membership associations

2023 Corporate Responsibility Report, Strategic Collaborations | 2023 Corporate Responsibility Report, Public Policy Participation. | 2023 Corporate Responsibility Report,
Patient Advocacy and Engagement.

2-29 Approach to stakeholder engagement

2023 Corporate Responsibility Report, Corporate Responsibility Approach.

2-30 Collective bargaining agreements

2023 Annual Report, Item 1. Business – Human Capital.

3-1 Process to determine material topics

2023 Corporate Responsibility Report, Materiality Assessment.

3-2 List of material topics

2023 Corporate Responsibility Report, Materiality Assessment.

3-3 Management of material topics

The 2023 Corporate Responsibility Report describes the management of material topics by section. | 2023 Annual Report, Item 1A. Risk Factors.

GRI 3: Material Topics 2021

© 2024 Ultragenyx Pharmaceutical Inc

102

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility ReportGRI Index (cont.)
GRI Standard

Disclosure2023 Location

GRI 201: Economic Performance 2016

201-1 Direct economic value generated anddistributed

2023 Annual Report, Item 7, Management’s Discussion and Analysis of Financial Condition and Results of Operations.

201-2 Financial implications and other risks andopportunities due to climate change

2023 Corporate Responsibility Report, Reducing Our Environmental Impacts.

201-3 Defined benefit plan obligations and otherretirement plans

2023 Annual Report, Item 15. Exhibits and Financial Statement Schedules.

203-1 Infrastructure investments and services
supported

2023 Corporate Responsibility Report, Access and Affordability. | 2023 Corporate Responsibility Report, Communities chapter.

203-2 Significant indirect economic impacts

2023 Corporate Responsibility Report, Access and Affordability. | 2023 Corporate Responsibility Report, Communities chapter.

205-1 Operations assessed for risks related tocorruption

2023 Annual Report, Item 1A. Risk Factors. | 2023 Corporate Responsibility Report, Responsible Supply Chain Management. | Global Code of Conduct. | Global Standard
for Suppliers.

205-2 Communication and training about anticorruption policies and procedures

2023 Corporate Responsibility Report, Ethics and Integrity. | Global Code of Conduct. | Global Standard for Suppliers.

205-3 Confirmed incidents of corruption andactions taken

2023 Corporate Responsibility Report, Ethics and Integrity.

GRI 206: Anti-competitive Behavior 2016

206-1 Legal actions for anti-competitive
behavior, anti-trust, and monopoly practices

2023 Annual Report, Item 3 Legal Proceedings.

GRI 303: Water and Effluents 2018

303-1 Interactions with water as a sharedresource

2023 Corporate Responsibility Report, Managing Waste and Water.

GRI 305: Emissions 2016305-5 Reduction of GHG emissions

2023 Corporate Responsibility Report, Reducing Environmental Impacts.

GRI 306: Waste 2020306-2 Management of significant waste-related
impacts2023 Corporate Responsibility Report, Managing Waste and Water.

GRI 203: Indirect Economic Impacts 2016

GRI 205: Anti-corruption 2016

© 2024 Ultragenyx Pharmaceutical Inc

103

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility ReportGRI Index (cont.)
GRI Standard

Disclosure2023 Location

GRI 401: Employment 2016

401-1 New employee hires and employee
turnover2023 Corporate Responsibility Report, Human Capital Development. | 2023 Corporate Responsibility Report, DEIB in Recruitment.

401-2 Benefits provided to full-time employeesthat are not provided to temporary or part-time
employees2023 Corporate Responsibility Report, Employee Compensation and Benefits. | Benefits.

401-3 Parental leave

2023 Corporate Responsibility Report, Employee Compensation and Benefits. | Benefits.

403-1 Occupational health and safety
management system

2023 Corporate Responsibility Report, Occupational Health, Safety and Wellness.

403-2 Hazard identification, risk assessment, andincident investigation

2023 Corporate Responsibility Report, Occupational Health, Safety and Wellness.

403-4 Worker participation, consultation, andcommunication on occupational health andsafety2023 Corporate Responsibility Report, Occupational Health, Safety and Wellness.

403-5 Worker training on occupational healthand safety2023 Corporate Responsibility Report, Occupational Health, Safety and Wellness.

403-6 Promotion of worker health2023 Corporate Responsibility Report, Occupational Health, Safety and Wellness.

403-8 Workers covered by an occupational healthand safety management system

2023 Corporate Responsibility Report, Occupational Health, Safety and Wellness.

403-9 Work-related injuries

2023 Corporate Responsibility Report, Occupational Health, Safety and Wellness.

403-10 Work-related ill health2023 Corporate Responsibility Report, Occupational Health, Safety and Wellness.

GRI 403: Occupational Healthand Safety 2018

© 2024 Ultragenyx Pharmaceutical Inc

104

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility ReportGRI Index (cont.)
GRI Standard

Disclosure2023 Location

GRI 404: Training and Education 2016404-1 Average hours of training per year per
employee2023 Corporate Responsibility Report, Employee Learning and Development.

404-2 Programs for upgrading employee skills
and transition assistance programs2023 Corporate Responsibility Report, Employee Learning and Development. | 2023 Corporate Responsibility Report, Career Development.

404-3 Percentage of employees receiving regularperformance and career development reviews2023 Corporate Responsibility Report, UltraPerformance Management.

GRI 405: Diversity and Equal Opportunity
2016

405-1 Diversity of governance bodies
and employees

2023 Corporate Responsibility Report, Workforce data (overall diversity data). | 2023 Corporate Responsibility Report, Board Diversity (board diversity).

GRI 406: Non-discrimination 2016406-1 Incidents of discrimination and corrective
actions taken

2023 Corporate Responsibility Report, Raising A Concern.

GRI 408: Child Labor 2016408-1 Child Labor 2016 408-1 Operations
and suppliers at significant risk of incidents
of child labor2023 Corporate Responsibility Report, Responsible Supply Chain Management | Global Code of Conduct. | Global Standard for Suppliers.

GRI 409: Forced or Compulsory Labor 2016409-1 Operations and suppliers at significant risk
for incidents of forced or compulsory labor2023 Corporate Responsibility Report, Responsible Supply Chain Management | Global Code of Conduct. | Global Standard for Suppliers.

GRI 413: Local Communities 2016413-1 Operations with local community
engagement, impact assessments, anddevelopment programs2023 Corporate Responsibility Report, Communities chapter. | Includes information on local community engagement, including corporate philanthropy and volunteering.

GRI 415: Public Policy 2016415-1 Political Contributions

2023 Corporate Responsibility Report, Public Policy Participation.

© 2024 Ultragenyx Pharmaceutical Inc

105

Letter From Our CEO

About Us

Corporate Responsibility Approach

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index

2023 Corporate Responsibility ReportGRI Index (cont.)
GRI Standard

Disclosure2023 Location

GRI 416: Customer Health and Safety 2016416-1 Assessment of the health and safety
impacts of product and service categories

2023 Corporate Responsibility Report, Safety.

416-2 Incidents of non-compliance concerning
the health and safety impacts of products andservices2023 Corporate Responsibility Report, Quality. | 2023 Corporate Responsibility Report, Safety. | FDA Data Dashboard.

417-1 Requirements for product and service
information and labeling2023 Annual Report, Item 1. Business. | Crysvita. | Mepsevii. | Dojolvi. | Evkeeza.

417-2 Incidents of non-compliance concerning
product and service information and labeling2023 Annual Report, Item 1A. Risk Factors.

417-3 Incidents of non-compliance concerningmarketing communications

2023 Corporate Responsibility Report, Interactions with Patients, Caregivers and Healthcare Professionals. | 2023 Annual Report, Item 1A. Risk Factors.

418-1 Substantiated complaints concerning
breaches of customer privacy and losses ofcustomer data

2023 Corporate Responsibility Report, Data Privacy.

GRI 417: Marketing and Labeling 2016

GRI 418: Customer Privacy 2016

© 2024 Ultragenyx Pharmaceutical Inc

106

Letter From Our CEO

About Us

Corporate Responsibility Approach2023 Corporate Responsibility Report
Ultragenyx.com© 2024 Ultragenyx Pharmaceutical Inc

Innovation

Patients

PeopleCommunities

PlanetGovernance

SASB Index

GRI Index