{"id":742,"date":"2026-07-23T23:22:00","date_gmt":"2026-07-23T14:22:00","guid":{"rendered":"https:\/\/onepress.co.kr\/index.php\/briefing\/2026-07-23-casgevy-age-2-expanded-approval-ja\/"},"modified":"2026-07-23T23:22:00","modified_gmt":"2026-07-23T14:22:00","slug":"2026-07-23-casgevy-age-2-expanded-approval-ja","status":"publish","type":"briefing","link":"https:\/\/onepress.co.kr\/index.php\/briefing\/2026-07-23-casgevy-age-2-expanded-approval-ja\/","title":{"rendered":"Casgevy\u304c2\u6b73\u4ee5\u4e0a\u3078\u62e1\u5927\u3001CRISPR\u6cbb\u7642\u304c\u3088\u308a\u4f4e\u5e74\u9f62\u306e\u60a3\u8005\u306b\u958b\u3044\u305f"},"content":{"rendered":"<p><strong>2026-07-23 23:18 KST<\/strong><\/p>\n<p>FDA\u306f2026\u5e747\u67081\u65e5\u3001Casgevy(exagamglogene autotemcel)\u30922\u6b73\u4ee5\u4e0a\u306e\u4e00\u90e8sickle cell disease\u307e\u305f\u306ftransfusion-dependent beta thalassemia\u60a3\u8005\u306b\u62e1\u5927\u627f\u8a8d\u3057\u305f\u3002\u91cd\u8981\u306a\u306e\u306f\u3001CRISPR\u30d9\u30fc\u30b9\u306e\u907a\u4f1d\u5b50\u6cbb\u7642\u306e\u7c73\u56fd\u9069\u5fdc\u304c\u5927\u5e45\u306b\u4f4e\u5e74\u9f62\u3078\u5e83\u304c\u3063\u305f\u3053\u3068\u3067\u3042\u308a\u3001\u5168\u60a3\u8005\u304c\u3059\u3050\u53d7\u3051\u3089\u308c\u308b\u7c21\u5358\u306a\u6cbb\u7642\u3067\u306f\u306a\u3044\u3002<\/p>\n<h2>\u8ab0\u306b\u95a2\u4fc2\u3059\u308b\u304b<\/h2>\n<p>\u5bfe\u8c61\u306f2\u6b73\u4ee5\u4e0a\u3067\u3001recurrent vaso-occlusive crises\u3092\u4f34\u3046sickle cell disease\u3001\u307e\u305f\u306ftransfusion-dependent beta thalassemia\u306e\u60a3\u8005\u3060\u3002FDA\u306fCasgevy\u304c\u4ee5\u524d\u306fSCD\u307e\u305f\u306fTDT\u306e12\u6b73\u4ee5\u4e0a\u60a3\u8005\u5411\u3051\u306b\u627f\u8a8d\u3055\u308c\u3066\u3044\u305f\u3068\u8aac\u660e\u3059\u308b\u3002\u82e5\u3044\u5b50\u3069\u3082\u306e\u5bb6\u65cf\u306b\u306f\u5927\u304d\u3044\u304c\u3001\u9069\u683c\u6027\u306f\u75be\u60a3\u57fa\u6e96\u3001\u65e2\u6cbb\u7642\u3001\u79fb\u690d\u30ea\u30b9\u30af\u3001\u5c02\u9580\u65bd\u8a2d\u3078\u306e\u30a2\u30af\u30bb\u30b9\u306b\u5de6\u53f3\u3055\u308c\u308b\u3002<\/p>\n<h2>\u4f55\u304c\u5909\u308f\u308b\u304b<\/h2>\n<p>FDA\u306f2026\u5e747\u67081\u65e5\u306bsupplemental approval\u3092\u51fa\u3057\u305f\u3002FDA\u306eCasgevy\u88fd\u54c1\u30da\u30fc\u30b8\u306fSTN 125787\u30922\u6b73\u4ee5\u4e0a\u306eSCD with recurrent VOCs\u307e\u305f\u306fTDT\u3068\u3057\u3066\u793a\u3059\u3002FDA\u306f\u3053\u308c\u30922\u6b73\u4ee5\u4e0aSCD\u60a3\u8005\u5411\u3051\u306b\u627f\u8a8d\u3055\u308c\u305f\u521d\u306egene therapy\u3068\u8aac\u660e\u3059\u308b\u3002Vertex\u306fSCD\u3068TDT\u306e\u4e21\u65b9\u30672\u6b73\u306e\u5b50\u3069\u3082\u307e\u3067\u5bfe\u8c61\u3068\u306a\u308b\u521d\u306e\u627f\u8a8d\u907a\u4f1d\u6cbb\u7642\u3068\u3059\u308b\u3002<\/p>\n<figure class=\"wp-block-image size-full\"><img decoding=\"async\" src=\"https:\/\/onepress.co.kr\/wp-content\/uploads\/2026\/07\/casgevy-age-2-expanded-approval-ja.png\" alt=\"\u5c0f\u5150\u8840\u6db2\u5185\u79d1\u76f8\u8ac7\u3068CRISPR\u5e79\u7d30\u80de\u6cbb\u7642\u7d4c\u8def\u3092\u8aac\u660e\u3059\u308b\u753b\u50cf\" loading=\"eager\" \/><figcaption class=\"op-briefing-image-caption\">\u5236\u4f5c\u753b\u50cf\uff1aONEPRESS\u304c\u5c0f\u5150\u8840\u6db2\u75be\u60a3\u76f8\u8ac7\u3068\u81ea\u5bb6CRISPR\u7de8\u96c6\u5e79\u7d30\u80de\u6cbb\u7642\u306e\u6d41\u308c\u3092\u8aac\u660e\u7528\u306b\u69cb\u6210\u3057\u305f\u3002\u5b9f\u969b\u306e\u6cbb\u7642\u5834\u9762\u3084\u7279\u5b9a\u88fd\u54c1\u306e\u5ba3\u4f1d\u3067\u306f\u306a\u3044\u3002<\/figcaption><\/figure>\n<h2>\u6cbb\u7642\u306e\u4e2d\u8eab<\/h2>\n<p>Casgevy\u306f\u60a3\u8005\u81ea\u8eab\u306ehematopoietic stem cells\u3092\u63a1\u53d6\u3057\u3001CRISPR\/Cas9\u3067\u7de8\u96c6\u3057\u3066\u3001\u4e00\u56de\u306e\u9759\u8108\u5185\u6295\u4e0e\u3067\u623b\u3059autologous cell therapy\u3060\u3002FDA\u306f\u7de8\u96c6\u7d30\u80de\u304c\u9aa8\u9ac4\u306b\u751f\u7740\u3059\u308b\u3068\u8aac\u660e\u3059\u308b\u3002\u305d\u306e\u305f\u3081\u85ac\u5c40\u3067\u53d7\u3051\u53d6\u308b\u9320\u5264\u3084\u901a\u5e38\u306e\u5916\u6765\u6ce8\u5c04\u3067\u306f\u306a\u304f\u3001\u7d30\u80de\u63a1\u53d6\u3001conditioning\u3001\u96c6\u4e2d\u7684\u306a\u7ba1\u7406\u3001\u9577\u671f\u8ffd\u8de1\u3092\u4f34\u3046\u3002<\/p>\n<h2>\u9650\u754c\u3068\u6ce8\u610f<\/h2>\n<p>\u3053\u308c\u306f\u7c73\u56fd\u3067\u306e\u627f\u8a8d\u3067\u3042\u308a\u3001\u56fd\u306b\u3088\u3063\u3066\u5e74\u9f62\u3084\u75be\u60a3\u306e\u9069\u5fdc\u306f\u7570\u306a\u308a\u5f97\u308b\u3002SCD\u3084TDT\u306e\u5168\u3066\u306e\u5b50\u3069\u3082\u304c\u5bfe\u8c61\u3068\u3044\u3046\u610f\u5473\u3067\u3082\u306a\u3044\u3002\u5c02\u9580\u65bd\u8a2d\u3067\u75be\u60a3\u306e\u91cd\u75c7\u5ea6\u3001\u65e2\u6cbb\u7642\u3001\u611f\u67d3\u3068\u79fb\u690d\u30ea\u30b9\u30af\u3001\u751f\u6b96\u6a5f\u80fd\u6e29\u5b58\u306e\u76f8\u8ac7\u3001\u8ffd\u8de1\u8a08\u753b\u3092\u78ba\u8a8d\u3059\u308b\u5fc5\u8981\u304c\u3042\u308b\u3002FDA\u306e\u88fd\u54c1\u6587\u66f8\u3068\u627f\u8a8d\u66f8\u304c\u51fa\u767a\u70b9\u3060\u3002<\/p>\n<h2>\u516c\u5f0f\u78ba\u8a8d\u5148<\/h2>\n<p>FDA\u767a\u8868\u3067\u627f\u8a8d\u65e5\u30012\u6b73\u4ee5\u4e0a\u3078\u306e\u62e1\u5927\u3001\u521d\u306e\u5c0f\u5150SCD gene therapy\u306e\u7bc4\u56f2\u3092\u78ba\u8a8d\u3059\u308b\u3002FDA Casgevy\u88fd\u54c1\u30da\u30fc\u30b8\u3067STN 125787\u9069\u5fdc\u3001package insert\u3001approval letter\u3092\u78ba\u8a8d\u3059\u308b\u3002Vertex\u767a\u8868\u3067\u88fd\u9020\u5143\u306e\u8aac\u660e\u3068SCD\/TDT\u8868\u73fe\u3092\u7167\u5408\u3059\u308b\u3002<\/p>\n<h2>\u516c\u5f0f\u51fa\u5178<\/h2>\n<p><a href=\"https:\/\/www.fda.gov\/news-events\/press-announcements\/fda-approves-first-gene-therapy-young-children-sickle-cell-disease\" target=\"_blank\" rel=\"noopener noreferrer\">FDA\u767a\u8868\uff1a\u627f\u8a8d\u65e5\u3001\u5e74\u9f62\u62e1\u5927\u3001\u521d\u306e\u5c0f\u5150SCD gene therapy\u7bc4\u56f2<\/a><\/p>\n<p><a href=\"https:\/\/www.fda.gov\/vaccines-blood-biologics\/casgevy\" target=\"_blank\" rel=\"noopener noreferrer\">FDA Casgevy\u30da\u30fc\u30b8\uff1aSTN 125787\u9069\u5fdc\u3068\u627f\u8a8d\u6587\u66f8<\/a><\/p>\n<p><a href=\"https:\/\/investors.vrtx.com\/news-releases\/news-release-details\/vertex-announces-us-fda-approval-expanded-use-casgevyr-treatment\" target=\"_blank\" rel=\"noopener noreferrer\">Vertex\u767a\u8868\uff1a\u62e1\u5927\u9069\u5fdc\u306b\u95a2\u3059\u308b\u88fd\u9020\u5143\u8aac\u660e<\/a><\/p>\n","protected":false},"excerpt":{"rendered":"<p>FDA\u306f2026\u5e747\u67081\u65e5\u3001Casgevy(exagamglogene autotemcel)\u30922\u6b73\u4ee5\u4e0a\u306e\u4e00\u90e8sickle cell disease\u307e\u305f\u306ftransfusion-dependent beta thalassemia\u60a3\u8005\u306b\u62e1\u5927\u627f\u8a8d\u3057\u305f\u3002\u91cd\u8981\u306a\u306e\u306f\u3001CRISPR\u30d9\u30fc\u30b9\u306e\u907a\u4f1d\u5b50\u6cbb\u7642\u306e\u7c73\u56fd\u9069\u5fdc\u304c\u5927\u5e45\u306b\u4f4e\u5e74\u9f62\u3078\u5e83\u304c\u3063\u305f\u3053\u3068\u3067\u3042\u308a\u3001\u5168\u60a3\u8005\u304c\u3059\u3050\u53d7\u3051\u3089\u308c\u308b\u7c21\u5358\u306a\u6cbb\u7642\u3067\u306f\u306a\u3044\u3002<\/p>\n","protected":false},"featured_media":0,"template":"","meta":[],"class_list":["post-742","briefing","type-briefing","status-publish","hentry"],"_links":{"self":[{"href":"https:\/\/onepress.co.kr\/index.php\/wp-json\/wp\/v2\/briefing\/742","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/onepress.co.kr\/index.php\/wp-json\/wp\/v2\/briefing"}],"about":[{"href":"https:\/\/onepress.co.kr\/index.php\/wp-json\/wp\/v2\/types\/briefing"}],"wp:attachment":[{"href":"https:\/\/onepress.co.kr\/index.php\/wp-json\/wp\/v2\/media?parent=742"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}