FDA Issues Draft Guidance on Gene-Editing Product Development

The US Food and Drug Administration (FDA) has published draft recommendations for potential manufacturers of gene therapy products that incorporate genome editing of human somatic cells.

Through these recommendations, the agency intends to provide guidance regarding information that should be provided in an Investigational New Drug application in order to assess the safety and quality of gene-therapy products. This includes product design, manufacturing, testing, preclinical safety assessment, and clinical trial design.

“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 its guidance document.

Source: FDA guidance document, March 2022.