BREAKFAST 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. For in-person events, the facilitator will lead from the front of the room while attendees remain seated. For virtual attendees, the format will be in an online networking platform. 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.

March 16, 8:00-8:45 am eastern time

Oligonucleotide Discovery and Delivery
DISCUSSION TOPIC: Innovations In Oligo Design & Delivery
Moderators:
Isabel Aznarez, PhD, VP, Discovery Research, Stoke Therapeutics
Thazha Prakash, PhD, Executive Research Fellow, Ionis Pharmaceuticals, Inc.
Arthur M. Krieg, Founder & CSO, Checkmate Pharmaceuticals

• What are the prospects for targeting of oligos to extrahepatic tissues? Will TFR targeting and pancreatic beta cells targeting using GLP1R peptide ligand will be broadly translatable to humans?
• Are there other oligosaccharide ligands that might give us highly specific delivery to organs/tissues that are currently not targetable?
• How important is the number of nanoparticles for viral/non-viral delivery vectors for extrahepatic delivery?
• Will artificial intelligence and computer modelling play a major role in the design of cell type specific targeted oligonucleotides in the near future?
• What are the prospects and challenges for designing ASO for allele selectivity?
• What physicochemical characteristics of oligos can be modulated for improving delivery and activity?

Oligonucleotide CMC and Regulatory Strategies
DISCUSSION TOPIC: CMC & Regulatory Considerations for Oligo Drug Development
Moderators:
Marc Lemaitre, PhD, Oligonucleotide Therapeutics CMC/Strategy Consultant, ML_Consult LLC
Jennifer Franklin, PhD, Executive Director, CMC Regulatory Affairs, Ionis Pharmaceuticals, Inc.
Mike Webb, PhD, Independent Consultant, MikeWebbPharma Ltd.; Former Vice President, API Chemistry & Analysis, GSK

• Oligonucleotide drug substance specifications 
• Starting material for Oligonucleotides
• Identity testing of oligonucleotides and impurity limits