Original Agenda
We are actively working with our speakers to confirm their availability for our new dates. Initial response from our speakers has been very positive, and we are optimistic we will have the new programs ready to share here soon.

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


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Oligonucleotide Discovery and Delivery