Guidance for Industry Human Gene Therapy Products Incorporating Human Genome Editing
Title:
Guidance for Industry Human Gene Therapy Products Incorporating Human Genome Editing
Origin/Publisher:
FDA Office of Communication, Outreach and Development (OCOD), 10903 New Hampshire Ave., Bldg. 71, Rm. 3128, Silver Spring, MD 20993-0002
Document Type:
Draft Guidance for Industry
Content:
This Guidance is 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.