대면식 브레이크아웃 디스커션

THURSDAY MARCH 19, 2026: 9:35 am - 10:20 am  

Breakout 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 this format, please come prepared to share examples from your work, be a part of a collective, problem-solving session, and participate in active idea sharing. These will take place IN-PERSON ONLY.

Oligonucleotide Discovery & Delivery

TABLE 1: Progress from Early Discovery to Late-stage Clinical Development
Moderator
Isabel Aznarez, PhD, Vice President, Discovery Research, Stoke Therapeutics

TABLE 2: Industry Perspectives on Platform Development and Pipeline Evolution
Moderator: 
Michael Byrne, PhD, VP Research Pharmacology, Research Pharmacology, WAVE Life Sciences

  • Developing new modalities with platform technologies
  • Balancing new biology with technology advances
  • Choosing delivery platforms
  • Portfolio development and filling the therapeutic pipeline

Oligonucleotide CMC & Manufacturing

TABLE 3: New NMPA Oligonucleotide Guideline: Considerations for Oligonucleotide Development and Regulatory Submissions
Moderator:
Benjamin Stevens, PhD, Director, CMC Policy and Advocacy, GSK

  • Comparison with existing EMA guideline and established practices by key global regulators such as FDA, PMDA
  • Considerations related to comparability and stereochemistry
  • Increasing focus on bioassays to support characterization and CMC changes
  • Potential impact to product lifecycle management
  • Treatment of enzymatic ligation and starting material designation

TABLE 4: Strategies for Outsourcing Manufacturing
Moderator: 
Khaled Yamout, Analytical Sciences, Quality and Manufacturing Consultant, Y-Chem Consulting LLC

  • What is the roadmap to your product development
  • Can the site support your Phase appropriate needs from per clinical and on.
  • What are the onsite capabilities? That is, systems, equipment, scale up and analytical support.
  • Is the process transferable and are there any IP limitation to the process
  • Level of support for analytical characterization/testing and CMC

mRNA & Emerging Oligonucleotide Modalities

TABLE 5: Design and Optimization of mRNA Drugs
Moderators:
Jaspreet Khurana, PhD, Senior Director, mRNA Programming, Strand Therapeutics, Inc.
Iris Grossman, PhD, Chief Therapeutics Officer, R&D, Eleven Therapeutics US, Inc.
Sizhen Li, PhD, Computational Scientist Lead, Digital R&D, Sanofi
Alex Zinoviev, PhD, Director of mRNA Platform, Gene Therapy, Eli Lilly & Co.

  • Defining what properties are most important to optimize. Are there trade-offs?
  • Optimizing innovative chemistries and modifications
  • Strategies for regulating mRNA expression
  • Addressing biodistribution, tissue specificity and safety
  • Utilizing AI/ML to predict mRNA design


TABLE 6: Tackling Challenges with mRNA Delivery
Moderators:
Ekkehard Leberer, PhD, Professor of Biochemistry, Technical University of Munich; Senior Consultant, ELBIOCON; Advisor, Neuway Pharma
Dan Peer, PhD, Professor & Director, Laboratory of Precision Nanomedicine, Vice President for Research, Tel Aviv University

  • Innovative mRNA formulations and delivery approaches
  • Emerging delivery vehicles, linkers and payloads for stability and targeted delivery
  • Improving selectivity and efficiency of delivery
  • Delivery across the blood-brain-barrier
  • Targeting specific cell types for treating various diseases
  • Low immunogenicity mRNA formulations

TABLE 7: Emerging Oligo Modalities for Diverse Therapeutic Applications
Moderators:
Namita Bisaria, Head, Research Strategy and Operations, AIRNA
Sushma Gurumurthy, PhD, Former Senior Director, Oncology Research, Moderna, Inc.
Edo Kon, PhD, Director of Business Development, RiboX Therapeutics

Arthur Krieg, MD, Founder, President and Acting CEO/CSO, Zola Therapeutics

  • Innovative circular RNAs and their applications
  • Optimizing tRNA design and delivery
  • Case studies in cancer, autoimmune, cardiovascular, genetic disorders
  • Establishing criteria for selection, application and success

Peptides & Emerging Drug Conjugates

TABLE 8: Use of AI-ML to Accelerate Drug Development
Moderators:
Ruben Abagyan, PhD, Professor, Skaggs School of Pharmacy and Pharmaceutical Sciences, University of California, San Diego
Alan Nafiiev, PhD, CEO & Founder, Receptor.AI

  • Effective use of generative models and machine learning to design new functional molecules
  • AI-driven prediction models and optimization strategies to enhance affinity and permeability of peptide drugs and conjugates
  • Need for benchmarks and optimized structure prediction algorithms

TABLE 9: Advances in Design and Delivery Using Novel Linkers, Conjugates and Payloads
Moderators:
Terry Moore, PhD, Associate Professor, Pharmaceutical Sciences, University of Illinois Chicago
Michael Weickert, PhD, CEO, Pacylex Pharmaceuticals
Sunny Zhou, PhD, Professor, Chemistry & Chemical Biology, Northeastern University

  • Defining what properties are most important to optimize 
  • What are the most practical and useful modifications in terms of overcoming resistance and enhancing durability of response
  • Testing efficacy, stability and safety of linkers and payloads
  • Finding ways to expand the structural and chemical diversity of peptides and conjugates

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Oligonucleotide Discovery & Delivery

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