{"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 The guidance announced in this notice finalizes the draft guidance of the same title dated March 2022.","action":"Notice of availability.","agencies":[{"raw_name":"DEPARTMENT OF HEALTH AND HUMAN SERVICES","name":"Health and Human Services Department","id":221,"url":"https://www.federalregister.gov/agencies/health-and-human-services-department","json_url":"https://www.federalregister.gov/api/v1/agencies/221","parent_id":null,"slug":"health-and-human-services-department"},{"raw_name":"Food and Drug Administration","name":"Food and Drug Administration","id":199,"url":"https://www.federalregister.gov/agencies/food-and-drug-administration","json_url":"https://www.federalregister.gov/api/v1/agencies/199","parent_id":221,"slug":"food-and-drug-administration"}],"body_html_url":"https://www.federalregister.gov/documents/full_text/html/2024/01/30/2024-01788.html","cfr_references":[],"citation":"89 FR 5913","comment_url":null,"comments_close_on":null,"correction_of":null,"corrections":[],"dates":"The announcement of the guidance is published in the Federal Register on January 30, 2024.","disposition_notes":null,"docket_ids":["Docket No. FDA-2021-D-0398"],"dockets":[{"supporting_documents":[{"title":"Ref. 12 - Formal Meetings Between the FDA and Sponsors or Applicants of PDUFA Products re Human Gene Therapy Products Incorporating Human Genome Editing; Draft Guidance for Industry; Availability","document_id":"FDA-2021-D-0398-0014"},{"title":"Ref. 11- Providing Clinical Evidence of Effectiveness for Human Drug and Biological Products re Human Gene Therapy Products Incorporating Human Genome Editing; Draft Guidance for Industry; Availability","document_id":"FDA-2021-D-0398-0013"},{"title":"Ref. 10 - Long Term Follow Up After Admin of Human Gene Therapy Products re Human Gene Therapy Products Incorporating Human Genome Editing; Draft Guidance for Industry; Availability","document_id":"FDA-2021-D-0398-0012"},{"title":"Ref. 9 - E6(R2) Good Clinical Practice: Integrated Addendum to ICH E6(R1) re Human Gene Therapy Products Incorporating Human Genome Editing; Draft Guidance for Industry; Availability","document_id":"FDA-2021-D-0398-0011"},{"title":"Ref. 8 - Considerations for the Design of-Early Phase Clinical Trials of Cellular and Gene Therapy Products Guidance for Industry re Human Gene Therapy Products Incorporating Human Genome Editing; Draft Guidance for Industry; Availability","document_id":"FDA-2021-D-0398-0010"},{"title":"Ref. 7 - Guidance for Industry Preclinical Assessment of Investigational Cellular and Gene Therapy Products re Human Gene Therapy Products Incorporating Human Genome Editing; Draft Guidance for Industry; Availability","document_id":"FDA-2021-D-0398-0009"},{"title":"Ref. 6 - Final Guidance for Industry; Potency Tests-for Cellular and Gene Therapy Products re Human Gene Therapy Products Incorporating Human Genome Editing; Draft Guidance for Industry; Availability","document_id":"FDA-2021-D-0398-0008"},{"title":"Ref. 5 - Current Good Manufacturing Practice for Phase 1Investigational Drugs re Human Gene Therapy Products Incorporating Human Genome Editing; Draft Guidance for Industry; Availability","document_id":"FDA-2021-D-0398-0007"},{"title":"Ref. 4 - Drug Master Files Draft Guidance re Human Gene Therapy Products Incorporating Human Genome Editing; Draft Guidance for Industry; Availability","document_id":"FDA-2021-D-0398-0006"},{"title":"Ref. 3 - Contract Manufacturing Arrangements for-Drugs: Quality Agreements re Human Gene Therapy Products Incorporating Human Genome Editing; Draft Guidance for Industry; Availability","document_id":"FDA-2021-D-0398-0005"}],"agency_name":"FDA","documents":[{"comment_count":0,"comment_start_date":"2024-01-30","updated_at":"2024-01-30T09:57:51.132-05:00","comment_url":"https://www.regulations.gov/commenton/FDA-2021-D-0398-0041","allow_late_comments":null,"id":"FDA-2021-D-0398-0041","comment_end_date":null,"regulations_dot_gov_open_for_comment":false}],"supporting_documents_count":12,"id":"FDA-2021-D-0398","title":"Human Gene Therapy Products Incorporating Human Genome Editing; Draft Guidance for Industry; Availability"}],"document_number":"2024-01788","effective_on":null,"end_page":5914,"executive_order_notes":null,"executive_order_number":null,"full_text_xml_url":"https://www.federalregister.gov/documents/full_text/xml/2024/01/30/2024-01788.xml","html_url":"https://www.federalregister.gov/documents/2024/01/30/2024-01788/human-gene-therapy-products-incorporating-human-genome-editing-guidance-for-industry-availability","images":{},"images_metadata":{},"json_url":"https://www.federalregister.gov/api/v1/documents/2024-01788?publication_date=2024-01-30","mods_url":"https://www.govinfo.gov/metadata/granule/FR-2024-01-30/2024-01788/mods.xml","not_received_for_publication":null,"page_length":2,"page_views":{"count":1107,"last_updated":"2026-10-23 22:15:05 -0400"},"pdf_url":"https://www.govinfo.gov/content/pkg/FR-2024-01-30/pdf/2024-01788.pdf","presidential_document_number":null,"proclamation_number":null,"public_inspection_pdf_url":"https://public-inspection.federalregister.gov/2024-01788.pdf?1706535923","publication_date":"2024-01-30","raw_text_url":"https://www.federalregister.gov/documents/full_text/text/2024/01/30/2024-01788.txt","regulation_id_number_info":{},"regulation_id_numbers":[],"regulations_dot_gov_info":{"supporting_documents":[{"title":"Ref. 12 - Formal Meetings Between the FDA and Sponsors or Applicants of PDUFA Products re Human Gene Therapy Products Incorporating Human Genome Editing; Draft Guidance for Industry; Availability","document_id":"FDA-2021-D-0398-0014"},{"title":"Ref. 11- Providing Clinical Evidence of Effectiveness for Human Drug and Biological Products re Human Gene Therapy Products Incorporating Human Genome Editing; Draft Guidance for Industry; Availability","document_id":"FDA-2021-D-0398-0013"},{"title":"Ref. 10 - Long Term Follow Up After Admin of Human Gene Therapy Products re Human Gene Therapy Products Incorporating Human Genome Editing; Draft Guidance for Industry; Availability","document_id":"FDA-2021-D-0398-0012"},{"title":"Ref. 9 - E6(R2) Good Clinical Practice: Integrated Addendum to ICH E6(R1) re Human Gene Therapy Products Incorporating Human Genome Editing; Draft Guidance for Industry; Availability","document_id":"FDA-2021-D-0398-0011"},{"title":"Ref. 8 - Considerations for the Design of-Early Phase Clinical Trials of Cellular and Gene Therapy Products Guidance for Industry re Human Gene Therapy Products Incorporating Human Genome Editing; Draft Guidance for Industry; Availability","document_id":"FDA-2021-D-0398-0010"},{"title":"Ref. 7 - Guidance for Industry Preclinical Assessment of Investigational Cellular and Gene Therapy Products re Human Gene Therapy Products Incorporating Human Genome Editing; Draft Guidance for Industry; Availability","document_id":"FDA-2021-D-0398-0009"},{"title":"Ref. 6 - Final Guidance for Industry; Potency Tests-for Cellular and Gene Therapy Products re Human Gene Therapy Products Incorporating Human Genome Editing; Draft Guidance for Industry; Availability","document_id":"FDA-2021-D-0398-0008"},{"title":"Ref. 5 - Current Good Manufacturing Practice for Phase 1Investigational Drugs re Human Gene Therapy Products Incorporating Human Genome Editing; Draft Guidance for Industry; Availability","document_id":"FDA-2021-D-0398-0007"},{"title":"Ref. 4 - Drug Master Files Draft Guidance re Human Gene Therapy Products Incorporating Human Genome Editing; Draft Guidance for Industry; Availability","document_id":"FDA-2021-D-0398-0006"},{"title":"Ref. 3 - Contract Manufacturing Arrangements for-Drugs: Quality Agreements re Human Gene Therapy Products Incorporating Human Genome Editing; Draft Guidance for Industry; Availability","document_id":"FDA-2021-D-0398-0005"}],"comments_count":0,"agency_id":"FDA","comments_url":"https://www.regulations.gov/docketBrowser?rpp=50&so=DESC&sb=postedDate&po=0&dct=PS&D=FDA-2021-D-0398","supporting_documents_count":12,"docket_id":"FDA-2021-D-0398","document_id":"FDA-2021-D-0398-0041","regulation_id_number":null,"title":"Human Gene Therapy Products Incorporating Human Genome Editing; Draft Guidance for Industry; Availability","checked_regulationsdotgov_at":"2024-01-31T14:00:07Z"},"regulations_dot_gov_url":null,"significant":null,"signing_date":null,"start_page":5913,"subtype":null,"title":"Human Gene Therapy Products Incorporating Human Genome Editing; Guidance for Industry; Availability","toc_doc":"Human Gene Therapy Products Incorporating Human Genome Editing","toc_subject":"Guidance:\n","topics":[],"type":"Notice","volume":89}