On March 15th, 2022 the FDA is announcing the availability of two draft guidance documents: “Human Gene Therapy Products Incorporating Human Genome Editing,” and “Considerations for the Development of Chimeric Antigen Receptor (CAR) T Cell Products.”
The draft guidance, “Human Gene Therapy Products Incorporating Human Genome Editing,” is intended to provide recommendations to sponsors developing human gene therapy products incorporating genome editing (GE) of human somatic cells. The draft guidance provides recommendations regarding information that should be provided in Investigational New Drug (IND) applications for GE products, including information on product design, product manufacturing, product testing, preclinical safety assessment, and clinical trial design.
The draft guidance, “Considerations for the Development of Chimeric Antigen Receptor (CAR) T Cell Products,” is intended to assist sponsors developing human gene therapy products in which the T cell specificity is genetically modified to enable recognition of a desired target antigen for therapeutic purposes. The guidance provides CAR T cell-specific recommendations regarding chemistry, manufacturing, and control (CMC), pharmacology and toxicology, and clinical study design. While this guidance specifically focuses on CAR T cell products, much of the information and recommendations provided will also be applicable to other genetically modified lymphocyte products, such as CAR Natural Killer (NK) cells or T cell receptor (TCR)‑modified T cells.
These draft guidance are being issued consistent with FDA’s good guidance practices regulation (21 CFR 10.115). Guidance documents describe FDA’s interpretation of their policy on a regulatory issue. A guidance, when finalized, will represent the current thinking of FDA on this topic.
Source – U.S. Food and Drug Administration
Several advocacy groups including the American Society of Gene & Cell Therapy and the Association for the Advancement of Blood & Biotherapies are preparing comments on the draft guidance.
Comments on the draft guidance must be submitted either electronically or written by June 14, 2022.