FDA发布基因编辑人类基因治疗产品利用先验知识开发指南草案

GUIDANCE DOCUMENT

Draft

Not for implementation. Contains non-binding recommendations.

Docket Number:

FDA-2026-D-1257

Issued by:

Guidance Issuing Office

Center for Biologics Evaluation and Research, Office of Therapeutic Products

For a high-level overview of this guidance document, please watch this recorded webinar featuring Stella Lee, PhD and Emily Wires, PhD from FDA’s Office of Therapeutic Products.

We, the FDA, are issuing this draft guidance to assist you (manufacturers, applicants, and other stakeholders) in developing human gene therapy (GT) products incorporating ex-vivo and in vivo genome editing (GE) of human somatic cells (GE products).  Specifically, this guidance reflects FDA’s current thinking on the type of prior knowledge (i.e., public and platform knowledge)  that may be scientifically appropriate to leverage  to advance product development. This guidance also provides recommendations on how sponsors may consider leveraging prior knowledge to increase review efficiency and accelerate product development across multiple programs.  These recommendations include how to leverage chemistry, manufacturing, and controls (CMC); nonclinical; and clinical prior knowledge.  The ability to leverage prior knowledge to expedite product development may be particularly helpful in the context of GE products intended to treat rare diseases, many of which may be serious and life threatening. 
 

Submit Comments

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-2026-D-1257.

Questions?