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Session 6 Track 2: Off Target Safety Assessment
Session Chair(s)
Patrik Andersson, PhD
Senior Director, RNA Therapeutics Safety
AstraZeneca R&D, Sweden
James Wild, PhD
Pharmacologist, CDER
FDA, United States
Hybridization-dependent off-target effects are a potential safety concern for both oligonucleotide and gene editing therapeutics. This session will start with updated recommendations from Industry on identification, verification and risk assessment of off-target sites for oligonucleotides. This will be followed by a presentation with concrete examples illustrating how the recommendations could be used. The final presentation will focus on assessment of off-target editing for in vivo gene editing approaches.
Learning Objective : At the conclusion of this session, participants should be able to:- Describe the different steps in the recommended approach to identify and evaluate potential off-target effects
- Compare and contrast off-target assessments for oligonucleotides and gene editing applications
- Discuss specific considerations for different oligonucleotide and gene editing applications, classes, and delivery systems
Speaker(s)
Assessing hybridization Dependent Off-Targets for Oligo Therapeutics – Updated Industry Recommendations
Patrik Andersson, PhD
AstraZeneca R&D, Sweden
Senior Director, RNA Therapeutics Safety
Discerning the Off-target Effects of RNase H-Dependent Antisense Oligonucleotides by Sequence Analysis and Transcriptomics
Representative Invited
Contera Pharma, Denmark
Assessing the Potential for Off-target Editing with in Vivo Liver-directed Base Editing Therapies
Representative Invited
Verve Therapeutics, United States
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