The FDA released draft guidance outlining how sponsors should use next-generation sequencing and bioinformatics in nonclinical studies to assess safety risks for genome-edited human gene therapies. The draft focuses on evaluating off-target editing and potential loss of genome integrity. In the draft framework, the sequencing strategy is intended to be incorporated into studies supporting investigational new drug applications and, later, biologics license applications. The FDA noted that unintended edits can disrupt normal cell function and harm patients, making robust off-target and genomic integrity assessment central to safety. The agency also tied the release to prior FDA efforts to accelerate individualized therapies for ultra-rare diseases and earlier gene-therapy guidance, while emphasizing standardized, scalable approaches that can help regulators compare submissions.