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GUIDANCE DOCUMENT

Human Gene Therapy Products Incorporating Human Genome Editing Guidance for Industry January 2024

Download the Final Guidance Document Read the Federal Register Notice
Final
Docket Number:
FDA-2021-D-0398
Issued by:
Guidance Issuing Office
Center for Biologics Evaluation and Research

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 and testing, nonclinical safety assessment, and clinical trial design. 

The FDA Center for Biologics Evaluation and Research (CBER) Office of Therapeutic Products (OTP) hosted a virtual public webinar on Thursday, February 29 at 1:00 p.m. to highlight key considerations in the final guidance. 

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Submit Comments Online

You can submit online or written comments on any guidance at any time (see 21 CFR 10.115(g)(5))

If unable to submit comments online, please mail written comments to:

Dockets Management
Food and Drug Administration
5630 Fishers Lane, Rm 1061
Rockville, MD 20852

All written comments should be identified with this document's docket number: FDA-2021-D-0398.

Questions?

Contact Point
Office of Communication, Outreach and Development (OCOD)
Center for Biologics Evaluation and Research
Food and Drug Administration
10903 New Hampshire Ave WO71-3128
Silver Spring, MD 20993-0002
ocod@fda.hhs.gov
(800) 835-4709
(240) 402-8010
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