April 7, 2022 - FDA, Healthcare, Regulatory, United States

FDA Issues Draft Guidance on Human Gene Therapy Products Incorporating Human Genome Editing

Jianyuan (Yuan) Hua, FDA Regulatory and Compliance Associate in New York, contributed to the drafting of this post.

On March 14, 2022, U.S. Food and Drug Administration (FDA) issued two draft guidances: “Human Gene Therapy Products Incorporating Human Genome Editing (GE)” and a draft guidance on Chimeric Antigen Receptor (CAR) T cell product development. These draft guidance documents were issued on the heels of FDA’s first low-risk determination for a food animal with an intentional genomic alteration. These concurrent developments are indicative of FDA’s continued assessment of gene therapy and genome editing technology and attempts to provide guidance to innovators. Moreover, they may portend a growing focus on GE technologies at the regulatory agency. To this end, Center for Biologics Evaluation and Research (CBER) Director Peter Marks is anticipated to publish an op-ed titled “Enhancing gene therapy regulatory interactions” in Expert Opinion on Biological Therapy.

The draft guidance on human genome editing provides recommendations to sponsors developing human gene therapy products incorporating GE in human somatic cells and recommendations on the information that should be included in investigational new drug applications (INDs) for such products. INDs should include information on component design, manufacture and testing, and preclinical and clinical studies; the guidance also details the specific manufacturing and testing data needed for each type of genome editing product. Although the general chemistry, manufacturing, and controls recommendations for GE products are generally the same as previously described in other FDA guidance, the draft guidance provides additional recommendations that are specific to human GE products. For example, FDA recommends product sponsors use a design platform that best fits their genomic target and type of intended modification. FDA further suggests sponsors “optimize the GE components to reduce the potential for off-target genome modification, to the extent possible.”

Comments to the draft guidance on Human Gene Therapy Products Incorporating Human GE may be submitted by June 14, 2022. 

  • Comments may be electronically submitted to https://www.regulations.gov/docket/FDA-2021-D-0398.
  • Hardcopies of written comments may be sent to Dockets Management Staff (HFA-305), Food and Drug Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852.