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DIA/FDA Oligonucleotide-Based Therapeutics Conference

DIA Your Way! Join us live in-person or live from the comfort of your home/office. This event offers two learning avenues depending on your preference!

Session 4 Track 2: Off-Target Effects

Session Chair(s)

Sebastien  Burel, PhD

Sebastien Burel, PhD

  • Executive Director
  • Ionis pharmaceuticals, United States
James  Wild, PhD

James Wild, PhD

  • Pharmacologist, CDER
  • FDA, United States
The sequence-specific interaction of single- and double-stranded oligonucleotides with a variable range of off-target sites on pre-mRNA, mRNA and miRNA can result in hybridization-dependent toxicity. Similar off-target concerns, and the risk of introducing nuclease-induced off-target mutations exist for guide RNA in CRISPR-Cas genome-editing nucleases. This session will examine in silico and in vitro methods and strategies for identifying, assessing, and de-risking off-target hybridization for oligonucleotides and CRISPR genome editors. In addition, novel off-target assessment of steric-blocking and splice modulating oligonucleotides will be presented and discussed.
Learning Objective : At the conclusion of this session, participants should be able to:
  • Recognize and differentiate the scope of mechanisms contributing to off-target effects for oligonucleotides and CRISPR genome editors
  • Compare and contrast the application of multiple in silico and in vitro methods for assessing off-target effects
  • Formulate strategies for analysis of off-target hybridization for different classes of oligonucleotides


Shengdar Q. Tsai, PhD

Off-target Effects of CRISPR

Shengdar Q. Tsai, PhD

  • Assistant Member, St. Jude Faculty, Department of Hematology
  • St. Jude Children's Research Hospital, United States
Joanna  Harding, MSc

De-risking of Off-Target Effects

Joanna Harding, MSc

  • Director, Toxicology Project Lead, CVRM Safety
  • AstraZeneca, United Kingdom
Erle  Holgersen

Off-target Assessment for Splice Modulators

Erle Holgersen

  • Research Scientist
  • Deep Genomics, Canada