Breakfast Roundtable Discussions

TABLE 1: Adoption and Implementation of Innovative Technologies Used in Discovery and Delivery

Marvin Caruthers, PhD, Distinguished Professor, University of Colorado


TABLE 2: Considerations When Developing Splice Modulating Therapies

Moderator: Annemieke Aartsma-Rus, PhD, Professor of Translational Genetics, Leiden University Medical Center

  • Developing splice modulating therapies for small patient numbers
  • Selecting model systems and controls for pre-clinical exon skipping tests
  • Deciding when to move from pre-clinical to clinical studies
  • Involve patients in therapy development and trial design


TABLE 3: 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 4: 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 5: 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 6: CMC Regulatory Hurdles in Rare Disease

Moderator: Kimberly Tyndall, Principle, CMC Tyndall Consultant

  • How to successfully proceed
  • Never a marketed product N=1
  • Overcoming CMC hurdles
  • Why

 

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