対面式のブレイクアウトディスカッション

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: Interfacing New Research with the Biotech Arena
Moderator: 
Marvin Caruthers, PhD, Distinguished Professor, University of Colorado

• Strategic collaboration models between pharma, biotech, and academia
• New academic modalities
• Models for technology transfer university to pharma
• Your interface with the university and pharma
• Exploring RNA as a potential therapeutic
• Criteria for developing new oligonucleotide therapeutics

TABLE 2: Industry Perspectives on Platform Development and Pipeline Evolution
Moderator: 
Chandra Vargeese, PhD, CTO & Head, Platform Discovery Sciences, 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
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