{"id":744,"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-zh\/"},"modified":"2026-07-23T23:22:00","modified_gmt":"2026-07-23T14:22:00","slug":"2026-07-23-casgevy-age-2-expanded-approval-zh","status":"publish","type":"briefing","link":"https:\/\/onepress.co.kr\/index.php\/briefing\/2026-07-23-casgevy-age-2-expanded-approval-zh\/","title":{"rendered":"Casgevy\u6269\u5927\u52302\u5c81\u4ee5\u4e0a\uff0cCRISPR\u7597\u6cd5\u9762\u5411\u66f4\u5c0f\u60a3\u8005"},"content":{"rendered":"<p><strong>2026-07-23 23:18 KST<\/strong><\/p>\n<p>FDA\u4e8e2026\u5e747\u67081\u65e5\u5bf9Casgevy\uff08exagamglogene autotemcel\uff09\u4f5c\u51fa\u8865\u5145\u6279\u51c6\uff0c\u9002\u7528\u4e8e\u90e8\u52062\u5c81\u53ca\u4ee5\u4e0asickle cell disease\u6216transfusion-dependent beta thalassemia\u60a3\u8005\u3002\u5173\u952e\u53d8\u5316\u662f\uff0cCRISPR\u57fa\u56e0\u7597\u6cd5\u5728\u7f8e\u56fd\u7684\u9002\u5e94\u75c7\u4e0b\u63a2\u5230\u66f4\u5c0f\u513f\u7ae5\uff0c\u800c\u4e0d\u662f\u6240\u6709\u60a3\u8005\u90fd\u80fd\u7acb\u5373\u63a5\u53d7\u7684\u7b80\u5355\u6cbb\u7597\u3002<\/p>\n<h2>\u8c01\u4f1a\u53d7\u5f71\u54cd<\/h2>\n<p>\u6269\u5927\u6279\u51c6\u5bf9\u8c61\u662f2\u5c81\u53ca\u4ee5\u4e0a\u3001\u60a3\u6709\u4f34recurrent vaso-occlusive crises\u7684sickle cell disease\u6216transfusion-dependent beta thalassemia\u7684\u60a3\u8005\u3002FDA\u79f0\uff0cCasgevy\u6b64\u524d\u5df2\u83b7\u6279\u7528\u4e8e12\u5c81\u53ca\u4ee5\u4e0aSCD\u6216TDT\u60a3\u8005\u3002\u8fd9\u4e2a\u53d8\u5316\u5bf9\u5e74\u5e7c\u513f\u7ae5\u5bb6\u5ead\u6709\u610f\u4e49\uff0c\u4f46\u8d44\u683c\u4ecd\u53d6\u51b3\u4e8e\u75be\u75c5\u6807\u51c6\u3001\u65e2\u5f80\u6cbb\u7597\u3001\u79fb\u690d\u98ce\u9669\u548c\u4e13\u4e1a\u4e2d\u5fc3\u53ef\u53ca\u6027\u3002<\/p>\n<h2>\u6539\u53d8\u4e86\u4ec0\u4e48<\/h2>\n<p>FDA\u4e8e2026\u5e747\u67081\u65e5\u4f5c\u51fasupplemental approval\u3002FDA Casgevy\u4ea7\u54c1\u9875\u5c06STN 125787\u5217\u4e3a2\u5c81\u53ca\u4ee5\u4e0aSCD with recurrent VOCs\u6216TDT\u60a3\u8005\u3002FDA\u79f0\uff0c\u8fd9\u662f\u9996\u4e2a\u83b7\u6279\u7528\u4e8e2\u5c81\u53ca\u4ee5\u4e0aSCD\u60a3\u8005\u7684gene therapy\u3002Vertex\u79f0\uff0c\u5b83\u662f\u9996\u4e2a\u540c\u65f6\u9762\u5411SCD\u548cTDT\u3001\u4f4e\u81f32\u5c81\u513f\u7ae5\u7684\u83b7\u6279\u9057\u4f20\u7597\u6cd5\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-zh.png\" alt=\"\u89e3\u91ca\u513f\u79d1\u8840\u6db2\u75c5\u54a8\u8be2\u548cCRISPR\u5e72\u7ec6\u80de\u6cbb\u7597\u8def\u5f84\u7684\u56fe\u7247\" loading=\"eager\" \/><figcaption class=\"op-briefing-image-caption\">\u5236\u4f5c\u56fe\u7247\uff1aONEPRESS\u7528\u8bf4\u660e\u6027\u753b\u9762\u5448\u73b0\u513f\u79d1\u8840\u6db2\u75c5\u54a8\u8be2\u548c\u81ea\u4f53CRISPR\u7f16\u8f91\u5e72\u7ec6\u80de\u6cbb\u7597\u8def\u5f84\u3002\u5b83\u4e0d\u662f\u5b9e\u9645\u6cbb\u7597\u573a\u666f\uff0c\u4e5f\u4e0d\u662f\u4ea7\u54c1\u5ba3\u4f20\u3002<\/figcaption><\/figure>\n<h2>\u6cbb\u7597\u662f\u4ec0\u4e48<\/h2>\n<p>Casgevy\u4f7f\u7528\u60a3\u8005\u81ea\u5df1\u7684hematopoietic stem cells\uff0c\u7ecfCRISPR\/Cas9\u7f16\u8f91\u540e\u4ee5\u4e00\u6b21\u9759\u8109\u8f93\u6ce8\u65b9\u5f0f\u56de\u8f93\u3002FDA\u79f0\uff0c\u7f16\u8f91\u540e\u7684\u7ec6\u80de\u4f1a\u690d\u5165\u9aa8\u9ad3\u3002\u56e0\u6b64\u5b83\u4e0d\u662f\u836f\u623f\u836f\u7247\u6216\u666e\u901a\u95e8\u8bca\u6ce8\u5c04\uff0c\u800c\u662f\u6d89\u53ca\u7ec6\u80de\u91c7\u96c6\u3001conditioning\u3001\u5bc6\u96c6\u62a4\u7406\u89c4\u5212\u548c\u957f\u671f\u968f\u8bbf\u7684\u6cbb\u7597\u8def\u5f84\u3002<\/p>\n<h2>\u9650\u5236\u548c\u6ce8\u610f<\/h2>\n<p>\u8fd9\u662f\u7f8e\u56fd\u6279\u51c6\uff0c\u5176\u4ed6\u56fd\u5bb6\u7684\u5e74\u9f84\u6216\u75be\u75c5\u9002\u5e94\u75c7\u53ef\u80fd\u4e0d\u540c\u3002\u5b83\u4e5f\u4e0d\u8868\u793a\u6bcf\u4e2aSCD\u6216TDT\u513f\u7ae5\u90fd\u7b26\u5408\u6761\u4ef6\u3002\u5bb6\u5ead\u9700\u8981\u7531\u4e13\u79d1\u4e2d\u5fc3\u8bc4\u4f30\u75be\u75c5\u4e25\u91cd\u7a0b\u5ea6\u3001\u65e2\u5f80\u6cbb\u7597\u3001\u611f\u67d3\u548c\u79fb\u690d\u98ce\u9669\u3001\u751f\u80b2\u80fd\u529b\u4fdd\u5b58\u54a8\u8be2\u4ee5\u53ca\u968f\u8bbf\u8ba1\u5212\u3002FDA\u4ea7\u54c1\u6587\u4ef6\u548c\u6279\u51c6\u51fd\u662f\u5224\u65ad\u8d77\u70b9\u3002<\/p>\n<h2>\u5b98\u65b9\u6838\u67e5\u8def\u5f84<\/h2>\n<p>FDA\u53d1\u5e03\u5185\u5bb9\u53ef\u6838\u67e5\u6279\u51c6\u65e5\u671f\u30012\u5c81\u4ee5\u4e0a\u6269\u5927\u548c\u9996\u4e2a\u513f\u79d1SCD gene therapy\u8303\u56f4\u3002FDA Casgevy\u4ea7\u54c1\u9875\u53ef\u6838\u67e5STN 125787\u9002\u5e94\u75c7\u3001package insert\u548c\u6279\u51c6\u51fd\u3002Vertex\u53d1\u5e03\u5185\u5bb9\u53ef\u5bf9\u7167\u5236\u9020\u5546\u5173\u4e8e\u6269\u5927\u9002\u5e94\u75c7\u7684\u8868\u8ff0\u3002<\/p>\n<h2>\u5b98\u65b9\u6765\u6e90<\/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\u53d1\u5e03\uff1a\u6279\u51c6\u65e5\u671f\u3001\u5e74\u9f84\u6269\u5927\u548c\u9996\u4e2a\u513f\u79d1SCD gene therapy\u8303\u56f4<\/a><\/p>\n<p><a href=\"https:\/\/www.fda.gov\/vaccines-blood-biologics\/casgevy\" target=\"_blank\" rel=\"noopener noreferrer\">FDA Casgevy\u9875\u9762\uff1aSTN 125787\u9002\u5e94\u75c7\u548c\u6279\u51c6\u6587\u4ef6<\/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\u53d1\u5e03\uff1a\u5236\u9020\u5546\u5bf9\u6269\u5927\u9002\u5e94\u75c7\u7684\u8bf4\u660e<\/a><\/p>\n","protected":false},"excerpt":{"rendered":"<p>FDA\u4e8e2026\u5e747\u67081\u65e5\u5bf9Casgevy\uff08exagamglogene autotemcel\uff09\u4f5c\u51fa\u8865\u5145\u6279\u51c6\uff0c\u9002\u7528\u4e8e\u90e8\u52062\u5c81\u53ca\u4ee5\u4e0asickle cell disease\u6216transfusion-dependent beta thalassemia\u60a3\u8005\u3002\u5173\u952e\u53d8\u5316\u662f\uff0cCRISPR\u57fa\u56e0\u7597\u6cd5\u5728\u7f8e\u56fd\u7684\u9002\u5e94\u75c7\u4e0b\u63a2\u5230\u66f4\u5c0f\u513f\u7ae5\uff0c\u800c\u4e0d\u662f\u6240\u6709\u60a3\u8005\u90fd\u80fd\u7acb\u5373\u63a5\u53d7\u7684\u7b80\u5355\u6cbb\u7597\u3002<\/p>\n","protected":false},"featured_media":0,"template":"","meta":[],"class_list":["post-744","briefing","type-briefing","status-publish","hentry"],"_links":{"self":[{"href":"https:\/\/onepress.co.kr\/index.php\/wp-json\/wp\/v2\/briefing\/744","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=744"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}