Human Gene Therapy Products Incorporating Human Genome Editing; Guidance for Industry; Availability

Issuing agencies

Abstract

The Food and Drug Administration (FDA or Agency) is announcing the availability of a final guidance entitled "Human Gene Therapy Products Incorporating Human Genome Editing; Guidance for Industry." The guidance document provides recommendations to sponsors developing human gene therapy products incorporating genome editing (GE) of human somatic cells. Specifically, the guidance provides recommendations regarding information that should be provided in an investigational new drug (IND) application to assess the safety and quality of the investigational GE product, including information on product design, product manufacturing and testing, nonclinical safety assessment, and clinical trial design. The guidance announced in this notice finalizes the draft guidance of the same title dated March 2022.