Draft Guidance on Safety Assessment of Genome Editing Using Next-Generation Sequencing

Tue, 14 Apr 2026 15:14:04 +0000
Pharmaceuticals

The FDA's Center for Biologics Evaluation and Research issued draft guidance recommending next-generation sequencing methods for assessing off-target editing risks and genome integrity in human gene therapy products using genome editing technologies. The guidance applies to both ex vivo and in vivo products and provides recommendations on sequencing strategies, sample selection, analysis parameters, and reporting. Sponsors submitting investigational new drug applications or Biologics License Applications for genome editing therapies should incorporate these safety assessment methods into nonclinical studies. The FDA is accepting public comments for 90 days.