GUIDANCE DOCUMENT
Human Gene Therapy Products Incorporating Human Genome Editing March 2022
Not for implementation. Contains non-binding recommendations.
This guidance is being distributed for comment purposes only.
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All written comments should be identified with this document's docket number: FDA-2021-D-0398
- Docket Number:
- FDA-2021-D-0398
- Issued by:
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Guidance Issuing OfficeCenter 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, product testing, preclinical safety assessment, and clinical trial design.
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