Human Gene Therapy Products Incorporating Human Genome Editing

Human Gene Therapy Products Incorporating Human Genome Editing guidance the 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 and testing, nonclinical safety assessment, and clinical trial design.