Breakout Roundtable Discussions

8:00 am Breakout Roundtable Discussions

Join us for a moderated roundtable discussion with interactive problem solving. These sessions bring together attendees from diverse backgrounds to exchange ideas and develop future collaborations around a focused topic, in an informal environment.

TABLE: Vaccine Development and Challenges for COVID-19

David Tabatadze, PhD, President, ZATA Pharmaceuticals

TABLE: Oligonucleotide Drug Discovery: From the Drawing Board to the Clinic

Moderator: Troels Koch, PhD, CTO, SVP Science & Technology, IneXos Therapeutics

Drug discovery is an exceptionally complex undertaking. What is of paramount importance is that the initial steps in the process are conducted as well-informed and qualified as possible. We will discuss drug discovery fundamentals and try to identify how an “ideal” program would look like. Discussion points:

• Delivery - target – modality triangulation: Where to begin, any preference?
• In vitro and in vivo assays: Toxicity and potency validation – Establish the right controls and secure high degree of translation
• How to make the best selection of target and indication to favor oligonucleotide therapeutics in relation to competing modalities?
• Advancing in silico discovery: Going beyond sequence based bioinformatics with structure based quantum mechanical modelling?

TABLE: Recent Advances in the Following Areas: The CNS, The Liver, Inhalation Drugs, Immuno- Oncology and Ocular Diseases

Moderator: Patrick Lu, PhD, President & CEO, Sirnaomics 

• What kind of siRNA deliveries can allow us move beyond GalNAc?
• What delivery modality is most suitable for lung siRNA therapeutics?
• What delivery modality is most suitable for CNS siRNA therapeutics?
• How can we enhance immuno-oncology efficacy using siRNA therapeutics?
• Is naked siRNA good enough for CNS therapeutics

TABLE: Building a Robust, Targeted-Centered Development Program

Moderator: David Corey, PhD, Professor, Department of Pharmacology, UT Southwestern

• What are criteria for a disease that is an appropriate target for ASO or dsRNA drug discovery and development
• What controls are necessary? 
• How can you tell a program that is built on a firm foundation from one that is not?
• What data does one look for when evaluating a program?
• What are red flags that might indicate that a company (or academic lab) is not built on a firm foundation of science

TABLE: Examining the Safety and Toxicity of Nucleic Acid Therapeutics

Moderator: Steven Kates, PhD, VP, Regulatory Affairs, Dicerna Pharmaceuticals

• What tox studies are needed and when?
• How to assess your impurities (analytical methods)
• Discussing mutagenicity
• Examining ADA assays
  

TABLE: Adoption and Implementation of Innovative Technologies Used in CMC And Manufacturing

Moderator: Roumen Radinov, PhD, Director Process Chemistry, Alnylam Pharmaceuticals

• The solid phase oligonucleotide synthesis based on sequential coupling of phosphoramidite monomers is a well-established industrial manufacturing process
• Currently performed routinely on kilo scale mainly due to limitations of the current synthesis and purification processes
• Novel approaches will need to be designed and developed to support high-volume oligonucleotide drugs in the near future
• Participants are invited to share their insights and experience in the development of alternative synthesis and purification technologies with potential for the cost-effective ton scale manufacture of oligonucleotides

TABLE: Identifying the Best Analytical Methods for Characterizing Impurities

Vidhya Gopalakrishnan, PhD, Senior Vice President, Pharmaceutical Development, Quark Pharmaceuticals