Federal Register #FDA-2026-D-1255

FDA Draft Guidance: Safety Assessment of Genome Editing in Human Gene Therapy Products Using NGS

This notice announces the FDA's release of a draft guidance on safety assessment for genome editing in human gene therapy products using next-generation sequencing (NGS): - Government Buyer: - Food and Drug Administration (FDA), Department of Health and Human Services - Purpose and Scope: - Provides recommendations for using NGS-based methods in nonclinical studies to support clinical trials of investigational genome editing (GE) products - Focuses on safety assessments, including risks of off-target editing and genome integrity - Audience: - Intended for sponsors developing human gene therapy products involving genome editing technologies - Procurement Details: - No products or services are being requested or procured - No OEMs or vendors are named - Unique Requirements: - Seeks public comment to inform the final guidance - Not a solicitation or contract opportunity - Location: - FDA headquarters in Rockville, MD is the point of contact for comments and inquiries

Description

The Food and Drug Administration (FDA) has released a draft guidance document providing recommendations for next-generation sequencing (NGS)-based methods used in nonclinical studies to support clinical trials of investigational human genome editing products. This guidance addresses safety assessments focusing on off-target editing and genome integrity risks associated with gene editing technologies. The draft aims to assist sponsors in designing studies for Investigational New Drug applications and Biologics License Applications. Comments on the draft are due by July 14, 2026.

View original listing