The FDA issued draft guidance on how to assess the safety of genome editing in human gene therapy products using next-generation sequencing (NGS). The agency’s recommendations focus on designing nonclinical studies with NGS and bioinformatics to characterize off-target editing risk and evaluate potential loss of genome integrity before therapies enter human trials or biologics license applications. The draft also outlines how sponsors can incorporate sequencing strategies to assess on- and off-target sites, including approaches suited to short edit regions (under 50 base pairs). FDA Commissioner Marty Makary said the regulator is “serious about moving this ball forward,” as sponsors develop CRISPR- and nuclease-based platforms. In parallel, the FDA also sent reminders to sponsors and researchers, urging timely results disclosures for clinical trials listed on ClinicalTrials.gov, in an attempt to reduce the share of studies that remain unpublished on outcomes.