The FDA released draft guidance describing how to use next-generation sequencing methods to assess safety for genome-edited gene therapy products, focusing on off-target editing and loss of genome integrity. The agency’s approach is intended to standardize nonclinical study designs that incorporate NGS and bioinformatics and can feed into both INDs and BLA submissions. FDA said sponsors should tailor sequencing strategies to the type of genomic change under assessment, including scenarios where short on- or off-target edits may be adequately captured with short-read approaches. The draft guidance aligns with the agency’s broader genome-editing frameworks for individualized ultra-rare therapies. Separately in the feed, an additional mention confirms FDA’s broader gene-editing safety work continues to evolve, reinforcing that developers should plan CMC and nonclinical work around sequencing-based risk assessment early in development.