{"id":4929,"date":"2023-02-23T14:48:45","date_gmt":"2023-02-23T19:48:45","guid":{"rendered":"https:\/\/www.med.unc.edu\/bloodresearchcenter\/?p=4929"},"modified":"2023-02-23T14:48:45","modified_gmt":"2023-02-23T19:48:45","slug":"hemophilia-gene-therapy-pivotal-trials-published","status":"publish","type":"post","link":"https:\/\/www.med.unc.edu\/bloodresearchcenter\/2023\/02\/hemophilia-gene-therapy-pivotal-trials-published\/","title":{"rendered":"Hemophilia Gene Therapy Pivotal Trials Published"},"content":{"rendered":"<p><img loading=\"lazy\" decoding=\"async\" class=\"size-medium wp-image-2338 alignleft\" src=\"https:\/\/www.med.unc.edu\/bloodresearchcenter\/wp-content\/uploads\/sites\/1089\/2019\/12\/Nigel_Key-257x300.jpg\" alt=\"Headshot of Nigel Key, M.B.,CH.B.,F.R.C.P.\" width=\"257\" height=\"300\" \/>The results of 2 phase 3 trials of AAV-mediated gene therapy for hemophilia A and hemophilia B were published back-to-back in the New England Journal of Medicine on February 23, 2023.\u00a0 Dr. Nigel Key was a collaborator and co-author on both studies, each of which included patients from UNC. The hemophilia A study (Biomarin Pharmacuticals) included 134 patients with severe hemophilia A (factor VIII deficiency). The AAV5-hFVIII product (Roctavian\u2122) is now licensed in Europe and the application for licensure in the US is currently under consideration by the FDA. The hemophilia B study (CSL Behring) included 54 patients with severe or moderately severe hemophilia B (factor IX deficiency). The results from this study were pivotal in obtaining licensure for AAV5-hFIX (Hemgenix\u2122) in both Europe and the US.<\/p>\n<p><a href=\"https:\/\/www.nejm.org\/doi\/full\/10.1056\/NEJMoa2211644?query=TOC\" target=\"_blank\" rel=\"noopener\">Read <em>Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B<\/em> here<\/a><\/p>\n<p><a href=\"https:\/\/www.nejm.org\/doi\/full\/10.1056\/NEJMoa2211075?query=TOC&amp;ssotoken=U2FsdGVkX182t%2FYXYWwuNaEp2%2FVS%2BBMhj9UAyZzMqoOtH0X6RVYRTL1a1%2FzD3KjGNXj5OgdtXP1zKHtrIb%2BgFskOyT%2B%2F9byyB5hR9Xf6qttI3n79woqi4upS5SWlx1uxBoKf4fh2%2FZCIRoTi9O7P4%2B30zYkMNK8u%2BUUuE2HHg2%2BqEinp%2FenEfvgw6cc%2FHPDvX%2FHVn4GPyTkRhndJgax44g%3D%3D&amp;cid=NEJM%20eToc,%20February%2023,%202023%20DM2044084&amp;bid=1427439262\" target=\"_blank\" rel=\"noopener\">Read <em>Two-Year Outcomes of Valoctocogene Roxaparvovec Therapy for Hemophilia A<\/em> here<\/a><\/p>\n","protected":false},"excerpt":{"rendered":"<p>The results of 2 phase 3 trials of AAV-mediated gene therapy for hemophilia A and hemophilia B were published back-to-back in the New England Journal of Medicine on February 23, 2023.\u00a0 Dr. Nigel Key was a collaborator and co-author on both studies, each of which included patients from UNC. The hemophilia A study (Biomarin Pharmacuticals) &hellip; <a href=\"https:\/\/www.med.unc.edu\/bloodresearchcenter\/2023\/02\/hemophilia-gene-therapy-pivotal-trials-published\/\" aria-label=\"Read more about Hemophilia Gene Therapy Pivotal Trials Published\">Read more<\/a><\/p>\n","protected":false},"author":112194,"featured_media":2338,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"layout":"","cellInformation":"","apiCallInformation":"","footnotes":"","_links_to":"","_links_to_target":""},"categories":[2],"tags":[],"class_list":["post-4929","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-news","odd"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v26.8 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>Hemophilia Gene Therapy Pivotal Trials Published | UNC Blood Research Center<\/title>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/www.med.unc.edu\/bloodresearchcenter\/2023\/02\/hemophilia-gene-therapy-pivotal-trials-published\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Hemophilia Gene Therapy Pivotal Trials Published | UNC Blood Research Center\" \/>\n<meta property=\"og:description\" content=\"The results of 2 phase 3 trials of AAV-mediated gene therapy for hemophilia A and hemophilia B were published back-to-back in the New England Journal of Medicine on February 23, 2023.\u00a0 Dr. Nigel Key was a collaborator and co-author on both studies, each of which included patients from UNC. The hemophilia A study (Biomarin Pharmacuticals) &hellip; Read more\" \/>\n<meta property=\"og:url\" content=\"https:\/\/www.med.unc.edu\/bloodresearchcenter\/2023\/02\/hemophilia-gene-therapy-pivotal-trials-published\/\" \/>\n<meta property=\"og:site_name\" content=\"UNC Blood Research Center\" \/>\n<meta property=\"article:published_time\" content=\"2023-02-23T19:48:45+00:00\" \/>\n<meta property=\"og:image\" content=\"https:\/\/www.med.unc.edu\/bloodresearchcenter\/wp-content\/uploads\/sites\/1089\/2019\/12\/Nigel_Key-scaled-e1587662183114.jpg\" \/>\n\t<meta property=\"og:image:width\" content=\"600\" \/>\n\t<meta property=\"og:image:height\" content=\"701\" \/>\n\t<meta property=\"og:image:type\" content=\"image\/jpeg\" \/>\n<meta name=\"author\" content=\"amshespe\" \/>\n<meta name=\"twitter:card\" content=\"summary_large_image\" \/>\n<meta name=\"twitter:label1\" content=\"Written by\" \/>\n\t<meta name=\"twitter:data1\" content=\"amshespe\" \/>\n\t<meta name=\"twitter:label2\" 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