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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, MS
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 presentations 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
Peter Hagedorn, PhD, MSc
Contera Pharma, Denmark
Scientific Director, Head of Bioinformatics
Assessing the Potential for Off-target Editing with in Vivo Liver-directed Base Editing Therapies
Joseph Bidenkapp, PhD
Verve Therapeutics, United States
Vice President, Editing Development
Overview of Current Genomic Analytical Tools to Enable Advancement of Investigational In Vivo Genome Editing Products into Clinical Studies
Jessica Lynn Seitzer
Intellia Therapuetics, United States
Vice President, Head of Genomic Operations
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