FDA Issues Draft Guidance on Genome Editing Safety Standards to Advance Gene Therapy Development
The FDA issued a draft guidance document outlining safety standards for sponsors developing human gene therapy products that utilize genome editing technologies.
This guidance affects sponsors, manufacturers, and clinical investigators involved in the development of human gene therapies utilizing genome editing.
The issuance of this guidance suggests the FDA is establishing specific safety expectations for genome editing, which may require developers to refine their preclinical and clinical data packages. Regulatory teams should anticipate more rigorous scrutiny of off-target effects and long-term safety profiles associated with these technologies.
Sponsors should review the draft guidance to assess current genome editing protocols against the FDA's proposed safety standards and prepare to submit formal comments if alignment is needed on technical requirements.
The U.S. Food and Drug Administration today issued a draft guidance for sponsors seeking approval of human gene therapy products involving genome editing technologies.
Open in openFDA / FDA.gov