OCTOBER 15-18, 2024 | WASHINGTON, D.C. & VIRTUAL

16

THE WATERGATE HOTEL

TH
AnnualTechnologies and Strategies for Safe and Efficacious Products in the Clinic

Conference Programs
Immunogenicity Assessment& Clinical Relevance

Register
Early

Immunogenicity
Prediction & Control

for
MAXIMUM
SAVINGS!

Optimizing Bioassays
for Biologics
Training Seminar:
A Guide to Statistical
Methods for Bioassay
Workshop: Biostatistics
for Beginners
Symposium: Immunology
for Biotherapeutics

Hear FDA Updates from

Plus! 5 Short Courses,

10 Speakers

Exhibits, Posters, and More

Keynote &Featured Speakers :Yow-Ming Wang

Murli Krishna

Zuben Sauna

Robert Siegel

Kannan Natarajan

Marisa Joubert

Weifeng Xu

FDABristol Myers Squibb Co.

FDA

Eli Lilly and Company

NIH

Amgen

Merck

Organized byCambridge Healthtech Institute#CHIImmunoBio | ImmunogenicitySummit.com

1#
Join us at the
Immunogenicity& Bioassay Event in the U.S.
Bringing Together Industry, Academia & Regulatory Authorities
RESOLVE the CHALLENGES of immunogenicity testing and risk assessment during preclinical and clinical development,
bioassay design & analysis, and how to manipulate the immune system for therapeutic advantage by attending CHI’s
16th Annual Immunogenicity & Bioassay Summit on October 15-18, 2024, at The Watergate Hotel in Washington, D.C.
We’ll examine challenges facing the industry with NEW case studies, NEW perspectives, and NEW approaches. Hearfrom the FDA, industry, academia, and regulatory authorities at 3 Conference Programs, Symposium, Training Seminar,
Workshop, and several Short Courses. Once again, we’ll host interactive breakouts on various niche topics so delegatescan share experiences and generate new ideas. Plus, attend a vibrant exhibit/poster hall with innovative products &services. We hope to greet you this fall in Washington, D.C.

Conference At-a-Glance*
Tuesday, October 15

S1:
Immunology
for Biotherapeutics

SC1:
Developmentof NAb Assays,
Technical
Considerations, Case
Studies

W1:
Biostatistics
for
Beginners

Wednesday, October 16

C1A:
Immunogenicity
Assessment & ClinicalRelevance

SC2:
Overcoming
Drug andTargetInterferencein ADA and
NAb Assays

TS1A: A Guide
to Statistical
Methods for
BioassayFriday, October 18

Thursday, October 17

C1A:
Immunogenicity
Assessment & Clinical Relevance

TS1A: A Guide
to Statistical
Methods for
Bioassay

C1B:
Immunogenicity
Prediction & Control

C2B:
Optimizing
Bioassays for
Biologics

C1B:
Immunogenicity
Prediction & Control

C2B:
Optimizing
Bioassays for
Biologics*Short Courses, Workshop & Training Seminar are In-Person Only. See
Registration Page for packages and pricing details.

SC3:
Validation of
ADA Assays
and Cut Point
Calculations

SC4: Recent
Advances with
Cell and Gene
Therapy

Corporate Sponsors

2 | ImmunogenicitySummit.com

SC5: Advice on
Putting Together
an Integrated
Summary of
Immunogenicity

Corporate Support Sponsors

Sponsorship & Exhibitor Opportunities
CHI offers comprehensive packages that can be customized to your budget and objectives. Sponsorshipallows you to achieve your goals before, during, and long after the event. Packages may include presentations,exhibit space, and branding, as well as the use of delegate lists. Signing on early will maximize your exposure toqualified decision-makers and drive traffic to your website in the coming months.

ExhibitPodium Presentations — Available within
Main Agenda!

Exhibitors will enjoy facilitated networking opportunities withqualified delegates, making it the perfect platform to launch a
new product, collect feedback, and generate new leads. Exhibit space sells out quickly, so reserve yours today! Additional
branding and promotional opportunities are available, including:Showcase your solutions to a guaranteed, targeted audience through a
15- or 30-minute presentation during a specific program, breakfast, lunch,
or a pre-conference workshop. Package includes exhibit space, onsite
branding, and access to cooperative marketing efforts by CHI. Lunches
are delivered to attendees who are already seated in the main session
room. Presentations will sell out quickly! Sign on early to secure your talk.

•
•
•
•
•

Invitation-Only VIP Dinner/Hospitality Suite

Select specific delegates from the pre-registration list to attend a private
function at an upscale restaurant or a reception at the hotel. From
extending the invitations, to venue suggestions, CHI will deliver yourprospects and help you make the most of this invaluable opportunity.

Conference Tote Bags
Literature Distribution (Tote Bag Insert or Chair Drop)
Badge Lanyards
Conference Materials Advertisement
Padfolios and More…

For more information regarding
exhibit and sponsorship,
please contact:

Focus Group

CHI will gladly provide you the opportunity of running a focus group.
This exclusive gathering can be useful to conduct market research,collect feedback on a new product idea, and collect marketing
intelligence from industry experts on a specific topic.

User Group Meeting/Custom Event

Aimee Croke

Co-locate your user group meeting or custom event. CHI will help
market the event, manage logistical operations, develop the agenda,
and more. CHI can handle the entirety of the meeting or select aspects.

Sr. Business Development Manager
T: +1 (781) 292-0777
acroke@cambridgeinnovationinstitute.com

2023 Attendee Demographics

LOCATIONCOMPANY TYPE

n Biotechnicaln Services
n Healthcare
n CRO

87%
9%
3%
1%

n USA
n Europen Rest of World

DELEGATE TITLE

84%
11%
5%

US BREAKDOWNn East Coast 66%
n West Coast 22%
n Midwest
12%n Scientist/Technologist
n Directorn Executive
n Sales & Marketingn Managern Assistant

37%
22%
18%
13%
9%
1%

ImmunogenicitySummit.com | 3

Distinguished Faculty
FDA Speakers

David Lansky, PhD, President, Precision Bioassay, Inc.

Sojin Bing, PhD, Staff Fellow, Office of Tissues & Advanced Therapies, FDA
CBERDaniel Leventhal, PhD, Head of Immunogenicity, Generate Biomedicines

Eric Brodsky, MD, Associate Director, Labelling Policy Team, CDER, FDA

Dawn Maier, PHD, Cell and Gene Therapy CMC, AD Preclinical TechnicalAdvisor, DGMAIER Consulting

Kristina Howard, Principal Investigator, Division of Applied Regulatory Science(DARS), CDER, FDA

Linlin Luo, PhD, Senior Director, Merck

Laurent P. Malherbe, PhD, Executive Director, Eli Lilly and Company

Daniel LaGasse, PhD, Research Regulator, CBER, FDA

Arkadi Manukyan, PhD, Senior Scientist, Bioassay Development, Sanofi

Ronit Mazor, PhD, Principal Investigator, CBER, FDA
Zuben Sauna, PhD, Director, Division of Hemostasis, CBER, FDA

Yan Mao, PhD, Senior Principal Scientist, Boehringer Ingelheim
Pharmaceuticals

Anurag Sharma, PhD, Gene Therapy Reviewer, CBER, FDA

Jim McNally, PhD, CSO, BioAgilytix

Daniela Verthelyi, MD, PhD, Chief, Laboratory of Immunology, CDER, FDA

Diana Montgomery, PhD, Principal Scientist, Pharmacokinetics, Merck & Co.,Inc.

Leslie Wagner, Consumer Safety Officer, CBER, FDA
Yow-Ming Wang, PhD, Associate Director for Biosimilars and Therapeutic
Biologics, CDER, FDA

Paul Moore, PhD, CSO, Zymeworks
Kannan Natarajan, PhD, Staff Scientist, NIAID, NIH
Ravish B. Patel, PhD, Senior Manager, Pharmacology, Sun Pharma Advanced
Research Center

Additional Distinguished Faculty
Kavitha Akula, PhD, Principal Scientist, Bristol Myers Squibb Co.
Bianca Bautista, PhD, Senior Scientist, Clinical Immunology, Amgen, Inc.
Daron Forman, PhD, Senior Principal Scientist, Discovery Biotherapeutics,
Bristol Myers Squibb Co.
Andrew Graves, Director, Immunogenicity Assessment, Specialty Analytics,
Teva Pharmaceuticals
Michele Gunsior, PhD, Senior Director, Astria Therapeutics
Kevin Gurney, PhD, Distinguished Scientist, Analytical Research andDevelopment, Merck

Ryan Peckner, PhD, Director, Machine Learning, Seismic Therapeutic
Jack A. Ragheb, MD, PhD, Senior Vice President, Translational Sciences andMedicine, NexImmune
Bonnie Rup, PhD, Biotechnology Consultant, Bonnie Rup Consulting
Nancy Sajjadi, Independent Quality Consultant, Sajjadi Consulting
Sumona Sarkar, PhD, Biomedical Engineer, Biosystems and Biomaterials
Division, Biomaterials Group, National Institute of Standards and Technology
Han-Yu Shih, PhD, Investigator, NeuroImmune Regulome, National Institutes of
Health National Eye Institute
Robert Siegel, PhD, Associate Vice President, Laboratory for Experimental
Medicine, Eli Lilly and Company

Robert Hamilton, PhD, D.ABMLI, Professor, Medicine & Pathology, Clinical
Immunology & Allergy, Johns Hopkins University

Matthew Stephenson, PhD, Director of Statistics, Quantics Biostatistics

Timothy Hickling, PhD, Investigative and Immunosafety Chapter, Roche

Michael D. Swanson, PhD, Associate Scientific Director, Bioanalytical Discovery& Development Sciences, Johnson and Johnson Innovative Medicine

Andreas Hollenstein, PhD, Principal Scientist, Immunosafety, Roche
Shannon Howell, PhD, Principal Scientist, Immunogenicity Bioanalytical,
Amgen, Inc.
Jeffrey A. Hubbell, PhD, Eugene Bell Professor Tissue Engineering & Vice
Dean, Molecular Engineering, University of Chicago
Susan Irvin, PhD, Senior Principal Scientist, Bioanalytical Strategy, Regeneron
Nicole Jarvi, PhD, Senior Scientist, Merck
Vibha Jawa, PhD, Executive Director, Nonclinical Disposition and Bioanalysis,
Bristol Myers Squibb Co.
Lynn Kamen, PhD, Scientific Officer, Executive Director, BioAgilytix
Emilee Knowlton, Senior Immunology Sales Specialist, ProImmune Inc
Alexander Kozhich, PhD, Director, Bristol Myers Squibb Co.
Murli Krishna, PhD, Senior Principal Scientist, Bristol Myers Squibb Co.

4 | ImmunogenicitySummit.com

Jiangbo Tang, PhD, Senior Scientist, Bristol Myers Squibb Co.
Jenny Valentine, PhD, Senior Principal Scientist, Bioanalytical Sciences,
Regeneron
Faye Vazvaei, Executive Director, Merck
Weifeng Xu, PhD, Director, Bioanalytical, Merck
James Zanghi, PhD, Senior Principal Scientist, BioAnalytical Sciences,
Genentech, Inc.
An Zhao, PhD, Senior Principal Scientist, Bioanalytical Sciences, Regeneron
Pharmaceuticals, Inc.

October 15 & 17, 2024

Short Courses**Separate registration is required, see Registration Page for pricing details.
Short courses are available in-person only.
TUESDAY, OCTOBER 15 9:00 AM – 12:00 PM

SC1: Development of NAb Assays, Technical
Considerations, Case Studies

Instructors:
Lynn Kamen, PhD, Scientific Officer, Executive Director, BioAgilytix
Jim McNally, PhD, CSO, BioAgilytix
The development of neutralizing antibody assays is a daunting task that iscomplicated by the specific nature of each biotherapeutic. Many factors must
be assessed to choose the proper assay format, to develop a robust assay, andchoose when to invest in the development and implementation of these assays.
This short course will focus on these topics and provide examples of current
industry practices and publications.

TUESDAY, OCTOBER 15 2:00 PM – 5:00 PM

SC2: Overcoming Drug and Target Interference in ADA
and NAb Assays
Instructors:
Lynn Kamen, PhD, Scientific Officer, Executive Director, BioAgilytix
Weifeng Xu, PhD, Director, Bioanalytical, Merck

Soluble drug, drug target, and matrix can often interfere in the detection of anti-drug
antibodies, including neutralizing Abs. Although not always straightforward, it
can be addressed and mitigated in a properly designed immunoassay. This shortcourse will give an overview of the different types of interferences, and current
methodologies and approaches being utilized to resolve or reduce them.

TUESDAY, OCTOBER 15 5:30 PM – 8:30 PM

SC4: Recent Advances with Cell and Gene Therapy
Instructor:
Jim McNally, PhD, CSO, BioAgilytix

Topics to be covered in this course include: Immunogenicity assessment ofcell therapies, examining recent developments with CAR T cells & edited stem
cell, immunogenicity assessment of gene therapies, recent data on pre-existing
reactivity for AAV, advances with redosing, application of current guidance to novel
modalities, and what is your product, the vector, the expressed product?

THURSDAY, OCTOBER 17 6:00 PM – 9:00 PM

SC5: Advice on Putting Together an Integrated
Summary of Immunogenicity

Instructor:
Bonnie Rup, PhD, Biotechnology Consultant, Bonnie Rup Consulting
The purpose of this workshop is to share experience gained in preparing andreviewing the “Integrated Summary of Immunogenicity (ISI)” for submission in
regulatory filings. We will overview examples of the multi-disciplinary informationthat is most useful for the regulator assessing the scale of risk of undesirable
immunogenicity for overall clinical benefit vs. risk. We will also examine thesponsor team’s role, the general format of an ISI, and provide examples of how
to anticipate and address potential issues (and how to avoid introducing any new
ones!) by generating a well-thought-out and constructed integrated summary.

TUESDAY, OCTOBER 15 5:30 PM – 8:30 PM

SC3: Validation of ADA Assays and Cut Point
Calculations

Instructors:
Kavitha Akula, PhD, Principal Scientist, Bristol Myers Squibb Co.
Krupa Ramani, Manager, Johnson & Johnson
Jiangbo Tang, PhD, Senior Scientist, Bristol Myers Squibb Co.
This short course will focus on the validation of ADA assays and cut-point
evaluations. We will provide an in-depth overview of the basic considerations
around ADA assay validation, with significant focus on the process of evaluating
different types of cut-points, and the translation of the cut-point established during
validation to the real-world implementation during a preclinical or clinical study.

“The Summit was a fantastic meeting. It was an
exciting opportunity to connect with colleagues
and collaborators and to discuss the cutting-edge
developments in protein drug immunogenicity.”- Assistant Professor, The University of Kansas

ImmunogenicitySummit.com | 5

S1: Immunology for Biotherapeutics
Understanding and Manipulating the Immune System for
Therapeutic Advantage

TUESDAY, OCTOBER 15
8:30 am Registration and Morning Coffee

CURRENT UNDERSTANDING OF IMMUNEMECHANISMS
9:50 Chairperson’s Remarks
Kannan Natarajan, PhD, Staff Scientist, NIAID, NIH
10:00 FEATURED PRESENTATION: Antigen

Processing and Presentation: The Basis of T Cell
Activation
Kannan Natarajan, PhD, Staff Scientist, NIAID, NIH

Antigen-presenting cells process protein antigens into peptides
for binding by either Major Histocompatibility Class I (MHC-I) or Class II(MHC-II) molecules, which are then displayed at the cell surface as peptide/
MHC complexes, where they are recognized by T cell receptors leading to
T cell activation. Cell biological, biochemical, and structural details of theseprocesses as we now understand them will be discussed.

10:30 Role of IgE and IgG/IgG4 in Modulating Type I Hypersensitivity
Reactions in Human Allergic Disease
Robert Hamilton, PhD, D.ABMLI, Professor, Medicine & Pathology, Clinical
Immunology & Allergy, Johns Hopkins University
Of the four areas of hypersensitivity, this presentation will focus on immediate
Type I IgE-mediated, allergic disease. The presentation examines pathophysiology,
current diagnostic strategies, four modes of disease management, and special
caveats relating to food, drug, venom, and respiratory allergic disease. Importantly,
the newly expanded discipline of molecular allergology will be highlighted with anemphasis on ten cross-reactive allergen families and how allergenic molecules
improve the accuracy of allergy diagnosis.

11:00 Networking Coffee Break
11:15 The Role of the Innate Immune System and Implications for
Biotherapeutics
Han-Yu Shih, PhD, Investigator, NeuroImmune Regulome, National Institutes of
Health National Eye Institute
The field of innate lymphoid cell (ILC) biology has progressed rapidly, highlighting
these cells’ roles in immunity, barrier tissue integrity, and homeostasis. ILCs can
be classified based on cytokine production, mirroring patterns in CD4 T helper (Th)cell analogs. Unlike Th cells, ILCs respond promptly to pathogens without needing
antigen-specific receptor recognition. Understanding ILC differentiation andimmunoregulation is key to developing new treatments for autoimmunity, infection,and cancer.

11:45 Complement as an Apex Regulator of Tissue Inflammation
Ben Afzali, MD, PhD, FRCP, Earl Stadtman Investigator, Immunoregulation
Section, Kidney Diseases Branch, NIDDK, NIH
Our lab investigates tissue inflammation, injury, and repair, focusing on gene
expression influenced by micro-environmental signals and transcription factors
using functional genomics approaches. An important aspect of our work centersaround complement components produced by cells in tissues. In this talk, wewill discuss the mechanisms and roles of locally produced complement ininflammation, how this influences tissue biology, and the key transcriptional
regulators involved.

12:15 pm Luncheon Presentation (Sponsorship Opportunity Available)

or Enjoy Lunch on Your Own
12:45 Session Break

6 | ImmunogenicitySummit.com

October 15, 2024

HARNESSING THE IMMUNE SYSTEM FOR
BIOTHERAPEUTICS
2:00 Chairperson’s Remarks
Paul Moore, PhD, CSO, Zymeworks
2:05 Pushing the Boundaries of Antibody-Based Therapeutics
through Multispecifics and Drug ConjugatesPaul Moore, PhD, CSO, Zymeworks
Antibody-based therapeutics have provided great therapeutic benefit to many
patients across various disease states. Multispecific antibodies afford therapeutic
opportunities not feasible with single-target antibodies or combinations, while drug
conjugates provide opportunity to extend therapeutic benefit through combining thetargeting specificity of an antibody with a “payload.” Examples of these advances
will be summarized in the context of molecule design, target selection, biologicalcharacterization, and clinical benefit.

2:35 Harnessing the Body’s Natural Immune Response to Fight
CancerDaron Forman, PhD, Senior Principal Scientist, Discovery Biotherapeutics,
Bristol Myers Squibb Co.
Immunotherapy has shown remarkable response rates in some previously hardto-treat cancers by redirecting the body’s own immune system to recognize andeliminate tumor cells. Here, we will discuss the current state of immunotherapy bybriefly covering cytokine therapy, cancer vaccines, adoptive immunotherapy, andimmunomodulation therapy.

3:05 Networking Refreshment Break
3:30 Emerging Immunogenicity Obstacles for Next-Generation
Biotherapeutics
Andreas Hollenstein, PhD, Principal Scientist, Immunosafety, Roche
Since the beginning of immunotherapy using monoclonal antibodies, we havepushed for higher efficiency of this therapeutic modality. This shift did not comewithout trade-offs for safety and immunogenicity. This presentation will cover
several examples of drug-enhancing approaches that also can have a profound
impact on immunogenicity. Understanding the underlying mechanisms could help
to stay far away from immunogenic transformation and lead to the development ofbetter drugs for patients.

4:00 PANEL DISCUSSION: Harnessing the Immune System for
Biotherapeutics
Moderator: Paul Moore, PhD, CSO, Zymeworks
Panelists:
Daron Forman, PhD, Senior Principal Scientist, Discovery Biotherapeutics,
Bristol Myers Squibb Co.
Andreas Hollenstein, PhD, Principal Scientist, Immunosafety, Roche
4:30 Close of Immunology for Biotherapeutics Symposium
5:00 Dinner Short Course Registration
5:30 Recommended Dinner Short Course*

SC4: Recent Advances with Cell and Gene Therapy*Separate registration required. See short course page for details.

THURSDAY, OCTOBER 17
6:00 pm Recommended Dinner Short Course*

SC5: Advice on Putting Together an Integrated Summary of Immunogenicity*Separate registration required. See short course page for details.

W1: Biostatistics for Beginners*
October 15, 2024
*Available in-person only
Nancy Sajjadi, Independent Quality
Consultant, Sajjadi Consulting

TUESDAY, OCTOBER 15 9:00 AM – 5:00 PM

W1: Biostatistics for Beginners

Instructor:
Nancy Sajjadi, Independent Quality Consultant, Sajjadi Consulting
Statistical analysis is an integral part of designing, developing, validating, andimplementing a bioassay. Statistical tools and techniques are required forexperimental design, selecting an appropriate model for the dose response
curve, evaluating system and sample suitability, and to measure, control, andcommunicate the uncertainty of reported potency results. Fundamental concepts
are also integral to developing product specifications and making decisions for
lot release. This workshop is intended for people who generate or review bioassay
data but have minimal training in statistics. This slower-paced course will help
participants understand the meaning of commonly encountered statisticalinformation in context and expand their knowledge of fundamental concepts andtools that are applicable to their daily work. Sufficient time is allocated at the endof the day to review specific topics by request and further discuss participantquestions.

Nancy Sajjadi, M.Sc. is Founder and Principal
Consultant of Sajjadi Consulting. She has over 30
years of experience in biopharmaceutical productdevelopment. She began her career as a benchscientist doing malaria vaccine research then joined
a start-up company developing cell and gene using
therapies for infectious disease, cancer and celltherapy applications. Her responsibilities included
research, assay development, and quality control. In 2000, she left her position
as Director of QC at Chiron Technologies Center for Gene Therapy to start her
own consulting business. For over 20 years, Ms. Sajjadi has provided services tobiopharmaceutical companies, contract laboratories, non-profit organizations,
universities and US government agencies. She has assisted clients in thedevelopment, implementation and improvement of quality programs for a rangeof cutting-edge products and provides technical expertise in assay development,
qualification and validation. Ms. Sajjadi has authored several articles pertaining
to bioassays and viral gene therapy and has served on 5 advisory panels for theUnited States Pharmacopeia (USP). She enjoys teaching introductory courses
in bioassay design, development and validation for non-statisticians. Her
company has recently expanded services to include leadership development andexecutive coaching to guide organizations toward sustaining a culture of quality.

“A great opportunity to meet FDA colleagues andinteract with experts in different fields.”- Bioanalytical Principal Investigator,
Senior Scientist, Pfizer
ImmunogenicitySummit.com | 7

C1A: Immunogenicity Assessment & Clinical Relevance
Assay Strategy for Meaningful Evaluation

TUESDAY, OCTOBER 15
9:00 am Recommended Short Course*

SC1: Development of NAb Assays, Technical Considerations, Case Studies*Separate registration required. See short course page for details.

2:00 pm Recommended Short Course*

SC3: Validation of ADA Assays and Cut Point Calculations*Separate registration required. See short course page for details.

5:30 Recommended Dinner Short Course*

SC5: Advice on Putting Together an Integrated Summary of Immunogenicity*Separate registration required. See short course page for details.

WEDNESDAY, OCTOBER 16
7:45 am Registration and Morning Coffee

CLINICAL RELEVANCE OF IMMUNOGENICITY—LATEST
FDA FEEDBACK
8:25 Chairperson’s Opening Remarks
Alexander Kozhich, PhD, Director, Bristol Myers Squibb Co.
8:30 KEYNOTE PRESENTATION: The Role of ClinicalPharmacology and Clinical Relevance of
Immunogenicity

Yow-Ming Wang, PhD, Associate Director for Biosimilars andTherapeutic Biologics, CDER, FDA
To address the concern of negative impact of immunogenicity on treatment
effects, therapeutic drug monitoring has been adopted as a mechanism ofpatient management in some clinical specialties. Advances in leveraging
clinical pharmacology data have offered an option for evaluating clinicalimpacts. This presentation will discuss the current challenges and potential
future directions based on the review experience in the Office of ClinicalPharmacology at the FDA.

9:00 Therapeutic Drug Monitoring for Biologics—Challenges andOpportunities

Michele Gunsior, PhD, Senior Director, Astria Therapeutics
Therapeutic drug monitoring (TDM) for large molecules is not widely practiced
despite reported benefits in patient outcomes for some immune-mediatedinflammatory diseases. A joint US-FDA (OCP)/AAPS workshop was convened
in February 2024, to discuss the potential benefits, limitations, and future
considerations for implementation of large molecule TDM. Among the potential
benefits is preventing secondary loss of response due to suboptimal dosing andpotential generation of anti-drug antibodies.

9:30 Rational ≠ Radical: A Scientific Perspective on

Immunogenicity Risk and Assessment

Lauren Stevenson, CSO & Head of Translational Sciences,
Immunologix LabsThis presentation will highlight the context in which current practices for
immunogenicity risk assessment and the 3-tiered testing paradigm were developed,
followed by discussion of lessons learned over the last 20+ years of implementation.
Leveraging those learnings, a scientific perspective that reframes immunogenicity asa biomarker will be presented that demonstrates how adopting a biomarker mindset
with focus on context-of-use can be applied to (1) refine risk assessments to focus
on likelihood of clinically meaningful immunogenicity (2) develop assay and testing
strategies that enable a more comprehensive view of immunogenicity data, and (3)bring clarity to data interpretation that simplifies communication of conclusions to
key stakeholders, with potential to reduce burden on regulatory reviewers.

8 | ImmunogenicitySummit.com

10:00 Networking Coffee Break
10:15 Challenges Associated with Assessing the Clinical Effects of

Immunogenicity

Diana Montgomery, PhD, Principal Scientist, Pharmacokinetics, Merck & Co., Inc.
Evaluating the clinical effects of immunogenicity has become more challenging
with the advent of highly sensitive ADA assays that result in a higher proportionof ADA-positive participants. This talk will discuss the challenges associated withconstructing a cross-functional group to investigate potential clinical effects on PK,
PD, efficacy, and safety. Displays helpful for exploration of the data and in distilling
meaningful clinical effects will be presented.

FDA UPDATES - LABELING, PEPTIDES, ASSESSMENT
10:45 Guidance on Immunogenicity in Drug Product Labeling
Eric Brodsky, MD, Associate Director, Labelling Policy Team, CDER, FDA
This presentation will discuss incorporating immunogenicity information into theprescribing information for human drugs and biological products.

11:05 Presentation to be Announced
11:25 Assessing Immunogenicity Using Immune Humanized Mice
Kristina Howard, Principal Investigator, Division of Applied Regulatory Science(DARS), CDER, FDA
This talk discusses the use of immune humanized mice to assess immunogenicity
in drug development. It explores the methodology for creating and utilizing thesemice to mimic human immune responses, providing a valuable preclinical tool for
predicting immunogenic reactions to therapeutics. The focus is on enhancing theaccuracy of immunogenicity assessments, which is crucial for the safety and efficacyof new drugs, particularly biologics and biosimilars.

11:45 Luncheon Presentation (Sponsorship Opportunity Available) or

Enjoy Lunch on Your Own

12:15 pm Session BreakIMMUNOGENICITY OF GENE THERAPIES AND AAVs
1:15 Chairperson’s Remarks
Ronit Mazor, PhD, Principal Investigator, CBER, FDA
1:20 Immunogenicity Assessment of Gene Therapy Products
Anurag Sharma, PhD, Gene Therapy Reviewer, CBER, FDA
Despite great promise, the immunogenicity of gene therapies remains one of thebiggest challenges. Immunogenicity can impact the safety, efficacy, and long-term
durability of gene therapies. Addressing immunogenicity-associated challenges iscritical to realizing the full potential of these life-saving therapies. In my talk, I will
discuss FDA’s perspective on the immunogenicity associated with gene therapies.

1:50 Rational Immunosilencing of a Promiscuous T Cell Epitope in theCapsid of an Adeno-Associated Virus

Sojin Bing, PhD, Staff Fellow, Office of Tissues & Advanced Therapies, FDA CBER
We aim to reduce immune responses to AAV therapeutics by elimination of capsid
T cell epitopes within the AAV capsid. To reduce or eliminate immunogenicity, it isvital that the method that eliminates MHC-binding epitopes does not disrupt vector
structure and function. Here, we describe the rationally designed chimeric AAV vector
using integration of ex vivo T cell assays, MHC epitope prediction, and sequenceconservation analysis in AAV phylogeny.

OCTOBER 16-17, 2024
INTERACTIVE DISCUSSIONS: IN-PERSON ONLY
2:20 Interactive Discussions

Interactive Discussions are informal, moderated discussions, allowing
participants to exchange ideas and experiences and develop futurecollaborations around a focused topic. Each discussion will be led by afacilitator who keeps the discussion on track and the group engaged. To get themost out of this format, please come prepared to share examples from yourwork, be a part of a collective, problem-solving session, and participate in active
idea sharing. Please visit the Breakout Discussions page on the conference
website for a complete listing of topics and descriptions.

THURSDAY, OCTOBER 17
7:30 am Registration and Morning Coffee

ANALYSIS OF IMMUNOGENICITY
7:55 Chairperson’s Remarks
Susan Irvin, PhD, Senior Principal Scientist, Bioanalytical Strategy, Regeneron
8:00 The Immunogenicity Journey towards Winrevair FDA Approval
Linlin Luo, PhD, Senior Director, Merck

TABLE 2: Recommendations for Harmonization of Neutralizing
ADA Testing and Reporting

With the recent approval for Winrevair, we believe it is valuable to share Merck’s
experience in immunogenicity data analysis and reporting, as well as our approaches
for analyzing the clinical impact of immunogenicity. These efforts have culminated in
the development of the Integrated Summary of Immunogenicity (ISI) contributing to
FDA approval for Winrevair.

TABLE 3: When One Size Does Not Fit All: Reevaluating
Immunogenicity Risk Assessments and The 3-Tiered Testing
Paradigm

8:30 Analysis of Clinical Immunogenicity Data for a Multi-Study Program
Susan Irvin, PhD, Senior Principal Scientist, Bioanalytical Strategy, Regeneron

Michele Gunsior, PhD, Senior Director, Astria Therapeutics

Lauren F. Stevenson, PhD, CSO & Head, Translational Sciences,
Immunologix Labs

3:10 Grand Opening Refreshment Break in the Exhibit Hall with Poster

Viewing

IMMUNOGENICITY OF NOVEL THERAPIES, CAR Ts
4:00 Immunogenicity Strategy and Challenges for Newer Modalities
Jenny Valentine, PhD, Senior Principal Scientist, Bioanalytical Sciences,
Regeneron
Many drug modalities in clinical development are significantly more complex than
traditional monoclonal antibodies. While the ADA assessment strategy for mAbs
is well-established in White Papers and regulatory guidance, the immunogenicity
evaluation requirements for newer modalities are still largely undefined. In thispresentation we will discuss several newer modalities and the immunogenicity
challenges for ADA/NAb testing, focusing on how the overall immunogenicity
assessment will be used to inform clinical decisions.

4:30 FEATURED PRESENTATION: Immunogenicity to

CAR T Therapy: Mechanisms, Clinical
Considerations, and Mitigation Strategies

Murli Krishna, PhD, Senior Principal Scientist, Bristol Myers
Squibb Co.
Immunogenicity of CAR T cell therapy can influence effectiveness and lead topotential side effects. This presentation will discuss factors contributing to
the immunogenicity, studies that demonstrate its impact on CAR T cell therapy
outcomes like persistence and functionality of CAR T cells, and CAR design
technologies for mitigating it.

5:00 Blood Processing Considerations for Clinical Cellular

Immunogenicity Assessment

Michael D. Swanson, PhD, Associate Scientific Director, Bioanalytical Discovery &Development Sciences, Johnson and Johnson Innovative Medicine
Cellular immune responses have the potential to impact clinical safety and efficacy
of various modalities including gene, cellular, and protein-based therapies. In thispresentation, we will discuss how variables such as patient blood draw, transit
conditions, peripheral blood mononuclear cell isolation, and storage can impact
downstream assays that assess cellular immune responses to therapeutics.

5:30 Welcome Reception in the Exhibit Hall with Poster Viewing
6:30 Close of Day

We report data from a mAb therapeutic across multiple studies that include an
examination of the impact of dose, route of administration, sample time points, andpatient populations. We highlight the relationships among these aforementioned
variables, as well as important implications for development of therapeutic proteinsin the future.

9:00 Presentation to be Announced
9:30 Coffee Break in the Exhibit Hall with Poster Viewing
10:15 Intravenous vs. Subcutaneous: How Delivery Route Impacts

Immunogenicity of Protein Therapeutics

An Zhao, PhD, Senior Principal Scientist, Bioanalytical Sciences, Regeneron
Pharmaceuticals, Inc.
Subcutaneous delivery of therapeutic proteins can increase patient compliance andreduce burden on healthcare systems compared to intravenous infusion. However,subcutaneous administration has been proposed to increase immunogenicity. We
reviewed anti-drug antibody (ADA) data for various protein therapeutics that wereadministered both intravenously and subcutaneously, and overall, no difference inimmunogenicity incidence was observed. Although administration route may in someinstances influence ADA, other risk factors are likely more impactful.

10:45 Detection and Characterization of Preexisting Antidrug

Antibodies towards Multi-Domain Biotherapeutics—A Case Study

Yan Mao, PhD, Senior Principal Scientist, Boehringer Ingelheim Pharmaceuticals
This presentation will focus on a case study from one of Boehringer-Ingelheim’s
multi-domain biotherapeutic programs to demonstrate our strategies for detection
and characterization of PE-ADA. This case will provide in detail the specialized tool
molecules along with the methods used to characterize the domain specificity andidentify the location of PE-ADA. It will also share our strategy for the development ofan ADA method to distinguish between PE-ADA and TE-ADA.

11:15 Revisiting Antidrug Antibody Assay Drug Tolerance
James Zanghi, PhD, Senior Principal Scientist, BioAnalytical Sciences,
Genentech, Inc.
Drug tolerance is a critical attribute of anti-drug antibody (ADA) assays for assessing
clinical immunogenicity. We present a case study where a previously approved
product required reassessment of the assay drug tolerance to meet both increased
drug exposure demands and more rigorous validation requirements since theproduct’s launch. The strategy to overcome these issues while working withinchallenging timelines can be broadly applied to other therapeutics.

11:45 Close of Immunogenicity Assessment & Clinical RelevanceConference

6:00 pm Recommended Dinner Short Course*

SC5: Advice on Putting Together an Integrated Summary of Immunogenicity*Separate registration required. See short course page for details.

ImmunogenicitySummit.com | 9

C1B: Immunogenicity Prediction & Control
Regulatory Perspectives, Risk Management, and Predictive Tools

4:00 KEYNOTE PRESENTATION: What Information Can

TUESDAY, OCTOBER 15

MHC Associated Peptide Proteomics (MAPPs) Assays
Provide during Immunogenicity Risk Assessment of
Therapeutic Proteins and Novel Modalities?

9:00 am Recommended Short Course*

SC1: Development of NAb Assays, Technical Considerations, Case Studies*Separate registration required. See short course page for details.

FDA
2:00 pm Recommended Short Course*

SC4: Recent Advances with Cell and Gene Therapy*Separate registration required. See short course page for details.

5:30 Recommended Dinner Short Course*

SC5: Advice on Putting Together an Integrated Summary of Immunogenicity*Separate registration required. See short course page for details.

THURSDAY, OCTOBER 17
12:00 pm Registration OpenIMMUNOGENICITY RISK ASSESSMENT—mAbs to ADCs
1:15 Chairperson’s Remarks
Jack A. Ragheb, MD, PhD, Senior Vice President, Translational Sciences andMedicine, NexImmune
1:20 Immunogenicity Risk Assessment and Mitigation
Timothy Hickling, PhD, Investigative and Immunosafety Chapter, Roche
Immunogenicity risk assessments can be used to optimize portfolios to acceptable
immunogenicity risk levels. ADA responses vary widely in clinical trials, withthe presence of ADA itself a risk factor for clinical impact. Whilst ‘predicting
immunogenicity’ might seem impossible, forecasting incidence of ADA formation in
first-in-human trials helps to prepare teams for potential consequences. I review a risk
assessment methodology, highlighting case studies to demonstrate application at theportfolio level.

1:50 Immunogenicity of the Next-Generation of Antibody-Drug
Conjugates
Alexander Kozhich, PhD, Director, Bristol Myers Squibb Co.
Over the last several years there has been exponential increase of antibody-drug
conjugates in various stages of development. Several novel ADC formats being
explored include unnatural antibodies (bispecifics, probodies, etc.) as well as novel
payloads (immune-stimulators, protein degraders, etc.). It is important to understandthe immunogenicity risk of these novel ADCs and their clinical relevance. Better
understanding of potential immunogenicity of these novel ADCs should help in theirclinical development.

2:20 Preclinical Risk Assessment
Jack A. Ragheb, MD, PhD, Senior Vice President, Translational Sciences andMedicine, NexImmune
Testing of biotherapeutics across species typically induces an Anti-Drug Antibody(ADA) response. When the therapeutic is a monoclonal antibody (mAb), these ADA are
generally directed against the constant regions of the mAb, but may be against other
epitopes. The impact of these ADA on PK, PD, and the toxicity profile of the therapeuticmAb, as well as novel ways to mitigate their formation, will be examined.

2:50 An Integrated Approach to Managing Immunogenicity
Risk and Optimum Protein Design
Andrew Isidoridy, Immunology Sales Specialist, ProImmune, Inc.
Immunogenicity risk assessment is an essential step in bringing therapeutic drugs
to the market. ProImmune’s risk management tools evaluate immunogenic epitopes
and the corresponding functional T cell responses that can lead to unwanted immune
responses. Case studies will highlight how the integrated platform is used to address
key questions in the drug development phase.

3:20 Refreshment Break in the Exhibit Hall with Poster Viewing

10 | ImmunogenicitySummit.com

Zuben Sauna, PhD, Director, Division of Hemostasis, CBER,

In vitro assays that assess the potential immunogenicity of protein therapeutics
are useful during drug development. I will provide specific examples of theapplication of MAPPs in assessing the immunogenicity of protein therapeutics(Factor VIIa and Factor VIII). I will also discuss MAPPs assays in the context ofnovel modalities. Many novel modalities rely on intracellular protein synthesis.
Novel workflows for MAPPs assays to capture these events will be provided.

4:30 FEATURED PRESENTATION: Evolution of the

MAPPs Assay: Gaining Greater Resolution forPopulations at Risk for ADA

Robert Siegel, PhD, Associate Vice President, Laboratory for
Experimental Medicine, Eli Lilly and Company

A novel B cell MAPPs platform suitable for the examination of a variety oftherapeutic antibody scaffolds has been developed. The sensitivity andreproducibility of the system was determined. Most importantly, experiments
were performed to assess the similarity of results to those obtained from thetraditional monocyte-derived dendritic cell approach. Observations regarding themodification state of cysteine residues involved in MHC-binding core will also be
discussed.

5:00 HESI/AAPS: Towards Harmonization of in vitro T Cell Assays to
Predict Immunogenicity of Biologics
Laurent P. Malherbe, PhD, Executive Director, Eli Lilly and Company
In vitro assays are widely used preclinically during the development of biologics toassess immunogenicity liabilities and select candidates. However, there is considerable
diversity in assay format among companies and no alignment on the analysis methods.
Here we will discuss the recent progress of a cross-industry work to develop common
reference biologics for preclinical immunogenicity risk assessment, a critical first step
toward assay standardization and harmonization.

5:30 Close of Day
5:30 Dinner Short Course Registration
6:00 Recommended Dinner Short Course*

SC5: Advice on Putting Together an Integrated Summary of Immunogenicity*Separate registration required. See short course page for details.

FRIDAY, OCTOBER 18
8:00 am Registration and Morning Coffee

UNDERSTANDING AND CIRCUMVENTING THE IMMUNESYSTEM
8:25 Chairperson’s Remarks
Laurent P. Malherbe, PhD, Executive Director, Eli Lilly and Company
8:30 Understanding and Circumventing the Immune Responses toApproved Protein Therapeutics
Daniel LaGasse, PhD, Research Regulator, CBER, FDA
Immunogenicity can compromise the safety and/or efficacy of therapeutic protein
products, and is a priority issue for regulatory agencies. In addition to poor patient
outcomes, the social and economic costs associated with neutralizing antibodies are
considerable. In this presentation, I survey the immunogenicity of approved therapeuticproteins, discuss strategies for clinical management of immunogenicity, and identify
challenges associated with circumventing the immune responses to approved protein
therapeutics.

OCTOBER 17-18, 2024
9:00 Presentation to be Announced

12:30 Luncheon Presentation (Sponsorship Opportunity Available) or
Enjoy Lunch on Your Own

INTERACTIVE DISCUSSIONS: IN-PERSON ONLY

1:00 Session Break

9:30 Interactive Discussions

1:30 Chairperson’s Remarks
Sofie Pattijn, Founder & CTO, ImmunXperts, a Q2 Solutions Company

Interactive Discussions are informal, moderated discussions, allowing
participants to exchange ideas and experiences and develop future collaborations
around a focused topic. Each discussion will be led by a facilitator who keeps
the discussion on track and the group engaged. To get the most out of thisformat, please come prepared to share examples from your work, be a part of acollective, problem-solving session, and participate in active idea sharing. Please
visit the Breakout Discussions page on the conference website for a complete
listing of topics and descriptions.

1:35 A Novel B Cell-CD4 T Cell Co-Culture Assay for Preclinical
Immunogenicity Risk Assessment
Bianca Bautista, PhD, Senior Scientist, Clinical Immunology, Amgen, Inc.

TABLE 1: HESI/AAPS: Towards Harmonization of in vitro T Cell
Assays to Predict Immunogenicity of Biologics

Effective preclinical prediction of immunogenicity risk is an ongoing challenge
for biologics development. A key driver of a clinically impactful anti-drug antibody
response is the interaction between antigen-specific B cells and CD4 T cells in thegerminal center. We developed a novel B-T cell co-culture assay that can flag moleculeswith both T and B cell epitopes, identifying those most likely to drive a clinically
impactful immune response.

TABLE 2: End-to-End Immunogenicity: Risk Assessment and
Mitigation

DEIMMUNIZATION, DEVELOPABILITY, AND MACHINELEARNING

Laurent P. Malherbe, PhD, Executive Director, Eli Lilly and Company

Timothy Hickling, PhD, Investigative and Immunosafety Chapter, Roche

TABLE 3: Immunogenicity of Next-Generation Modalities, Including
Antibody-Drug Conjugates
Alexander Kozhich, PhD, Director, Bristol Myers Squibb Co.

10:20 Coffee Break in the Exhibit Hall & Last Chance for Poster Viewing

RISK ASSESSMENT AND MANAGEMENT
11:00 FEATURED PRESENTATION: Innate Immune

Response Modulating Impurities Testing for
Immunogenicity Risk Assessments

Daniela Verthelyi, MD, PhD, Chief, Laboratory of Immunology,
CDER, FDA

Comparative in vitro analytical methods to characterize innate immune response
modulating impurities can inform the immunogenicity risk and help streamline
their development of generic peptides and biosimilar proteins. This talk will
discuss the risk posed by innate immune response modulating impurities to
product immunogenicity, available assays and data interpretation, as well as
common pitfalls and remaining knowledge gaps.

11:30 Application of in vitro Immunogenicity Assays for Generic Peptide
ANDA Submissions
Andrew Graves, Director, Immunogenicity Assessment, Specialty Analytics, Teva
Pharmaceuticals
In vitro assays are a requirement for characterizing the immunogenicity risk of
impurities in generic peptide candidates submitted for ANDA consideration to the FDA
in lieu of clinical immunogenicity assessment. Guidance issued in 2021 has led to amaturing of the science and assays supporting these ANDA submissions today. Here,
we review an industry perspective on applying these assays for generic peptides andpossibly for other classes of drugs.

12:00 pm Evaluating Pre-Existing Reactivity in Biotherapeutic

Development

Shannon Howell, PhD, Principal Scientist, Immunogenicity Bioanalytical, Amgen,Inc.
As the landscape of biologic therapeutics continues to evolve with new and intricate
innovations, it is imperative to conduct a thorough assessment of immunogenicity
risks and devise a clinical strategy to mitigate potential immunological adverse events.
An integral component of this risk assessment involves evaluating the presence ofpre-existing antibodies (pre-ADAs). This presentation will introduce a multi-platform
approach for detecting and characterizing pre-ADAs, complemented by a detailed casestudy.

2:05 Accurate HLA Antigen Presentation Prediction Across all HLA-II
Loci and its Application for Protein Immunogenicity Assessment
Morten Nielsen, Professor, PhD, Department of Health Technology, Technical
University of Denmark
HLA antigen presentation is the cornerstone of adaptive immunity. My talk will describe
our journey towards accurate prediction of HLA antigen presentation. I will demonstrate
how the end result has unprecedented accuracy across all three HLA-II loci, and how
this reveals an earlier underappreciated contribution of DRB345, DQ and DP to theimmunopeptidome. Further, I will showcase how these loci share a critical role wheninterpreting MAPPs data and predicting immunogenicity.

2:35 FEATURED PRESENTATION: Glycotargeting as

Induction to Reduce Antidrug Immune Responses

Jeffrey A. Hubbell, PhD, Eugene Bell Professor Tissue
Engineering & Vice Dean, Molecular Engineering, University of
Chicago

The liver plays key roles in maintenance of peripheral tolerance, for example,
clearing and processing apoptotic debris including post-translational modifications
that are not centrally tolerized. We have developed glycopolymer conjugation
approaches to exploit the same antigen processing pathways utilized in the above,
but for protein drugs. Administration of the glycoconjugates in an induction regimen
reduces generation of anti-drug antibodies via modulation of T cell help.

3:05 From De-Immunization to Developability: Elucidating the Principles
behind the Uptake of Antibodies by Antigen Presenting Cells
Daniel Leventhal, PhD, Head, Immunogenicity, Xaira Therapeutics
Immunogenicity mitigation is a multifactorial design challenge. As a proof-of-concept
study, we sought to reduce MHC-II presentation of an immunogenic monoclonal
antibody. While T cell antigenicity was reduced, this correlated with improvements indevelopability (self-association and polyspecificity) rather than elimination of MHC
II epitopes. This prompted an expanded analysis of over 100 clinically evaluatedantibodies that confirmed a correlation between self-association and polyspecificity
with dendritic cell uptake and immunogenicity risk.

3:35 Machine Learning for Deimmunization and Multi-Objective
Optimization of Biologics
Ryan Peckner, PhD, Director, Machine Learning, Seismic Therapeutic
We develop machine learning models to optimize in parallel multiple drug-like
properties of biologics while minimizing liabilities including immunogenicity. We apply
these to the bacterial enzyme IdeS to design a therapeutic for chronic autoantibodymediated diseases, and demonstrate the success of this approach via in vivo and invitro assays. We also illustrate its generalizability by applying it to deimmunize a widearray of clinically approved biologics in silico.

4:05 Close of Summit

ImmunogenicitySummit.com | 11

C2B: Optimizing Bioassays for Biologics
Successful Bioassay Development in an Era of Emerging Therapeutic Modalities

TUESDAY, OCTOBER 15
9:00 am Recommended Short Course*

SC1: Development of NAb Assays, Technical Considerations, Case Studies*Separate registration required. See short course page for details.

2:00 pm Recommended Short Course*

SC2: Overcoming Drug and Target Interference in ADA and NAb Assays*Separate registration required. See short course page for details.

5:30 Recommended Dinner Short Course*

SC3: Validation of ADA Assays and Cut Point Calculations*Separate registration required. See short course page for details.

THURSDAY, OCTOBER 17
12:00 pm Registration OpenBIOASSAY DESIGN AND POTENCY ASSAY DEVELOPMENT
1:15 Chairperson’s Remarks
Ravish B. Patel, PhD, Senior Manager, Pharmacology, Sun Pharma Advanced
Research Center
1:20 Impact of Product Variants on Product Functional Activity andStability in the Context of the Biosimilars
Ravish B. Patel, PhD, Senior Manager, Pharmacology, Sun Pharma Advanced
Research Center

extensively applied.
Challenges and Barriers: Identification of major obstacles preventing widespread
adoption in bioanalysis. Discussion on technical, regulatory, and operational
challenges.
Benefits of Automation: Potential improvements in efficiency, accuracy, andreproducibility. Impact on data integrity, compliance, and throughput.
Case Studies and Success Stories: Examples of successful implementation of
automation in bioanalytical settings. Lessons learned and best practices from these
implementations.
Future Prospects: Emerging trends and future directions for bioanalytical automation.
Potential for integration with AI and machine learning for enhanced capabilities.

3:20 Refreshment Break in the Exhibit Hall with Poster Viewing
4:00 Novel Cell Construct Design for Development of a Functional CellBased Potency Assay
Kevin Gurney, PhD, Distinguished Scientist, Analytical Research and Development,
Merck
An immunotherapy that binds to ILT3 can block the ligand-ILT3 interaction, reversing an
inhibitory signal cascade. A novel chimeric receptor reporter cell-line was designed andengineered that inverted ILT3 from an inhibitory to an activation motif, ITIM to ITAM tomeasure drug-induced regulation of gene transcription. This cell-based potency assay
enabled anti-ILT3 drug disruption of ligand interaction and was sensitive to anti-ILT3
drug stability stresses.

4:30 Considerations for Potency Assurance of CGT Products
Diana Colleluori, PhD, MBA, Principal CMC Consultant, CMC Analytical, Biologics
Consulting Group
Sponsors must develop a potency assurance strategy early in drug productdevelopment. The recently issued FDA draft guidance on potency assurance of CGT
products will be discussed, along with a risk-based approach to developing a productpotency assurance and bioassay development strategy.

A potency assay is a regulatory requirement for the release of every lot of abiotherapeutic. For complex biologics, manufacturing processes may be optimized
to yield an active ingredient, but a company may end up with reduced activity due
to removal of key components. Changes in potency can occur through a variety ofphysicochemical or structural changes. A meaningful and reliable assay is the mostimportant tool in drug development.

5:00 Standards Development and Measurement Assurance Strategies
for Cell Characterization Assays

1:50 Limiting Potency Bias from Allowed Non-Similarity While

Sumona Sarkar, PhD, Biomedical Engineer, Biosystems and Biomaterials Division,
Biomaterials Group, National Institute of Standards and Technology

Protecting the Similarity Pass RateDavid Lansky, PhD, President, Precision Bioassay, Inc.
Protecting bioassay-based estimates of relative potency against bias and unacceptably
high similarity failure rates, while allowing for changes in assay capability appears to be
impractical. While power calculations for both detecting non-similarity (via difference
tests) and for passing similar samples (via equivalence tests) are helpful, thesecalculations are not simple. This presentation will illustrate tools for these calculations
and how they support modifying similarity criteria based on data from bioassay.

2:20 Simplifying Cell-Based Potency Assays for Biologics and Gene

Therapy Products

Arkadi Manukyan, PhD, Senior Scientist, Bioassay Development, Sanofi
Potency assays are essential to demonstrate the mechanism-of-action of drug
products in clinical trials for regulatory approval. Here, we discuss the strategies andchallenges of the development and qualification of potency assays for gene therapy
products and biologics. Two case studies will be used to demonstrate how to simplify
the method by making it more robust, less time-consuming, easy to maintain, andrequiring fewer critical reagents.

STANDARDS AND REFERENCE MATERIALSThe manufacturing and release of cellular therapy products requires high-quality,
robust, and validated analytical methods. Here I will describe recent developments
in analytical method standardization, including the FDA Standards Recognition
Program and NIST public-private partnerships to support the development of criticalanalytical methods. A key aspect of analytical development is the need for a fit-forpurpose approach. Efforts to identify and establish fit-for-purpose process analyticaltechnologies will also be described.

5:30 Close of Day
5:30 Dinner Short Course Registration
6:00 Recommended Dinner Short Course*

SC5: Advice on Putting Together an Integrated Summary of Immunogenicity*Separate registration required. See short course page for details.

FRIDAY, OCTOBER 18

2:50 Title: Unlocking the Potential of Bioanalytical

8:00 am Registration and Morning Coffee

Tom Zhang, Chief Scientist, Large Molecule Bioanalysis, www.worldwide.com

GETTING DOWN TO BIOASSAY BRASS TACKS: MEASURING
POTENCY-RELATED CRITICAL QUALITY ATTRIBUTES FOR
CELL AND GENE THERAPY (CGT) PRODUCTS

Automation: Bridging the Gap between Development andApplication
Introduction to Bioanalytical Automation: Overview of the development of bioanalytical
automation over the years. Key technological advancements and innovations in thefield.
Current State of Application: Examination of the limited application of automationin bioanalytical laboratories. Comparison with other fields where automation is

12 | ImmunogenicitySummit.com

8:25 Chairperson’s Remarks
Nancy Sajjadi, Independent Quality Consultant, Sajjadi Consulting
8:30 Biologist and Biostatistician Regulatory Perspectives onOCTOBER 17-18, 2024
Development of Bioassays to Support CGT Products
Jennifer Kirk, PhD, Lead Mathematical Statistician, FDA CBER
Leslie Wagner, Consumer Safety Officer, FDA CBER
9:00 Presentation to be Announced
9:15 Presentation to be Announced (Opportunity Available)

INTERACTIVE DISCUSSIONS: IN-PERSON ONLY
9:30 Interactive Discussions

Interactive Discussions are informal, moderated discussions, allowing
participants to exchange ideas and experiences and develop future collaborations
around a focused topic. Each discussion will be led by a facilitator who keeps
the discussion on track and the group engaged. To get the most out of thisformat, please come prepared to share examples from your work, be a part of acollective, problem-solving session, and participate in active idea sharing. Please
visit the Breakout Discussions page on the conference website for a complete
listing of topics and descriptions.

TABLE 4: Potency Assays for Personalized Therapeutics Like CAR
T Cells
Dawn Maier, PhD, Cell and Gene Therapy CMC, AD Preclinical TechnicalAdvisor, DGMAIER Consulting• What are the types of biological assays available?
• What are the challenges associated with those assays?
• Why is potency for personalized therapy more complex than traditional
biologics?
• What are potential surrogate assays​?

TABLE 5: The Impact of Glycosylation on the Functional Activity of
Therapeutic Proteins
Ravish B. Patel, PhD, Senior Manager, Pharmacology, Sun Pharma Advanced
Research Center

10:20 Coffee Break in the Exhibit Hall & Last Chance for Poster Viewing
11:00 The Challenges Associated with Developing a Potency Assay

for Advanced Therapies Such as CAR T Cells and the Importance of
Qualifying Those Assays Early in Clinical Development—Lessons Learned

Dawn Maier, PhD, Cell and Gene Therapy CMC, AD Preclinical Technical Advisor,
DGMAIER Consulting
Advanced therapies such as CAR T cells are complex living drugs with multiple
mechanisms-of-action. Due to this complexity, the strategy for developing andqualifying a single potency assay that correlates with efficacy has been challenging. In
this talk, we will address the pros and cons of various assays commonly used, as wellas the advantage of developing these assays in early clinical development.

11:30 Key Statistical Considerations in Interpreting USP General Chapter
Matthew Stephenson, PhD, Director of Statistics, Quantics Biostatistics
The United States Pharmacopeia (USP) General Chapter <1033> provides guidanceon the validation of biological assays. We highlight key statistical considerations in
the interpretation of this guidance, including setting acceptance criteria based on
the probability of out-of-specification results and the use of total analytical error toquantify accuracy and precision. These reflect an evolving approach towards bioassay
validation, underscoring current best practices and continual improvement.

12:00 pm PANEL DISCUSSION: Getting Down to Bioassay Brass Tacks
Moderator: Nancy Sajjadi, Independent Quality Consultant, Sajjadi Consulting
Uncertainty about how regulatory authorities determine the suitability of potency
assays still persists. Successful bioassay development takes into consideration (1)current regulatory requirements and guidance, (2) the nature of the product, and (3)the statistical tools needed to evaluate decisions. This session is intended to stimulatedialogue about the challenges faced by stakeholders in the development of bioassays
to support CGT products and increase clarity about current regulatory thinking.

Panelists:
Dawn Maier, PhD, Cell and Gene Therapy CMC, AD Preclinical Technical Advisor,
DGMAIER Consulting

Matthew Stephenson, PhD, Director of Statistics, Quantics Biostatistics
Leslie Wagner, Consumer Safety Officer, FDA CBER
Jennifer Kirk, PhD, Lead Mathematical Statistician, FDA CBER
12:30 Luncheon Presentation (Sponsorship Opportunity Available) or
Enjoy Lunch on Your Own
1:00 Session BreakIN VITRO ASSESSMENTS FOR IMMUNOGENICITY
1:30 Chairperson’s Remarks
Kevin Gurney, PhD, Distinguished Scientist, Analytical Research and Development,
Merck
1:35 A Mechanism-Based Dendritic Cell Migration Assay to Predict
Subcutaneous Immunogenicity
Nicole Jarvi, PhD, Senior Scientist, Merck
Subcutaneous (SC) delivery can exacerbate immunogenicity of biologics where
injected protein is exposed to skin-derived dendritic cells (DC) migrating into theinjection site and toward lymph nodes. In a newly developed, mechanism-based
dendritic cell migration assay, chemokine receptor expression on DCs and their invitro migration correlated strongly with clinical immunogenicity incidence. This assay
can inform decision-making of SC immunogenicity risk, with applicability to screen
formulation and quality attributes.

2:05 FEATURED PRESENTATION: Immunogenicity Risk

Assessment of Biotherapeutic Attributes Using invitro, in vivo, and Clinical Data

Marisa Joubert, PhD, Scientific Director and Group Leader,
Amgen

Understanding the risk of immunogenicity of Critical Quality Attributes (CQAs) is
crucial to ensuring the safety and efficacy of biotherapeutic drug products. There
are a variety of approaches and model systems for assessing risk that leveragein vitro, in vivo, and clinical data. This talk will share a few case studies assessing
risk of immunogenicity of biotherapeutic attributes and the model systems
employed.

2:35 Immunogenicity Risk Assessment and Mitigation Strategy for
Biotherapeutic Lead Selection
Yinyin Li, PhD, Principal Scientist, Biochemical & Cellular Pharmacology,
Genentech, Inc.
To understand, manage, and minimize the impact of immunogenicity on the clinicaltolerance and activity of drug discovery, we’ve established a suite of in-vitro tools for
immunogenicity risk assessment and mitigation during therapeutic lead identificationand optimization. I will share a case study demonstrating the application of multiple
tools in assessing immunogenicity risk. You will gain insights into how they can beused to drive decision-making for therapeutic lead selection.

BIOASSAY STRATEGY FOR NEW MODALITIES
3:05 Neutralization Antibody Assay for Antibody-Drug Conjugate (ADC):
Killing or Non-Killing?
Weifeng Xu, PhD, Director, Bioanalytical, Merck
All approved ADCs have cell-killing NAb assays and industrial consensus is consistent
with this. Multiple MOAs exist in vivo for ADCs; however, in vitro NAb assays can only
monitor target-involved internalization and killing. In addition, artifacts may exist for invitro cell-killing NAb assays, for example, FcgR-involved non-specific killing. We thus
propose that like mAb therapeutics, cell-binding or competitive ligand-binding NAb
assays could be considered for ADC.

4:05 Close of Summit

ImmunogenicitySummit.com | 13

TS1A: A Guide to Statistical Methods for Bioassay*
OCTOBER 16-17, 2024
*Available in-person only

TS1: A Guide to Statistical Methods for Bioassay

David Lansky, PhD, President, Precision Bioassay, Inc.

Instructor:
David Lansky, PhD, President, Precision Bioassay, Inc.

This course introduces statistical ideas supporting bioassays (illustrated
with relevant examples) and reviews properties of bioassays. The course
covers how practical constraints in the laboratory create the ‘statisticaldesign structure’ of assays. Adapting design of experiments (DOE) tobioassay development, validation, and monitoring involves using the assay
design structure. Examples (mostly from cell-based bioassays; some using
robotics) illustrate strategic approaches to early development, measuring
assay capability, improving a bioassay, and doing validation. Strategic
assay design considerations combine with good assay analysis methods
to offer good assay monitoring with graphical and quantitative tools as
part of a lifecycle approach.

David has been practicing statistics on bioassays (andother non-clinical applications in Pharma) for 35 years.
This includes Searle/Monsanto/Pharmacia (10 years)
and as the owner of Precision Bioassay, Inc. (since 2002).
Most of his bioassay experience involves helping teams
improve and validate cell-based bioassays. His experience
includes some early (late 1990’s) success as part of ateam that used lab automation for a series of cell-based
bioassays. He has been and is still an active participant
in the work to revise the USP bioassay chapters. His
education includes a year of Electrical and Computer Engineering (University ofMichigan), a BS in Botany (San Francisco State), an MS in Entomology (Cornell) andfinally both an MS and Ph.D. in Biometry (both Cornell).

See the website for agenda & further details

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Hotel & Travel Information
Conference Hoteland Venue:
The Watergate Hotel
2650 Virginia Ave NW
Washington, D.C. 20037

Discounted Room Rate: $309 s/dDiscounted Room Rate Cut-off Date:
September 16, 2024
Reservations and Additional Travel
Information:
Go to the travel page of
ImmunogenicitySummit.com

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further exposure by presenting their work in the poster sessions.
To secure an onsite poster board and/or ensure your poster is
included in the conference materials, your full submission must be
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Register and indicate that you would like to present a poster. Once
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Reasons you should present your research poster at this conference:• Your research will be seen by our international delegation, representing leadersfrom top pharmaceutical, biotech, academic, and government institutions• Discuss your research and collaborate with other attendees• Your poster will be published in our conference materials• Receive $50 off your registration

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