FDA shares gene therapy research guidance

March 17, 2022

The FDA is asking gene therapy developers to take it slow in a recently published draft guidance.

Through a recommendations draft issued earlier this week, the agency offered general guidance about the present and future of gene therapy research, outlining general considerations for product development, preclinical and clinical studies and more.  

Due to the growing interest in human gene editing and unprecedented scientific advancements, the technology available to treat human disease has increased significantly, the agency said.

"While the potential of such products for the treatment of human disease is clear, the potential risks are not as well understood," the FDA wrote in the 19-page document, which expands on previous recommendations for other forms of gene therapy drugs.

The document lays out the agency’s current grasp on gene editing components and outlines expectations for how companies should manufacture and test them. Perhaps more importantly, the document expands on what information should be provided to the agency when asking for human testing clearance.

Overall, the guidance document asks companies to thoroughly assess any complications or risks that could appear later as a result of gene editing, by tracking trials for at least 15 years. Along those lines, the agency recommended that companies stagger participant enrollment to allow for careful assessment of any side effects before increasing trial size.

The document covers a wide range of gene-editing techniques, including the cool kid in school CRISPR, older methods like transcription activator-like effector nucleases, and even methods that don’t involve breaking both DNA strands.