Thursday, March 14: 2:30 – 3:10 PM

Breakout discussions provide an opportunity to discuss a focused topic with peers from around the world in an open, collegial setting. Select from the list of topics available and join the moderated discussion to share ideas, gain insights, establish collaborations or commiserate about persistent challenges.  These will take place IN-PERSON ONLY.

Oligonucleotide Discovery & Delivery

TABLE 1: Successful Oligonucleotides for Extrahepatic Tissues and Strategic Collaboration between Academia and Industry
Moderator: Ken Yamada, PhD, Assistant Professor, RNA Therapeutics Institute, University of Massachusetts Medical School

  • What are the current chemistry demands and promising strategies towards successful therapeutic oligonucleotides for extrahepatic tissues?
    • Oligonucleotide backbone modification
    • Delivery strategies
    • Strategies to modulate cell-type specificity
    • Strategies to facilitate endosomal escape
  • How can we further amplify the benefits of collaboration between academic labs and biotechs?
    • Ideal research partnerships between academic labs and biotechs that synergistically facilitate oligonucleotide drug development
    • Future path for n=1 therapeutics

TABLE 2: From Early Discovery and the Clinic to Collaborations Between Big Pharma and Small Biotechs
Moderator: Marvin Caruthers, PhD, Distinguished Professor, University of Colorado

  • Developments from Early Discovery Through to Late-Stage Clinical Programs
    • How do you know your discovery should move toward the clinic
    • Patents
    • Steps toward the clinic-biology/biochemistry/chemistry, cell biology, animal studies, toxicology, and who/how to carry out a clinical trial
  • Strategic Collaboration Between Big Pharma and Small Biotechs
    • How do you define a strategic collaboration-from the perspective of big pharma and small biotech (i.e. why a collaboration)
    • Conditions for a strategic/successful collaboration
    • Oligonucleotide CMC and Regulatory Strategies​

Oligonucleotide CMC & Regulatory Strategies

TABLE 3: Analytical Challenges in Controlling Purity in Double-Stranded Oligonucleotide Drug Substances and Drug Products
Moderator: Mike Webb, PhD, Founder and CEO, Mike Webb Pharma; Former Vice President, API Chemistry & Analysis, GSK

  • Identifying and controlling impurities in complex UPLC separations
  • Orthogonal methods and detection
  • The application and use of assays and purity determination of drug substances
  • Non-denaturing methods and purity

TABLE 4: In Your Experience, to What Extent is RNA a Multi-Product Platform Technology? 
Moderator: Zoltán Kis, PhD, Assistant Professor, Chemical and Biological Engineering, The University of Sheffield

  • Do you see substantial differences in yield when using different template DNA sequences?
  • Do you see substantial differences in dsRNA when using different template DNA sequences?
  • Do you have purification processes that work as a platform, for different RNA sequences?
  • Does your formulation/encapsulation process work as a platform, for different RNA sequences?
  • Are there any best practices that you are willing to share?​

mRNA Design & Delivery

TABLE 5: Designing and Optimizing mRNA Therapeutics
Vikram Agarwal, PhD, Head of mRNA Platform Design Data Science, mRNA Center of Excellence, Sanofi
Wendy Gilbert, PhD, Professor, Molecular Biophysics & Biochemistry, Yale University
Yujian Frank Zhang, PhD, Independent Consultant

  • Innovative mRNA modifications
  • Emerging mRNA modalities (circular, replicating etc.)
  • Tackling mRNA safety
  • Strategies for translation control and mRNA stability
  • Tissue specificity design strategy
  • What properties are most important to optimize? Are there tradeoffs?
  • How does one define if the desired properties have already been sufficiently optimized?

TABLE 6: Tackling Challenges with mRNA Delivery
Iris Grossman, PhD, Chief Therapeutics Officer, R&D, Eleven Therapeutics US, Inc.
Dan Peer, PhD, Professor & Chair, Cancer Biology Research Center, Laboratory of Precision Nanomedicine, Tel Aviv University

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

Applications of mRNA Therapeutics

TABLE 7: Lipids and LNPs as Signalling Molecules in the Immune System
Moderator: Robert W. Georgantas, PhD, President, CTO, Providence Therapeutics

  • There is a significant literature on lipid signalling and metabolism in immune cell activation
  • Can different lipids/LNPs be exploited to affect the immune response?
  • Strategy of immune activation for different therapeutic modalities
  • How would one screen for the desired effects on the immune system?

Present a Poster

2024 Conference Programs

Oligo Discovery & Delivery