Safety Assessment of Genome Editing in Human Gene Therapy Products Using Next-Generation Sequencing; Draft Guidance for Industry; Availability

The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft document entitled "Safety Assessment of Genome Editing in Human Gene Therapy Products Using Next-Generation Sequencing; Draft Guidance for Industry." The draft document provides recommendations for next-generation sequencing (NGS)-based methods used in nonclinical studies that will likely be needed to support initiation of clinical trials of investigational human genome editing (GE) products.

Abstract as published in the Federal Register.

The complete document — every provision, the docket, and the comment instructions — is published in the Federal Register. SPEQ surfaces and indexes it for reference; the authoritative text lives on federalregister.gov.