{"abstract":"The Food and Drug Administration is announcing the availability of a final guidance entitled \"Human Gene Therapy for Retinal Disorders; Guidance for Industry.\" The final guidance provides recommendations to stakeholders developing human gene therapy (GT) products for retinal disorders affecting adult and pediatric patients. The guidance focuses on issues specific to GT products for retinal disorders and provides recommendations related to product development, preclinical testing, and clinical trial design for such GT products. The guidance announced in this notice finalizes the draft guidance of the same title dated July 2018.","action":"Notice of availability.","agencies":[{"raw_name":"DEPARTMENT OF HEALTH AND HUMAN SERVICES","name":"Health and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/2020/01/30/2020-01703.html","cfr_references":[],"citation":"85 FR 5454","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, 2020.","disposition_notes":null,"docket_ids":["Docket No. FDA-2018-D-2236"],"dockets":[],"document_number":"2020-01703","effective_on":null,"end_page":5455,"executive_order_notes":null,"executive_order_number":null,"explanation":null,"full_text_xml_url":"https://www.federalregister.gov/documents/full_text/xml/2020/01/30/2020-01703.xml","html_url":"https://www.federalregister.gov/documents/2020/01/30/2020-01703/human-gene-therapy-for-retinal-disorders-guidance-for-industry-availability","images":{},"images_metadata":{},"json_url":"https://www.federalregister.gov/api/v1/documents/2020-01703?publication_date=2020-01-30","mods_url":"https://www.govinfo.gov/metadata/granule/FR-2020-01-30/2020-01703/mods.xml","not_received_for_publication":null,"page_length":2,"page_views":{"count":366,"last_updated":"2026-04-04 18:15:03 -0400"},"pdf_url":"https://www.govinfo.gov/content/pkg/FR-2020-01-30/pdf/2020-01703.pdf","presidential_document_number":null,"proclamation_number":null,"public_inspection_pdf_url":"https://public-inspection.federalregister.gov/2020-01703.pdf?1580305539","publication_date":"2020-01-30","raw_text_url":"https://www.federalregister.gov/documents/full_text/text/2020/01/30/2020-01703.txt","regulation_id_number_info":{},"regulation_id_numbers":[],"regulations_dot_gov_info":{"supporting_documents":[{"title":"Reference 13 Formal Meetings between FDA and Sponsors or Applicants Guidance re Human Gene Therapy for Retinal Disorders; Guidance for Industry; Availability","document_id":"FDA-2018-D-2236-0029"},{"title":"Reference 12 Expedited Programs for Regenerative Medicine re Human Gene Therapy for Retinal Disorders; Guidance for Industry; Availability","document_id":"FDA-2018-D-2236-0028"},{"title":"Reference 11  Expedited Programs Guidance Serious Conditions re Human Gene Therapy for Retinal Disorders; Guidance for Industry; Availability","document_id":"FDA-2018-D-2236-0027"},{"title":"Reference 10 Rare Diseases Common Issues in Drug Development Guidance re Human Gene Therapy for Retinal Disorders; Guidance for Industry; Availability","document_id":"FDA-2018-D-2236-0026"},{"title":"Reference 9 Providing Clinical Evidence of Effectiveness for Human Drug and Biological Products re Human Gene Therapy for Retinal Disorders; Guidance for Industry; Availability","document_id":"FDA-2018-D-2236-0025"},{"title":"Reference 8 Considerations for Design of Early Phase Clinical Trials Guidance re Human Gene Therapy for Retinal Disorders; Guidance for Industry; Availability","document_id":"FDA-2018-D-2236-0024"},{"title":"Reference 7 ICH Comparability Guideline re Human Gene Therapy for Retinal Disorders; Guidance for Industry; Availability","document_id":"FDA-2018-D-2236-0023"},{"title":"Reference 6 Comparability Protocols for Human Drugs and Biologics CMC Information re Human Gene Therapy for Retinal Disorders; Guidance for Industry; Availability","document_id":"FDA-2018-D-2236-0022"},{"title":"Reference 5 IPRP GTWG ReflectionPaper BD Final 2018 0713 re Human Gene Therapy for Retinal Disorders; Guidance for Industry; Availability","document_id":"FDA-2018-D-2236-0021"},{"title":"Reference 4 Long Term_Follow Up GT Guidance re Human Gene Therapy for Retinal Disorders; Guidance for Industry; Availability","document_id":"FDA-2018-D-2236-0020"}],"comments_url":"https://www.regulations.gov/docketBrowser?rpp=50&so=DESC&sb=postedDate&po=0&dct=PS&D=FDA-2018-D-2236","supporting_documents_count":14,"docket_id":"FDA-2018-D-2236","regulation_id_number":null,"title":"Human Gene Therapy for Retinal Disorders; Draft Guidance for Industry; Availability ","checked_regulationsdotgov_at":"2020-02-02T04:30:08Z"},"regulations_dot_gov_url":null,"significant":null,"signing_date":null,"start_page":5454,"subtype":null,"title":"Human Gene Therapy for Retinal Disorders; Guidance for Industry; Availability","toc_doc":"Human Gene Therapy for Retinal Disorders","toc_subject":"Guidance:","topics":[],"type":"Notice","volume":85}