RARE Daily

FDA Releases Draft Guidance for Gene Editing Products

March 15, 2022

The U.S. Food and Drug Administration released draft guidance for gene therapy products that incorporate human genome editing that seeks to clarify the agency’s position on what information should be included in an application to begin human clinical trials in order to assess the safety and quality of the experimental gene editing product.

The agency noted that over the past 10 years, the level of interest in human gene editing has increased substantially. While the potential for these products is well recognized, the potential risks are not as well understood.

The agency defined a gene editing product covered by the guidance as one that uses a process by which DNA sequences are added, deleted, altered, or replaced at specified location(s) in the genome of human somatic cells, ex vivo or in vivo, using nuclease-dependent or nuclease-independent GE technologies.

The document provides guidance for product development, preclinical studies, and clinical studies. Much of the guidance is focused on the safety of these products and the means of identifying and minimizing off-target effects of these therapies.

In addition to a thorough safety monitoring strategy, the agency calls for a well-defined toxicity grading system and toxicity management plan including adequate monitoring for off-target editing.

It also calls for long-term follow-up of at least 15 years after product administration in line with the FDA’s previous guidance for gene therapy.

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