In 2018, the United States FDA released six draft guidance documents regarding gene therapy, and the final versions of these documents were released in January 2020. More details can be found in the Just the FACTs newsletter article, FDA Releases Six Draft Guidance Documents for Gene Therapy (2018).
The final documents can be accessed online:
- Chemistry, Manufacturing, and Control Information for Human Gene Therapy Investigational New Drug Applications; Guidance for Industry
- Testing of Retroviral Vector-Based Human Gene Therapy Products for Replication Competent Retrovirus During Product Manufacture and Patient Follow-up; Guidance for Industry
- Long Term Follow-Up After Administration of Human Gene Therapy Products; Guidance for Industry
- Human Gene Therapy for Retinal Disorders; Guidance for Industry
- Human Gene Therapy for Rare Diseases; Guidance for Industry
- Human Gene Therapy for Hemophilia; Guidance for Industry