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 The guidance announced in this notice finalizes the draft guidance of the same title dated March 2022.

The FDA Center for Biologics Evaluation and Research (CBER) Office of Therapeutic Products (OTP) is hosting a virtual public webinar on Thursday, February 29 at 1:00 p.m. to highlight key considerations in the final guidance. This event is free and open to the public. However, registration is required.

Registrants can submit questions in advance during the registration process. The deadline to submit questions via registration is February 13, 2024.

  • Date: Thursday, February 29, 2024
  • Time: 1:00 p.m. – 2:00 p.m. ET
  • Location: The webinar will be held via Zoom.

Avance Biosciences™ offers a series of services that support our clients’ discoveries in gene therapy and cell therapy including: potency assay development and testing, edited gene testingDNA/RNA biodistribution testing and NGS-based ID testing. Contact our technical staff to discuss how we can support you in your project!