{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,14]],"date-time":"2026-08-14T01:49:45Z","timestamp":1786672185663,"version":"build-2736575974"},"reference-count":11,"publisher":"SAGE Publications","issue":"15-16","license":[{"start":{"date-parts":[[2023,8,1]],"date-time":"2023-08-01T00:00:00Z","timestamp":1690848000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/journals.sagepub.com\/page\/policies\/text-and-data-mining-license"}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["Human Gene Therapy"],"published-print":{"date-parts":[[2023,8]]},"DOI":"10.1089\/hum.2023.29251.bfs","type":"journal-article","created":{"date-parts":[[2023,8,17]],"date-time":"2023-08-17T14:54:42Z","timestamp":1692284082000},"page":"665-668","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":33,"title":["BioMarin's ROCTAVIAN Wins Food and Drug Administration Approval As First Gene Therapy for Severe Hemophilia A"],"prefix":"10.1177","volume":"34","author":[{"given":"Alex","family":"Philippidis","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","published-online":{"date-parts":[[2023,8,16]]},"reference":[{"key":"e_1_3_1_2_2","unstructured":"BioMarin Pharmaceutical. U.S. Food and Drug Administration Approves BioMarin's ROCTAVIAN\u2122 (valoctocogene roxaparvovec-rvox) the First and Only Gene Therapy for Adults with Severe Hemophilia A. June 29 2023. Available from: https:\/\/investors.biomarin.com\/2023-06-29-U-S-Food-and-Drug-Administration-Approves-BioMarins-ROCTAVIAN-TM-valoctocogene-roxaparvovec-rvox- -the-First-and-Only-Gene-Therapy-for-Adults-with-Severe-Hemophilia-A [Last accessed: July14 2023]."},{"key":"e_1_3_1_3_2","unstructured":"Myshko D. FDA Approves $2.9 Million Gene Therapy for Hemophilia A Formulary Watch. June 29 2023. Available from: https:\/\/www.formularywatch.com\/view\/fda-approves-2-9-million-gene-therapy-for-hemophilia-a [Last accessed: June14 2023]."},{"key":"e_1_3_1_4_2","unstructured":"Scribe Therapeutics. Scribe Therapeutics Expands Collaboration with Sanofi to Advance In Vivo Genetic Medicines for Sickle Cell and Other Genomic Diseases. July 17 2023. Available from: https:\/\/www.scribetx.com\/press-release-scribe-therapeutics-expands-collaboration-with-sanofi-to-advance-in-vivo-genetic-medicines-for-sickle-cell-and-other-genomic-diseases\/ [Last accessed: July17 2023]."},{"key":"e_1_3_1_5_2","unstructured":"Astellas Pharma and 4D Molecular Therapeutics. Astellas and 4D Molecular Therapeutics (4DMT) Enter into License Agreement to Use 4DMT's Proprietary Intravitreal R100 Vector for Rare Ophthalmic Targets. July 11 2023. Available from: https:\/\/www.astellas.com\/en\/news\/28136 [Last accessed: July11 2023]."},{"key":"e_1_3_1_6_2","unstructured":"Sarepta Therapeutics. Sarepta Therapeutics Announces FDA Approval of ELEVIDYS the First Gene Therapy to Treat Duchenne Muscular Dystrophy. June 22 2023. Available from: https:\/\/investorrelations.sarepta.com\/news-releases\/news-release-details\/sarepta-therapeutics-announces-fda-approval-elevidys-first-gene [Last accessed: June14 2023]."},{"key":"e_1_3_1_7_2","unstructured":"Blank C. First Gene Therapy for Duchenne Priced at $3.2 Million. June 23 2023. Formulary Watch. Available from: https:\/\/www.formularywatch.com\/view\/fda-approves-2-9-million-gene-therapy-for-hemophilia-a [Last accessed: June14 2023]."},{"key":"e_1_3_1_8_2","unstructured":"AVROBIO. AVROBIO to Explore Strategic Altrnatives. July 12 2023. Available from: https:\/\/investors.avrobio.com\/news-releases\/news-release-details\/avrobio-explore-strategic-alternatives [Last accessed: July14 2023]."},{"key":"e_1_3_1_9_2","unstructured":"U.S. Securities and Exchange Commission. Form 8-K filed July 12 2023. Available from: https:\/\/investors.avrobio.com\/static-files\/9cc9a41d-3e74-4bca-955a-522c94847e65 [Last accessed: July14 2023]."},{"key":"e_1_3_1_10_2","unstructured":"Pagliarulo N. Avrobio halts gene therapy research and considers a sale BioPharma Dive. July 12 2023. Available from: https:\/\/www.biopharmadive.com\/news\/avrobio-strategic-alternatives-sale-gene-therapy-research\/686643\/ [Last accessed: July14 2023]."},{"key":"e_1_3_1_11_2","doi-asserted-by":"crossref","unstructured":"Philippidis A. AGT Spins Out Addimmune Focusing on Functional HIV Cure. Genetic Engineering and Biotechnology News. July 7 2023. Available from: https:\/\/www.genengnews.com\/gen-edge\/agt-spins-out-addimmune-focusing-on-functional-hiv-cure\/ [Last accessed: July11 2023].","DOI":"10.1089\/genedge.5.1.93"},{"key":"e_1_3_1_12_2","doi-asserted-by":"crossref","unstructured":"Muvarak N Li H Lahusen T et al. Safety and durability of AGT103-T autologous T cell therapy for HIV infection in a Phase 1 trial. Frontiers in Medicine. November 14 2022. Available from: https:\/\/www.frontiersin.org\/articles\/10.3389\/fmed.2022.1044713\/full [Last accessed: July11 2023].","DOI":"10.3389\/fmed.2022.1044713"}],"container-title":["Human Gene Therapy"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/journals.sagepub.com\/doi\/full-xml\/10.1089\/hum.2023.29251.bfs","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/journals.sagepub.com\/doi\/pdf\/10.1089\/hum.2023.29251.bfs","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/journals.sagepub.com\/doi\/pdf\/10.1089\/hum.2023.29251.bfs","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,4,29]],"date-time":"2026-04-29T07:56:39Z","timestamp":1777449399000},"score":1,"resource":{"primary":{"URL":"https:\/\/journals.sagepub.com\/doi\/10.1089\/hum.2023.29251.bfs"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2023,8]]},"references-count":11,"journal-issue":{"issue":"15-16","published-print":{"date-parts":[[2023,8]]}},"alternative-id":["10.1089\/hum.2023.29251.bfs"],"URL":"https:\/\/doi.org\/10.1089\/hum.2023.29251.bfs","relation":{},"ISSN":["1043-0342","1557-7422"],"issn-type":[{"value":"1043-0342","type":"print"},{"value":"1557-7422","type":"electronic"}],"subject":[],"published":{"date-parts":[[2023,8]]}}}