FDA Issues Draft Guidance on NGS Methods for Genome Editing Products

The Food and Drug Administration issued a draft guidance on Tuesday outlining recommendations for the use of next-generation sequencing (NGS) methods in nonclinical studies supporting investigational human genome editing (GE) products.

The guidance provides recommendations for sequencing strategies, analysis and reporting to evaluate risks such as off-target editing and loss of genome integrity. It is intended to support nonclinical studies submitted with Investigational New Drug applications and Biologics License Applications and applies to both ex vivo and in vivo genome editing approaches. The recommendations build on the agency’s January 2024 guidance on genome editing therapies.

The draft guidance is open for public comment through July 14. Submission instructions are available in the Federal Register.