Genome editing is a tool that enables a new generation of medicines by directly targeting with high precision the genetic cause of a disease. It also allows for immune evasion and prevents microenvironment responses: concepts vital to more potent, off the shelf, cell, and gene therapies. To ensure safe and efficacious administration of gene edited products to patients, minimizing unintended effects is paramount. This session will review approaches to characterize off target genotoxicity at different stages of development for different class of genome/epigenome editing medicines. Current regulatory standards will be discussed, and lessons learned may be applied to relieve bottlenecks for future therapies.

Session Speakers:

Overview of Current Genomic Analytical Tools to Enable Advancement of Investigational in vivo Genome Editing Products into Clinical Studies

Jessica Seitzer, Intellia Therapeutics, Inc.

Analytical Control Strategies for Ensuring Genomic Integrity in CAR-T Cell Therapies

Athea Vichas, Bristol-Myers Squibb Company

Off-Target Analysis of Genome Editing Products

Yongwook Choi, CBER, FDA

Additional Panelist:

Jennifer Dashnau, Century Therapeutics, Inc.

Isabella Palazzolo

Appearances