19-21 March 2024
Westin Miyako Kyoto
Kyoto, JapanThe Only Event in Asia for Oligo,
Peptide, mRNA, Genome Editing &Delivery Experts to Exchange Strategies
to Accelerate Molecules to Market
Industry-Leading Experts Share New Science, Applicationsand Lessons Learned
Developing Next Generation Oligonucleotide
Therapeutics

Editing for Atherosclerotic Cardiovascular Disease
Andrew Bellinger, M.D., Ph.D.,
Chief Scientific Officer, Verve Therapeutics

Shalini Andersson, Ph.D.,
Vice President Oligonucleotide
Discovery, AstraZeneca, Sweden

From Bench to Bedside: Development of a
GalXC-Plus siRNA, DCR-STAT3, for 		
Immunotherapy in Refractory Cancer Patients

Strategies for mRNA Analytics
Andreas Czech, Ph.D., Associate Director
RNA Analytics, BioNTech SE, Germany

Jennifer Lockridge, Ph.D.,
VP, Dicerna TRU Early Development,
Novo Nordisk

From Discovery to the Clinic: Development of a
Novel Bioactive Peptide that Improves Mood andCognition by Engaging the Gut-Brain Axis

Discovery and Delivery of Oligonucleotide
Therapeutics

Kousaku Ohinata, Ph.D.
Associate Professor, Kyoto University
and Founder and Scientific Advisor,
Viage Therapeutics

Jayaprakash Nair, Ph.D., 			
Vice President, Research, Chemistry 		
and Delivery Science, Alnylam

Plus Featured Presentations from:
Ionis Pharmaceuticals, Beam Therapeutics, WAVE, GlyTech, Shionogi,
Prime Medicine, Acuitas, Stoke Therapeutics, Merck, Ethrisand MANY MORE!

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www.AsiaTIDESevent.com

Apply the Latest Innovations in Oligonucleotide,
Peptide and mRNA Development

SCIENCE
Accelerate Your Product To Market
Hear CMC/manufacturing, discovery, delivery andclinical case studies, best practices and lessons
learned from leading oligonucleotide, peptide andmRNA therapeutic developers. Ensure productapproval by hearing regulatory guidance androadmaps to successful IND/IMPD submissions fromindustry leaders.

TECHNOLOGY

Evaluate New Technologies And Services
Improve your discovery, clinical, processdevelopment, analytical and manufacturing efforts
by meeting with 20+ global technology leaders in
the exhibit hall. The exhibit hall also features peersubmitted posters that contain new and unpublishedresearch from global scientists working across allphases of oligonucleotide and peptide development.

NETWORKING

Meet Your Next Partner At TIDES ASIA
Connect with 250+ oligonucleotide, peptide andmRNA leaders across Asia, Europe and North
America during networking lunches, poster
sessions, dinners and cocktail receptions.

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2

Tuesday, March 19, 2024
* OPTIONAL PRE-CONFERENCE WORKSHOPS * 9:00AM-12:45PM

8:00 Registration and CoffeeWorkshop 1

Workshop 2

Overcoming Manufacturing Challenges andDefining CMC Control Strategies of Synthetic RNA
Therapeutics

Analytical Characterization and Bioanalysis of
PeptidesWorkshop Moderator:
Thomas Rupp, Owner and Principal, Thomas Rupp Consulting AG, Germany

Bruce Morimoto, Ph.D., Vice President, Drug Development, Alto
Neuroscience, USA

Workshop Description:
This workshop will address different strategies for overcoming manufacturing
challenges of synthetic RNA therapeutics. It will also discuss methods fordefining CMC control strategies along the development pathway from earlyclinical development through later stages of development. A variety ofdifferent examples and case studies will be presented to give attendees agood understanding of the current bottlenecks, potential solutions and future
directions in oligonucleotide CMC and manufacturing.
Who should attend?
Anyone interested in development of oligonucleotide therapeutics; Anyone
interested in outsourcing the manufacturing of oligonucleotide therapeutics
to a CMO / CRO. This includes R&D Researchers, Manufacturing Personnel,
Quality Assurance, Project Management, Business Development and ScientificManagement.

9:00 Workshop Moderator’s Opening Remarks

9:15 Synthetic Peptide Purity Method Development Challenges –Illustrated Case Studies
The purity and impurities analysis of peptides is a complex task requiring
reliable methods when going into a clinical program, with each project
bringing its share of unknowns. For traditional APIs, a robust purity
method development strategy is required, whereas in the high-throughput
individualised medicines field, speed is of the essence to reach patients assoon as possible. This presentation will address those challenges throughrecent case studies.
Alaric Desmarchelier, Ph.D., Business Development Manager –Peptides, Almac Group, United Kingdom
10:00 Updates on Peptide and Protein Characterization Methodologies
LC/MS is a powerful method for the characterization of peptides, proteinsand drug conjugates; however other tools like MALS add complementary
information that provide a better understanding of therapeutic molecules.
In our lab we have been using these tools to get a more complete pictureon the structural characteristics of peptide, protein, and gene therapy drugcandidates and will discuss some of the unique challenges faced with each.
Michael McGinley, Director, Global Applications, Phenomenex, USA
10:45 Networking Refreshment Break
11:15 Solubility and Physical Stability Challenges in Peptide
Formulation Development
Solubility and physical stability challenges may be encountered during thedevelopment of injectable peptide and protein drugs. Most peptide andprotein injections as liquid formulations or reconstituted solids need to comply
with particulate matter specifications set by USP guidelines and be devoid of
insoluble aggregates to reduce or eliminate cardiovascular risk and undesired
immune responses. Case studies will be presented on how to overcome latent
physical instability of small and linear cyclic peptides in liquid formulations
during long-term storage.
Juerg F. Tschopp, Ph.D., Principal Scientist, Stratum Medical
Corporation

12:00 Bioanalytical Challenges, Best Practices and Case Studies
Speaker TBA

Workshop Description:
Peptides fall between small molecules and biologics in terms of molecularweight and size. Additionally, the complexity of peptide therapeutics isincreasing with unnatural amino acid substitutions and various covalentmodifications (lipidation, PEGylation). This results in analytical challenges!
Learn from the experts on strategies and best practices. Hear case studies
from companies working in this area.

12:45 Close of Workshop and Luncheon for Morning Workshop Attendees

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3

Tuesday, March 19, 2024
* MAIN CONFERENCE PLENARY KEYNOTE SESSION * 2:00PM-5:00PM

1:55 Chairperson’s Remarks
2:00 Developing Next Generation Oligonucleotide Therapeutics
Advances in drug-like properties of oligonucleotides and targeted delivery approaches in the last decades haveenabled the development of this class of molecules to modulate intracellular targets precisely. This has led to the
expansion of druggable target space and development of oligonucleotide therapeutics for previously untreateddiseases. This talk will give an overview of the opportunities and challenges that need to be addressed to develop
oligonucleotides into meaningful therapies beyond rare diseases.
Shalini Andersson, Ph.D., Vice President Oligonucleotide Discovery, AstraZeneca, Sweden

2:30 Chemical Engineering of Oligonucleotides: Applications for RNA Therapeutic
RNA therapeutics has become an established drug discovery and development platform with approved drugs
that employ various mechanisms of action. Chemistry has played a critical role in providing drug-like properties tooligonucleotides. Recently, we have employed chemical engineering, an approach of using established chemical
modifications at strategic positions in an oligonucleotide, modifying the accessibility of both 3’- and 5’-ends, andcreating transient shapes. These changes have improved the delivery, specificity, and potency of antisense. Thesedesigns are broadly applicable to various mechanisms of action of RNA therapeutics.
Sudhir Agrawal, D. Phil., President and Founder, Arnay Sciences, USA

3:00 Peptide and Protein Therapeutics Conjugated with Human N-Linked Glycans
N-glycans are useful modifier molecules to improve the in vivo half-lives of various bioactive peptides and proteins.
Our chemical glycosylation procedure, which exploits the beneficial characteristics of human N-glycans, enables
to more quickly develop glycopeptide/protein therapeutics that are superior to the original molecules in terms ofpharmacokinetic and physicochemical properties.
Yuji Nishiuchi, Ph.D., Director of Research and Development, GlyTech, Inc., Japan

3:30

Networking Refreshment Break

4:00 RNAi Therapeutics in Japan: Needs and Solutions-An Alnylam Perspective
Yutaka Okada, President and Representative Director, Alnylam Japan KK, Japan

4:30 The Endosomal Escape Vehicle Platform of Cyclic Cell-Penetrating Peptides Enhances the

Delivery of Oligonucleotides

To overcome current limitations of oligonucleotide therapeutic delivery, we have designed a family of proprietarycyclic cell-penetrating peptides that form the core of our Endosomal Escape Vehicle (EEV™) technology and covalently
conjugated it to oligonucleotides. Using preclinical models of Duchenne muscular dystrophy (DMD), we demonstrated
the ability of our EEV platform technology to efficiently deliver oligonucleotides to skeletal and cardiac muscle, theprimary sites of pathology in DMD.
Leo Qian, Ph.D., Co-Founder and Vice President, Discovery Research, Entrada Therapeutics, USA

5:00 Realizing the Potential of RNA-Targeted Oligonucleotide Therapeutic; Pipeline Progress andPartnerships

Brett Monia, Ph.D., Chief Executive Officer, Ionis Pharmaceuticals

5:30

Close of Day 1

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4

Wednesday, March 20, 2024
MAIN CONFERENCE SESSIONSPlenary Session
7:45 Registration and Coffee
8:10 Chairperson’s Remarks
8:15 Transforming the Care of Cardiovascular Disease Through Single-course Gene Editing Medicines
Andrew Bellinger, M.D., Ph.D., Chief Scientific Officer, Verve Therapeutics, USA

8:45 From Circles to Conjugates: Rethinking the Architecture of guide RNAHigh quality gRNA is needed for the effective application of CRISPR/Cas-based therapeutics. Several challenges face the development of therapeuticgRNA, including the need for more robust synthesis processes and higher potency (particularly, in vivo). Here I will describe two new gRNA architectures,
circular gRNA and peptide-gRNA conjugates, and show how each, in part, addresses these challenges. I will also discuss why we should reconsider themost common gRNA design and review alternatives.
Brian Cafferty, Ph.D., Director, Beam Therapeutics, USA

9:15 Improving Oligonucleotide Pharmacology Across Tissues and Modalities through Base, Sugar, and 			

Backbone Modifications

Wave’s PRISMTM platform enables synthesis of stereopure oligonucleotides with position-controlled chemistry and stereochemistry, facilitating
optimization for the target, tissue, and modality. We describe our progress in improving the pharmacological properties of oligonucleotides for RNA
interference (RNAi) and RNA editing in hepatic and extrahepatic tissues through base, sugar, and backbone modifications.
Michael Byrne, Ph.D., Vice President, In Vivo and CNS Biology, Wave Life Sciences, USA

9:45 Networking Refreshment Break with Poster and Exhibit Viewing

Track 1

Track 2

10:15 Chairperson’s Remarks

10:15 Chairperson’s Remarks

10:20 Bivalent Nucleic Acid Recognition and Its

10:20 Synthesizing Long Guide RNAs for In Vivo Prime

Applications in Targeting RNA Triplet-Repeats

This presentation highlights our ongoing efforts to develop a novel
set of bivalent (Janus-type) nucleic acid recognition codes for targeting
RNAs, primarily focusing on CUG-RNA repeats associated with
Myotonic Dystrophy type 1 (DM1). The underlying molecular platform
is quite versatile, as it can be tailored to bind with various RNA
repeats in a highly specific and selective manner. The newly crafted“ligands,” named as such due to their compact size—comprising only
three units in length—bear a closer resemblance to small molecules
than they are to traditional oligonucleotides. However, unlike thelatter, they engage their targets in a sequence-specific and strandselective fashion through bifacial H-bonding with the adjacentnucleobases in both strands of the RNA double helix. This work
substantiates the proof-of-concept that such diminutive nucleic acid“ligands” can indeed be developed for the recognition of CUGexp-RNAtranscripts, and potentially other members within this relatively large
class of neuromuscular and neurodegenerative disorders.

Editing

Prime Editing Guide RNAs (pegRNA and nickRNA) are key componentsof Prime Editing (PE) Systems that require innovative and robust
manufacturing processes and testing to generate guide RNAs suitablefor in-life applications of PE. Various chemistries and methods thatenable the generation of high-quality guide RNA from mg to gramscale supporting pre-clinical development of several in vivo programs
will be described.
Rowshon Alam, Ph.D., Senior Director, Head of Process
Chemistry, Prime Medicine, USA

Danith Ly, Ph.D., Professor of Chemistry, Carnegie Mellon
University, USA

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5

Wednesday, March 20, 2024
MAIN CONFERENCE SESSIONS

Track 1
10:50 Amide-Modified Oligonucleotides for Chemical

Control of Functional RNAsThis presentation will discuss synthesis, structure, and RNAi and
CRISPR activity and specificity of amide modified RNA. Amides are
excellent mimics of the phosphodiester linkages in RNA. Amide
modifications of siRNAs significantly reduced the off-target activityof guide and passenger strands. Amides did not interfere withCRISPR-Cas9 activity when placed in the protospacer adjacent motif
distal region of crRNAs. Our results suggest that amides have strong
potential to optimize biological and pharmacological properties ofsiRNAs and crRNAs for in vivo applications.
Eriks Rozners, Ph.D., Professor and Chair of Chemistry
Department, Binghamton University, USA

11:20 Chemical Approaches to Enhance siRNA Selectivity
Exogenous siRNAs do not always exhibit specificity, potentiallyresulting in unintended consequences on the expression of nontargeted genes, referred to as off-target effects. Among these
off-target effects, the most common is the miRNA-like effect, whicharises from either perfect or partial seed sequence complementarity
with non-targeted mRNAs. Here, we report that placing a bulky
alkyl phosphonate backbone in the seed region of the guide strand
improves specificity of siRNA.

Track 2
10:50 Addressing the Regulatory Expectations for Guide

RNA Quality While Providing Manufacturing
Capacity

Blake Unterreiner, Associate Vice President, Business
Development and Customer Relations, Agilent Technologies, USA

11:20 Delivery of Genetic Medicine with Hydrophilic

Nanoparticles

Conventional delivery technologies for genetic medicine facechallenges: off-target delivery, innate immune response, unable torepeat dose, or costly manufacturing. NanoGalaxy platform consistsof a diverse library of hydrophilic polymers and, through systematicand iterative screening, has been used to identify NPs with selective
delivery to the nervous system via intrathecal administration andto the innate immune system via intravenous administration. This
presentation will introduce NanoGalaxy platform and share thedelivery results of genetic medicine payloads.
Kunwoo Lee, Ph.D., Chief Executive Officer, GenEdit, USA

Mehran Nikan, Ph.D., Research Fellow, Ionis Pharmaceuticals,USA

11:50 Networking Luncheon with Poster and Exhibit Viewing

Track 1

Track 2

12:55 Chairperson’s Remarks

12:55 Chairperson’s Remarks

1:00 Comprehensive Analysis of Brain Distribution for

1:00 Acceleration of Process Development for GMP

Antisense Oligonucleotides Using Whole Tissue
Imaging Technique

A direct administration into the cerebrospinal fluid is conventionallyemployed in antisense oligonucleotides (ASOs) therapy for central
nervous system diseases. However, the intra-brain behavior of ASOs
after intrathecal / intracerebroventricular injection and have notbeen fully clarified. In this study, we aimed to figure out the brain
distribution behavior of ASOs in mice, rats, and marmosets using
whole-brain microscopic imaging with tissue clearing technique.
Syunsuke Yamamoto, Associate Director, Center of Excellence forDrug Metabolism, Pharmacokinetics and Modeling, Preclinicaland Translational Sciences Research, Takeda Pharmaceutical
Company, Japan

Production of Peptides

This presentation will discuss high-throughput process development
via microwave-assisted SPPS and optimizing the synthesis of ahydrophobic 19 amino acid peptide.
Dewey Sutton, Ph.D., Research and Development Supervisor,
AmbioPharm, USA

1:30 Recent Technological Innovation in Peptide

Manufacturing

Accelerating the development of peptides and oligonucleotides as
pharmaceuticals requires technological development that disrupts
conventional approaches and overcomes cost and quality challenges
in all manufacturing processes, including synthesis, purification,and lyophilization. We will introduce specific examples of newtechnologies that can solve these issues using our model peptides.
Yoshitaka Nemoto, General Manger R&D, PeptiStar, Japan

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6

Wednesday, March 20, 2024
MAIN CONFERENCE SESSIONS

Track 1
1:30 Synthetic Strategy for Ligand Conjugated

Oligonucleotides via Diamine Modified Stable
Phosphoroamidite

This presentation provides the methodologies for synthesis of ligand
conjugated oligonucleotides along with developing a novel diamine
modified stable phosphoroamidite, which has favorable physicalcharacteristics that can help to specify and regulate quality attributesof oligonucleotides.
Mitsuaki Sekiguchi, Ph.D., Principal Scientist, Biopharmaceutical
Research Division, Shionogi, & Co., Ltd., Japan

2:00 Nucleic Acid Therapeutics: Process Optimization and

Purification

Innovative cell-free DNA template production, and the usage of
Dynabeads for in vitro transcription and crude mRNA purification
could help optimize current mRNA workflow and possibly become thealternative method for the oligo synthesis.
Lulu Zhang, Ph.D., Field Application Scientist, Thermo Fisher
Scientific

2:30 Overcoming Oligonucleotide Manufacturing

Challenges

Track 2
2:00 De Novo Discovery of Natural Product-like

Thiopeptides with Designed Biological Activities

The talk will discuss how an in vitro reconstituted and reengineered
natural product biosynthesis pathway can be integrated into mRNA
display, a powerful in vitro selection technique. The resulting platform
enables de novo discovery of natural product-like macrocyclic
peptides active against proteins of interest. The high potency,
selectivity, proteolytic stability, and cell uptake of the discovered
compounds highlight how the established platform can accelerate
early drug discovery efforts.
Alexander Vinogradov, Ph.D., Project Assistant Professor,
Department of Chemistry, The University of Tokyo, Japan

2:30 Discovery of Macrocycles for Delivery of RNA andTargeted Radiopharmaceuticals

The talk will describe genetically encoded and DNA encode pipeline
used by 48Hour Discovery to discovery nonomolar and pioconolar
lead compounds that bind to extra cellular receptors; we will discussboth internal pipeline of 48Hour discovery and overview partnershipprojects and discuss advance of these assets though the preclinicalpipeline.
Ratmir Derda, Ph.D., Founder and Chief Scientific Officer, 48Hour
Discovery, CanadaLoïc Cornelissen, Ph.D., Sales Manager, PolyPeptide Group,
Belgium

3:00 Networking Refreshment Break with Poster and Exhibit Viewing

Track 1
3:30 Going Large-scale with Manufacturing of

Oligonucleotides

Track 2
3:30 Macrocyclic Peptide Drug Development by Combining

the Strengths of all Small Fragments, mRNA Display
and AI Technologies

The growing number of oligonucleotide-based APIs is accompanied
by an increasing need for efficient routes for their large-scale
manufacturing. It is therefore essential to develop more efficient,more sustainable, and highly scalable manufacturing techniques. The
speaker will give an overview of Bachem’s existing oligonucleotide
capacity based on traditional packed bed synthesizers from small-,mid-, pilot- to large-scale and according chromatography. Besides
scalability considerations and equipment comparisons, the talk will
also outline currently ongoing capacity expansion, where a new,additional large-scale line for metric ton oligonucleotide output iscommissioned.

Quantum Intelligence Corporate (QIC) provides hit compound
discovery and lead optimization for therapeutic peptides topharmaceutical and biotech companies. Our advanced technology(QUEST) is based upon quantum mechanical electrostatic potential
calculations and artificial intelligence such as neural networks
and deep-learning techniques performed on GPU-powered High
Performance Computing clusters. Our supernatural module predicts
the optimal unnatural pharmacophore substitution of a template
peptide that enhances the binding affinity to a target protein andincreases the lipophilicity of the lead.

Daniel Samson, Ph.D., Vice President, Head Oligonucleotides,
Bachem AG, Switzerland

Hwanho Choi, M.D., Ph.D., CEO and Founder, Quantum
Intelligence Corporation, South Korea

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7

Wednesday, March 20, 2024
MAIN CONFERENCE SESSIONS

Track 1

Track 2

4:00 Synthesis PAT: IR Spectroscopy for Real-Time

Phosphoramidite Identification

The use of innovative PAT approaches reduces manufacturing risk
and improves reliability. FTIR spectra collected by an in-line ATR
probe can accurately identify amidites during synthesis operations.
Software compares collection data against a spectral databank via
cosine similarity and performs amidite solution identification in realtime, with direct feedback to the synthesizer during priming setup andmanufacturing operations.
Spenser Pruett, Scientist II, Process Development, Nitto Avecia

4:30 Addressing Complex Oligonucleotide Therapeutics andApproach Towards Endotoxin Removal

Juergen Mueller, Ph.D., Vice President of Commercial Operations,
LGC Axolabs, Germany

4:00 From Discovery to the Clinic: Development of a Novel

Bioactive Peptide that Improves Mood and Cognition
by Engaging the Gut-Brain Axis

We have developed a peptide drug that improves neurologic
impairment by acting through a novel gut-brain circuit. DGX-001 is
a potent small peptide, orally administered with a mechanism thatinvolves engagement of unique sensory cells in the gut lumen andactivation of afferent vagal signals that reach the brain. In healthy
human volunteers, DGX-001 was well-tolerated and following shortterm dosing led to measurable increases in cognitive function andmood that tracked with EEG changes in the brain.
Kousaku Ohinata, Ph.D. Associate Professor, Kyoto University
and Founder and Scientific Advisor, Viage Therapeutics, Japan

4:30 A Purely Thermodynamic Anti-prionic Mode of Action
5:00 New Ligation Approach: Technology for High Quality

Manufacturing of Over 150 mer RNA

Masato Sanosaka, Ph.D., Group Leader of Research & Process
Development, Ajinomoto Biopharma Services

for Protein-misfolding Diseases is Realized by All-Dpeptides

Thermodynamic stabilization of aggregation-prone proteins, likeAβ and α-synuclein, is not only inhibiting their aggregation, but also
disassembling already existing aggregates into harmless monomers.
We achieved thermodynamic stabilization of the monomers by all-Dpeptides that are highly affin and specific for the protein species in its
monomeric conformation, which is intrinsically disordered. The all-Denantiomeric ligand for Aβ, RD2, demonstrated target engagement
ex vivo and disassembled Aβ oligomers obtained from brain tissue
of former AD patients. I will present the results of a clinical phase Ib,
double-bind, placebo-controlled study with mild cognitively impaired(MCI) and mild AD patients treated once daily orally with RD2 orplacebo for 4 weeks. A phase II study is scheduled.
Dieter Willbold, Ph.D., Director and Full Professor,
Forschungszentrum Jülich and Co-Founder, Priavoid GmbH,Germany

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8

Wednesday, March 20, 2024
MAIN CONFERENCE SESSIONS

Track 1

Track 2
5:00 In Cellulo Library-derived Peptide-based Inhibitors of

Alpha-synuclein Aggregation and Toxicity

A major group focus is the design and selection of peptides
that target amyloidogenic proteins involved in age-relateddiseases. Amyloid proteins are known to be important in a
number of such diseases that include Alzheimer’s, Parkinson’s,
Lewy Body Dementia, Huntington’s, and CJD. We use a novelin cellulo library-screening platform to select peptides that canbind amyloidogenic target proteins to sequester and detoxifythem. Utimising a Protein-fragment Complementation approach(PCA), we have identified both strand and helix-based peptide
antagonists of α-synuclein proteins implicated in PD and related
synucleinopathies. PCA is multiplexed, making no mechanistic
assumptions about the target oligomeric state or conformer
populated. Rather, library members must bind to and reduce
associated toxicity to become selected. Those that either
generate, or fail to prevent formation of a toxic species, resultin cell death or retarded cell growth rates relative to effective
binders. Library members that confer the most rapid bacterial
growth are then selected from the PCA by increased stringency
during further competition selection. Our antagonists havebeen characterised using a range of biophysical and cell-based
approaches and been downsized / refined using truncation,
alanine-scanning, and incorporation of structure-inducing
constraints and non-natural sequences. Our work in this area iscurrently funded by an Alzheimer’s Research UK Major ProjectAward.
Jody Mason, Ph.D., Professor of Biochemistry, University of Bath,United Kingdom

5:30 Networking Cocktail Reception with Posterand Exhibit Viewing
Please join your fellow attendees in the exhibit hall foran evening of networking while enjoying beverages
and appetizers.
SPONSORED BY:

6:30 Close of Day 2

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9Thursday, March 21, 2024
MAIN CONFERENCE SESSIONSPlenary Session
8:00 Registration and Coffee
8:10 Chairperson’s Remarks
8:15 Modulation of Host Immunity in the Airways with Interferon Lambda Encoding mRNA
Type III interferons play an important role in the innate antiviral, antifungal and antiprotozoal defences of mucosal barriers and enhance adaptive
immune responses in the respiratory mucosa. Based on its proprietary Stabilized Non-Immunogenic mRNA (SNIM®RNA) and lipidoid nanoparticle
delivery platforms, Ethris has developed interferon lambda encoding mRNA as a drug candidate for prophylactic and therapeutic administration to
the airways for prevention and treatment of viral infections. In the presentation, formulation of mRNA suitable for administration to the airways as anaerosol will be discussed. Furthermore, preclinical proof of concept data will be presented, demonstrating the potency of interferon lambda endcodingmRNA in mouse and ferret influenza A and SARS-CoV-2 virus challenge models in mice and ferrets when administered to the airways.
Christian Plank, Ph.D., Chief Technology Officer, Ethris GmbH, Germany

8:45 Strategies for mRNA Analytics
Andreas Czech, Ph.D., Associate Director RNA Analytics, BioNTech SE, Germany

9:15 Fine Tuning a PCR based mRNA Manufacturing Platform for each mRNA Sequence
The increasing demand for mRNA therapeutics requires a flexible technology platform and a cost-effective manufacturing process with well-defined andcharacterized product quality attributes. Different mRNA sequences and lengths can impact the impurity profiles and create additional challenges which
can impact the purification steps and challenges. We will discuss the approach we took to tackle these challenges and optimize/fine tune our process forsuch challenging mRNAs, by shedding light on a case study for a gene editor encoding mRNA.
Aditi Mehta, Ph.D., Associate Director, Head of mRNA Process & Delivery, Merck KGaA, Germany

9:45 Lipid Nanoparticles to Enable Clinical Development of mRNA-based Therapeutics
Acuitas’ is developing lipid nanoparticle (LNP) delivery systems to efficiently and safely deliver messenger RNA (mRNA). Through a combination ofindustry partnerships, academic collaborations and internal research, Acuitas is enabling mRNA-based medicines in a broad range of therapeutic areas.
This presentation will focus on recent preclinical and analytical results from this research and development work, as these mRNA LNP medicines are
rapidly translated into the clinic.
Ying Tam, Ph.D., Chief Scientific Officer, Acuitas Therapeutics, Canada

10:15 Networking Refreshment Break with Poster and Exhibit Viewing

Track 1
10:55 Chairperson’s Remarks

Track 2
10:55 Chairperson’s Remarks

11:00 Protein-based Nano-capsules for Delivery of

Therapeutic RNAs Across the Blood-Brain-Barrier

The presentation will describe the generation and the use of proteinbased nano-capsules to deliver therapeutic RNAs across the bloodbrain-barrier to treat CNS diseases. The therapeutic potential of thisdelivery technology will be illustrated for the mRNA treatment of
monogenetic CNS disorders such as metachromatic leukodystrophy(MLD), a lysosomal storage disease.
Ekkehard Leberer, Ph.D., Scientific Advisor, Neuway Pharma,
Germany

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10Thursday, March 21, 2024
MAIN CONFERENCE SESSIONS

Track 1
11:00 Preclinical Data for STK-002, an Antisense

Oligonucleotide Being Developed for the Treatment ofAutosomal Dominant Optic Atrophy (ADOA)

Using the TANGO (Targeted Augmentation of Nuclear Gene Output)
approach, we design ASOs that bind to pre-mRNA and help thetarget genes produce more protein. The initial application for thistechnology is haploinsufficient diseases in which one copy of a genefunctions normally and the other is mutated. ADOA is a rare genetic
disease characterized by severe and progressive visual decline due
to loss of retinal ganglion cells. Most patients harbor loss-of-function
mutations in the OPA1 gene that codes for OPA1 protein. Stoke isdeveloping STK-002 that reduces the inclusion of a non-productive
event in OPA1 gene resulting in increase in productive OPA1 mRNAand protein. The preclinical data supporting the clinical developmentof STK-002 will be presented here.
Shobha Ravipaty, Ph.D., Director, Stoke Therapeutics, USA

11:30 Discovery and Delivery of Oligonucleotide

Therapeutics

Jayaprakash Nair, Ph.D., Vice President, Research, Chemistry and
Delivery Science, Alnylam, USA

12:00 From Bench to Bedside: Development of a GalXC-Plus

siRNA, DCR-STAT3, for Immunotherapy in Refractory
Cancer Patients

Track 2
11:30 Redefining Non-viral Delivery for Novel Genomic

Medicines with Tissue-targeted Lipid Nanoparticle(ttLNP) Platform

We will present the In-depth characterization of tissue and cell
tropism of LNP-mRNA which led us to achieve desired PK/PD profileof therapeutic drug candidates for pulmonary diseases. Beyond
the targeted delivery to the tissue and cells of interest, we will alsopresent how the diverse lipid chemistry library allows us to selectright lipids to deliver the complex gene editing cargoes, including the
mixture of RNA and pDNA for the therapeutic meaningful level ofediting in vitro and in vivo.
Kate Zhang, Ph.D., Chief Scientific Officer, Hopewell Therapeutics,
USA

12:00 Tuning Lipid Nanoparticles for Specific Applications
Lipid Nanoparticles (LNP) are a well-established platform for deliveryof nucleic acids (NA) to hepatocytes and for vaccine applications.
However, many potential applications for diverse NA modalities
exist outside of these areas. LNP with altered biodistribution can beachieved by changing route of administration, and modulating lipid
composition accordingly. This presentation will describe the latest
advances for hepatocyte delivery, as well as specialized LNP designed
for extrahepatocyte use, including compositions targeting the hepatic
stellate cell, lung, muscle and CNS.
Ed Yaworski, Chief Technology Officer, Genevant Sciences Corp,
CanadaJennifer Lockridge, Ph.D., VP, Dicerna TRU Early Development,
Novo Nordisk, USA

12:30 Networking Luncheon with Poster and Exhibit Viewing

Track 1

Track 2

1:40 Chairperson’s Remarks

1:40 Chairperson’s Remarks

1:45 Examples of Antisense Oligonucleotides that Target

1:45 Gate2Brain Shuttles, Going Beyond the Transport of

the Immunosuppressive Tumor Microenvironment for
Treatment of Cancer

Secarna Pharmaceuticals is an antisense oligonucleotide (ASO)
discovery company that has a diversified pre-clinical in house pipeline
based on the locked nucleic acid (LNA) oligonucleotide platform
LNAplus™. Our lead programs are within the areas of oncology
and inflammatory / fibrotic diseases. We will present our immuneoncology strategy including data showing promising anti-tumor
activity of ASOs targeting the multifunctional target neuropilin 1(NRP1) in mouse tumor models.
Frank Jaschinski, Ph.D., Chief Scientific Officer, Secarna
Pharmaceuticals, Germany

Small Molecules

Gate2Brain is a biotech company focused on the development oftherapeutics that efficiently cross biological barriers such as the
blood-brain barrier using a radically innovative peptide-based
patented technology platform. The potential of Gate2Brain’s peptide
blood-brain barrier shuttles goes beyond the transport of small
molecules but also drug-loaded nanoparticles and even antibodies
can be delivered to the brain. These drug delivery systems could beconsidered a game-changer in the treatment of CNS diseases where
there is a drug candidate that needs a better transport.
Meritxell Teixidó, Ph.D., CEO and CSO, Gate2Brain, Spain

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11Thursday, March 21, 2024
MAIN CONFERENCE SESSIONS

Track 1

Track 2

2:15 CIVI 008: An Orally Active LNA Drug Against PCSK9

2:15 Control Strategy Set-Up for Efficient & Safe Large Scale

CiVi Biopharma is developing an LNA oligo drug against PCSK9, whichin early clinical studies has demonstrated robust, dose-dependent
reduction in atherogenic lipoproteins when dosed subcutaneously.
To provide a more convenient, patient friendly dosing format thecompany has developed an oral formulation of the drug, which isscheduled to soon commence clinical trials. The presentation will
discuss the oral PCSK 9 program and the broader implication of CiVi
Biopharma’s oral platform for the delivery of oligonucleotide drugs in
general.
Henrik Oerum, Ph.D., Founder and Chief Scientific Officer, CiVi
Biopharma Inc., Denmark

Peptide Manufacturing Process

Process and analytical development in peptide pharmaceuticalindustries are the key foundations to build an in-depth process
understanding allowing to set the best control strategy of peptide
manufacturing processes. Throughout the scale-up manufacturing
process, the robustness of the control strategy is consolidated anddemonstrated in process validation batches. This presentation
illustrates the journey to set an efficient, safe and robust commercial
manufacturing process through an in-depth investment in the control
strategy design and green chemistry application. Successful casestudies will be shared.
El Djouhar Rekai, Ph.D., Head of Business Unit Peptide Process
Development & Manufacturing, PolyPeptide Group, Belgium

2:45 Development of TTX-MC138, a First-in-Class miRNA-

10b-Targeted Therapeutic Against Metastatic Cancersof Multiple Primary Disease Origins

We have previously developed a therapeutic miRNA-10b inhibitor,
named TTX-MC138, which can elicit life-long disease remissions in
preclinical models of adenocarcinoma. To de-risk further clinicaldevelopment of TTX-MC138, we have embarked on a Phase 0 clinical
trial with Cu64-labeled TTX-MC138. The trial involves microdose
injection of the agent into stage IV cancer patients, followed bypositron emission tomography-magnetic resonance imaging(PET-MRI) to determine its pharmacokinetics and uptake in clinicalmetastases.
Zdravka Medarova, Ph.D., Founder and Chief Technology Officer,
TransCode Therapeutics, USA

2:45 In-line Monitoring Method Integrating Flow Chemistry

for Peptide/Nucleotide Synthesis

Utilization of continuous flow method for peptide/nucleotides not
only brings about faster and more efficient coupling/decoupling
cycles, but also leads to less waste and less use of solvents/starting
materials. However, as conventional method, in some cases, thefailure of peptide synthesis reactions (e.g. low yield, generation of
unwanted impurities) cannot be detected in a timely manner due tothe lack of effective and high-precision in-line monitoring. Herein we
present several case studies to demonstrate how different in-line
spectroscopic measurements can significantly improve the results aswell as describe the superiority and inferiority of these methods.
Pengyu Xu, Ph.D., President and Representative Director,
SynCrest Inc., Japan

3:15 Networking Refreshment Break with Poster and Exhibit Viewing

Track 1
3:45 Chimeric PN-containing Oligonucleotides Yield

Exon Skipping in Preclinical Models and Boys with
Duchenne Muscular Dystrophy

Chimeric PN-containing oligonucleotides demonstrate high levelsof muscle exposure, exon-skipping, and dystrophin restoration invarious preclinical DMD models. Preliminary clinical data provide
evidence that WVE-N531, being developed for patients with DMD
amenable to exon 53 skipping, is leading to substantial accumulationand exon-skipping in the muscle after three biweekly doses.
Elizabeth Wagner, Ph.D., Director, Biology, Wave Life Sciences,
USA

Track 2
3:45 Flow SPPS – Towards Greener and More Efficient

Peptide Manufacturing

Traditional peptide synthesis relies on an orthogonal protecting
group strategy, employing excess coupling reagents and generating
significant waste. To enhance sustainability, our collaboration
with PeptiSystems explores the potential of flow-through column
technology. Initial experiments show promising results, reducing
amino acid and coupling agent consumption, while maintaining
comparable crude purities. In addition, coupling times weredecreased, and the PMI significantly improved using high loaded
resins without compromising the overall process. This initiative aligns
with Corden Pharma’s commitment to greener and more efficientmanufacturing.
Eike-Fabian Sachs, Ph.D., Head of Development Frankfurt,
CordenPharma International GmbH, Germany

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12Thursday, March 21, 2024
MAIN CONFERENCE SESSIONS

Track 1

Track 2

4:15 Key Elements of a Well Composed Nonclinical Section

4:15 Biocatalysis for Green Manufacturing of Amino Acidsof an IND Application

New oligonucleotide therapeutics under investigation offer uniquechallenges relative to other pharmaceuticals. During preparation andsubmission of an investigational new drug (IND) application there are
a number of regulatory gray areas due to the large heterogeneity inthe type of oligonucleotide therapeutics and our knowledge of any
particular subclass. Based on lessons learned, this talk will describe
selected general considerations to a successful non-clinical IND
submission.
Emily Place, Ph.D., Senior Consultant Nonclinical, Biologics
Consulting, USA

Aralez Bio uses biocatalysis to unlock noncanonical amino acids.
Using directed evolution we develop enzymes to synthesize newamino acids needed to drive innovation in drug discovery andmanufacturing. Our process is 10x cheaper, 10x faster, and 50x
greener than conventional approaches, while simultaneously
expanding amino acid chemical space 100x. Noncanonical aminoacids are one of the fastest growing areas in peptide development,
ushering in an era of therapeutics built on unique, low-cost, green
building blocks.
Christina Boville, Ph.D., Co-founder and CEO, Aralez Bio, USA

4:45 Individualised Cancer Vaccines: Developing a High-throughput cGMP Manufacturing Philosophy

4:45 GalAhead™: A Proprietary GalNAc-RNAi Therapeutic

Platform to Downregulate Single and Multiple Genes

Sirnaomics has developed the proprietary GalNAc-RNAi therapeutic
delivery platform, GalAhead™, comprising two key technologicalcomponents – mxRNA™ (miniaturized RNAi triggers) and muRNA™
(multi-unit RNAi triggers). mxRNAs are composed of single ~30nt
oligonucleotides to downregulate individual genes, while muRNAs
are comprised of 2 or more oligonucleotides to simultaneously
silence two or more targets. We will present data validating thesetechnologies, as well as developments with quickly expanding andprogressing GalAhead™ therapeutic pipeline.

Personalised neoantigen cancer vaccines are a revolutionary
technology relying on an array of original molecules tailored to eachpatient’s tumour profile. Creating dozens of unique peptides for eachpatient, with tight turnaround times, required a paradigm shift to abespoke manufacturing mindset, facilities, and quality culture. We’ll
present how Almac successfully led the charge in this challenging fieldwith recent case studies (Pharmaceutics 2022, 14(7), 151), and discuss
the future of this approach in other applications.
Alaric Desmarchelier, Ph.D., Business Development Manager –Peptides, Almac Group, United Kingdom

Jim Weterings, Ph.D. Senior Director, Head of Technology
Development, Sirnaomics, USA

5:15 Close of Conference

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13

Tides Hosts The Highest Concentration Of Senior
Decision Makers In The Industry - Don’t Miss This
Opportunity To Reach Your Ideal Audience
Connect face-to-facewith highly influential scientists, engineers and executives from industry
and academia with the budget and authority to recommend, specify
and approve the purchase of products and services to accelerateoligonucleotide and peptide research, discovery efforts and clinical andmanufacturing programs. Contact us today to learn about the costeffective ways to drive lead generation and network with key buyers,
including:• Exhibit Booth Packages• Luncheon Briefing• Scientific Briefings• Executive Breakfast• Custom Emails• Private Meeting Space• Branded items – lanyards,delegate tote bag, pads &pens, hotel key cards, etc.

A wide variety of options are available tohelp you meet your company objectives
before, during and after the conference.
We provide flexible and tailored solutions to
optimize your investment.
To get involved and begin building your custom
sponsorship package today, contact:
Jennifer Wickett at
jennifer.wickett@informa.com

PLATINUM SPONSORSGOLD SPONSORSSILVER SPONSORSBRONZE SPONSOR

EXHIBITORS
Almac Groupemp Biotech

Nippon Shokubai

Asahi Kasei Bioprocess

Hefei KS-V Peptide

Orilife

Asymchem Laboratories

Hongene Biotech Corp

W.L. Gore

Aurisco Pharmaceutical Co

Kactus Bio

RiboBio

Biosynth

Kinovate Life Sciences

Syncrest

ChemGenes

KnauerSynthgene Biotechnology (Jiangsu)

Chinese Peptide Co

LGC Biosearch Technologies

Wuhu Huaren Science & Technology

EUROAPI

Neuland Labs

YamasaFor more information on how you can connect withkey buyers at TIDES 2024, contact:(Companies B,C,D) Jennifer Wickett | Jennifer.Wickett@informa.com(Companies A,E-Z) Michael Moriarty | Michael.Moriarty@Informa.com

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14