{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,11,6]],"date-time":"2026-11-06T23:27:53Z","timestamp":1794007673310,"version":"4.8.0"},"reference-count":11,"publisher":"SAGE Publications","issue":"23-24","license":[{"start":{"date-parts":[[2026,10,19]],"date-time":"2026-10-19T00:00:00Z","timestamp":1792368000000},"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":[[2026,12,1]]},"DOI":"10.1177\/10430342261499483","type":"journal-article","created":{"date-parts":[[2026,10,20]],"date-time":"2026-10-20T06:16:12Z","timestamp":1792476972000},"page":"1075-1078","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":0,"title":["Food and Drug Administration Grants Ultragenyx\u2019s Fayuvi\u2122 First-Ever Approval for Sanfilippo Syndrome Type A Treatment"],"prefix":"10.1177","volume":"37","author":[{"given":"Alex","family":"Philippidis","sequence":"first","affiliation":[{"name":"Genetic Engineering &amp; Biotechnology News, Mary Ann Liebert, Inc., Publishers, New Rochelle, New York, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","published-online":{"date-parts":[[2026,10,19]]},"reference":[{"key":"e_1_3_1_2_1","unstructured":"1.Ultragenyx Pharmaceutical. Ultragenyx Announces Approval of FAYUVI\u2122 Gene Therapy the First-Ever FDA-Approved Treatment for Sanfilippo Syndrome Type A (MPS IIIA). 2026. Available from: https:\/\/ir.ultragenyx.com\/news-releases\/news-release-details\/ultragenyx-announces-approval-fayuvitm-gene-therapy-first-ever [Last accessed: October4 2026]."},{"key":"e_1_3_1_3_1","unstructured":"2.Roy S Choudhury K. Ultragenyx sets $3.95 million price for rare disease gene therapy. Reuters; 2026. Available from: https:\/\/www.reuters.com\/business\/healthcare-pharmaceuticals\/us-fda-approves-ultragenyxs-gene-therapy-rare-disorder-2026-09-17\/ [Last accessed: October4 2026]."},{"key":"e_1_3_1_4_1","unstructured":"3.Encoded Therapeutics. Encoded Therapeutics Raises $275 Million in Series F Financing to Advance ETX101 Through Pivotal Development and Accelerate its Precision Genetic Medicines Neurology Pipeline. 2026. Available from: https:\/\/encoded.com\/encoded-therapeutics-raises-275-million-in-series-f-financing-to-advance-etx101-through-pivotal-development-and-accelerate-its-precision-genetic-medicines-neurology-pipeline\/ [Last accessed: October3 2026]."},{"key":"e_1_3_1_5_1","unstructured":"4.Anthropic. Claude discovers a novel enzyme system with CRISPR-like repeats. 2026. Available from: https:\/\/www.anthropic.com\/news\/claude-discovers-novel-enzyme-system [Last accessed: October5 2026]."},{"key":"e_1_3_1_6_1","unstructured":"5.Amodei D. Today we announced the Claude-led discovery of a molecular machine that we suspect could represent a new gene editing mechanism. Its precise function. X (formerly Twitter); 2026. 2:43 pm. Available from: https:\/\/x.com\/DarioAmodei\/status\/2102831170299834652?s=43 [Last accessed: October5 2026]."},{"key":"e_1_3_1_7_1","article-title":"Anthropic\u2019s \u201creminiscent of CRISPR\u201d discovery dents gene editing stocks","author":"Philippidis A","year":"2026","unstructured":"6.Philippidis A. Anthropic\u2019s \u201creminiscent of CRISPR\u201d discovery dents gene editing stocks. Genetic Engineering & Biotechnology News 2026. Available from: https:\/\/www.genengnews.com\/topics\/translational-medicine\/stockwatch-nektar-shares-flat-as-90m-jury-verdict-less-than-sought-in-lilly-suit\/","journal-title":"Genetic Engineering & Biotechnology News"},{"key":"e_1_3_1_8_1","unstructured":"7.Olson P. \u201cAnthropic\u2019s $2 Trillion IPO Is Still On. And So Is the AI Race.\u201d Bloomberg News 2026. Available from: https:\/\/www.bloomberg.com\/opinion\/articles\/2026-09-15\/anthropic-s-2-trillion-ipo-is-still-on-and-so-is-the-ai-race [Last accessed: October5 2026]."},{"key":"e_1_3_1_9_1","unstructured":"8.REGENXBIO. Form 8-K Current Report. 2026. Available from: https:\/\/ir.regenxbio.com\/node\/13966\/html [Last accessed: October3 2026]."},{"key":"e_1_3_1_10_1","unstructured":"9.Sarepta Therapeutics. Form 8-K Current Report. 2026. Available from: https:\/\/investorrelations.sarepta.com\/static-files\/d86d4ea1-6aa9-40d2-a47b-5ad3ca16745f [Last accessed: October3 2026]."},{"key":"e_1_3_1_11_1","unstructured":"10.uniQure. uniQure Announces Additional Data from Ongoing Phase I\/II Studies of ifezuntirgene inilparvovec (AMT-130) in Huntington\u2019s Disease Showing Continued Slowing of Disease Progression. 2026. Available from: https:\/\/uniqure.gcs-web.com\/node\/13001\/pdf [Last accessed: October6 2026]."},{"key":"e_1_3_1_12_1","unstructured":"11.Northway Biotech. First in the Baltics: Northway Biotech opens \u20ac61 million Cell Therapy and Personalized Medicine CDMO in Vilnius. 2026. Available from: https:\/\/www.northwaybiotech.com\/blog\/first-in-the-baltics-northway-biotech-opens-e61-million-cell-therapy-and-personalized-medicine-cdmo-in-vilnius\/ [Last accessed: October6 2026]."}],"container-title":["Human Gene Therapy"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/journals.sagepub.com\/doi\/pdf\/10.1177\/10430342261499483","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/journals.sagepub.com\/doi\/full-xml\/10.1177\/10430342261499483","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/journals.sagepub.com\/doi\/pdf\/10.1177\/10430342261499483","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,11,6]],"date-time":"2026-11-06T23:16:24Z","timestamp":1794006984000},"score":1,"resource":{"primary":{"URL":"https:\/\/journals.sagepub.com\/doi\/10.1177\/10430342261499483"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,10,19]]},"references-count":11,"journal-issue":{"issue":"23-24","published-print":{"date-parts":[[2026,12,1]]}},"alternative-id":["10.1177\/10430342261499483"],"URL":"https:\/\/doi.org\/10.1177\/10430342261499483","relation":{},"ISSN":["1043-0342","1557-7422"],"issn-type":[{"value":"1043-0342","type":"print"},{"value":"1557-7422","type":"electronic"}],"subject":[],"published":{"date-parts":[[2026,10,19]]}}}