FDA – Human Gene Therapy Products Incorporating Human Genome Editing; Draft Guidance for Industry

FDA – Human Gene Therapy Products Incorporating Human Genome Editing; Draft Guidance for Industry
[Docket No. FDA-2021-D-0398];
In this guidance, we, FDA, are providing recommendations to sponsors developing human gene therapy  products incorporating genome editing (GE) of human somatic cells.  Specifically, this guidance provides recommendations regarding information that should be provided in an Investigational New Drug (IND) application in order to assess the safety and quality of the investigational GE product, as required in Title 21 of the Code of Federal Regulations 312.23 (21 CFR 312.23).  This includes information on product design, product manufacturing, product testing, preclinical safety assessment, and clinical trial design.