The FDA released draft guidance outlining how sponsors should use next-generation sequencing (NGS) to assess off-target risks in genome-edited human gene therapy products. The draft document—“Safety Assessment of Genome Editing in Human Gene Therapy Products Using Next-Generation Sequencing”—describes non-clinical study design expectations using NGS and bioinformatics for on- and off-target edit analysis. The agency says the resulting data would be submitted in investigational new drug applications to support entry into human trials, and later in biologics license applications for market approval. The FDA also highlighted sequencing strategies that may be appropriate depending on the size of target-site changes, including the use of short-read sequencing for short DNA stretches under 50 base pairs. FDA Commissioner Marty Makary said the agency is focused on standardizing approaches to help sponsors evaluate safety risks more efficiently while minimizing unintended edits that could disrupt chromosomal integrity.