FDA drafts guidance on genome editing, CAR-T cell therapies

The US Food and Drug Administration (FDA) has issued two draft guidances addressing the development of human gene therapy products that incorporate genome editing of human somatic cells and chimeric antigen receptor (CAR) T cell products.

In the draft guidance on human gene therapy products that incorporate human genome editing, FDA offers recommendations on the information that should be included in an investigational new drug (IND) application. The draft guidance, issued on 15 March by FDA’s Center for Biologics Evaluation and Research (CBER), provides information on component design, component manufacture and testing, product manufacture and testing, and preclinical and clinical studies.

“FDA evaluates human [genome editing] products using a science-based approach weighing the benefits and risks of each product,” the agency wrote. “The benefit-risk profile for each product depends on the proposed indication and patient population, the extent and duration of therapeutic benefit achieved, and the availability of alternative therapeutic options. Some of the specific risks associated with [genome editing] approaches include off-target editing, unintended consequences of on- and off-target editing, and the unknown long term effects of on- and off-target editing.”

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