The FDA released draft guidance on using next-generation sequencing to assess safety risks for genome-edited gene therapy products, focusing on off-target editing and chromosomal integrity. The document outlines how sponsors can design nonclinical studies with sequencing and bioinformatics analyses, and how results would be submitted within INDs and BLA applications. In parallel, the guidance ties into the FDA’s broader plausible mechanism framework and earlier guidance on individualized therapies for ultra-rare diseases. The agency’s commissioner, Marty Makary, said the agency is “serious about moving this ball forward,” reflecting an effort to standardize safety assessment methods as gene-editing tools scale.