FDA published a final guidance document, Human Gene Therapy Products Incorporating Human Genome Editing, on Jan. 30, 2024. The document provides recommendations for developing gene therapy products incorporating genome editing (GE) of human somatic cells. Recommendations include information needed in an investigational new drug application for the assessment of the safety and quality of GE products. This information pertains to product design, manufacturing and testing, nonclinical safety assessment, and clinical trial design.
According to the agency, the use of human GE has increased, which has resulted in rapid development of gene therapy products that incorporate GE. FDA advises that the risks of these products is not well understood.