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DIA/FDA Oligonucleotide-Based Therapeutics Conference


Session 8 Track A & B: Hybridization-Dependent Off-Target Events

Session Chair(s)

Arthur A. Levin, PhD

Arthur A. Levin, PhD

  • Executive Vice President, Research and Development
  • Avidity Biosciences , United States
Imran  Khan, PhD

Imran Khan, PhD

  • Pharmacologist, OMPT, OND, ODEI, DPP, CDER
  • FDA, United States
In 2012, the Oligonucleotide Safety Working Group published guidelines that outline approaches for assessing hybridization-dependent off-target events. Significant progress has been made in the intervening years in our understanding of this phenomena and new technologies have been more widely accepted that allow us to assess off-target events more thoroughly. Also, in the intervening years, there have been increasing reports of hybridization dependent toxicities being identified in non-clinical studies. It is now time to re-interrogate how to best study off-target events and define new guidelines. The attendees at this session should come away with a greater understanding of the nature and mechanisms of off-target effects and how bioinformatics approaches can minimize them and how to assess them.

Speaker(s)

Arthur A. Levin, PhD

Update from OSWG-Oligo Hybridization-Dependent Off-Target Events and Surveillance Across Platforms/Mechanism of Action

Arthur A. Levin, PhD

  • Executive Vice President, Research and Development
  • Avidity Biosciences , United States
Sagar  Damle, PhD

Large-scale Selectivity Assessment of Gapmer Antisense Oligonucleotides

Sagar Damle, PhD

  • Associate Director, Functional Genomics
  • Ionis Pharmaceuticals, Inc., United States
Mark K. Schlegel, PhD

Preclinical and Clinical PK of GalNAc SSOs at Doses Below and Above ASGPR Saturation Approaches to siRNA “Off-Target” Assessment

Mark K. Schlegel, PhD

  • Principal Scientist, RNAi Discovery
  • Alnylam Pharmaceuticals, Inc. , United States
Eric F. Donaldson, PhD

Concerns with Antisense and siRNA Antiviral Drugs from a Clinical Virology Perspective

Eric F. Donaldson, PhD

  • Virology Reviewer, CDER/OND/OAP/Division of Antiviral Products
  • FDA, United States