{"id":743,"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-vi\/"},"modified":"2026-07-23T23:22:00","modified_gmt":"2026-07-23T14:22:00","slug":"2026-07-23-casgevy-age-2-expanded-approval-vi","status":"publish","type":"briefing","link":"https:\/\/onepress.co.kr\/index.php\/briefing\/2026-07-23-casgevy-age-2-expanded-approval-vi\/","title":{"rendered":"Casgevy m\u1edf r\u1ed9ng xu\u1ed1ng t\u1eeb 2 tu\u1ed5i, \u0111\u01b0a li\u1ec7u ph\u00e1p CRISPR \u0111\u1ebfn b\u1ec7nh nh\u00e2n nh\u1ecf h\u01a1n"},"content":{"rendered":"<p><strong>2026-07-23 23:18 KST<\/strong><\/p>\n<p>FDA ban h\u00e0nh ph\u00ea duy\u1ec7t b\u1ed5 sung ng\u00e0y 1\/7\/2026 cho Casgevy (exagamglogene autotemcel) \u1edf m\u1ed9t s\u1ed1 b\u1ec7nh nh\u00e2n t\u1eeb 2 tu\u1ed5i tr\u1edf l\u00ean m\u1eafc sickle cell disease ho\u1eb7c transfusion-dependent beta thalassemia. Thay \u0111\u1ed5i ch\u00ednh l\u00e0 ch\u1ec9 \u0111\u1ecbnh t\u1ea1i M\u1ef9 c\u1ee7a li\u1ec7u ph\u00e1p gene CRISPR m\u1edf xu\u1ed1ng nh\u00f3m tr\u1ebb nh\u1ecf h\u01a1n, kh\u00f4ng ph\u1ea3i \u0111i\u1ec1u tr\u1ecb \u0111\u01a1n gi\u1ea3n m\u00e0 m\u1ecdi b\u1ec7nh nh\u00e2n nh\u1eadn ngay.<\/p>\n<h2>Ai b\u1ecb \u1ea3nh h\u01b0\u1edfng<\/h2>\n<p>Ph\u00ea duy\u1ec7t m\u1edf r\u1ed9ng \u00e1p d\u1ee5ng cho b\u1ec7nh nh\u00e2n t\u1eeb 2 tu\u1ed5i tr\u1edf l\u00ean b\u1ecb sickle cell disease v\u1edbi recurrent vaso-occlusive crises, ho\u1eb7c transfusion-dependent beta thalassemia. FDA n\u00f3i Casgevy tr\u01b0\u1edbc \u0111\u00f3 \u0111\u01b0\u1ee3c duy\u1ec7t cho b\u1ec7nh nh\u00e2n t\u1eeb 12 tu\u1ed5i tr\u1edf l\u00ean v\u1edbi SCD ho\u1eb7c TDT. \u0110i\u1ec1u n\u00e0y quan tr\u1ecdng v\u1edbi gia \u0111\u00ecnh c\u00f3 tr\u1ebb nh\u1ecf, nh\u01b0ng \u0111\u1ee7 \u0111i\u1ec1u ki\u1ec7n c\u00f2n ph\u1ee5 thu\u1ed9c ti\u00eau ch\u00ed b\u1ec7nh, \u0111i\u1ec1u tr\u1ecb tr\u01b0\u1edbc \u0111\u00f3, r\u1ee7i ro gh\u00e9p v\u00e0 ti\u1ebfp c\u1eadn trung t\u00e2m chuy\u00ean m\u00f4n.<\/p>\n<h2>\u0110i\u1ec1u g\u00ec thay \u0111\u1ed5i<\/h2>\n<p>FDA ban h\u00e0nh supplemental approval ng\u00e0y 1\/7\/2026. Trang s\u1ea3n ph\u1ea9m Casgevy c\u1ee7a FDA li\u1ec7t k\u00ea STN 125787 cho b\u1ec7nh nh\u00e2n t\u1eeb 2 tu\u1ed5i tr\u1edf l\u00ean c\u00f3 SCD v\u1edbi recurrent VOCs ho\u1eb7c TDT. FDA g\u1ecdi \u0111\u00e2y l\u00e0 gene therapy \u0111\u1ea7u ti\u00ean \u0111\u01b0\u1ee3c ph\u00ea duy\u1ec7t cho b\u1ec7nh nh\u00e2n t\u1eeb 2 tu\u1ed5i tr\u1edf l\u00ean v\u1edbi SCD. Vertex m\u00f4 t\u1ea3 \u0111\u00e2y l\u00e0 genetic therapy \u0111\u1ea7u ti\u00ean \u0111\u01b0\u1ee3c ch\u1ec9 \u0111\u1ecbnh cho tr\u1ebb t\u1eeb 2 tu\u1ed5i v\u1edbi c\u1ea3 SCD v\u00e0 TDT.<\/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-vi.png\" alt=\"H\u00ecnh gi\u1ea3i th\u00edch t\u01b0 v\u1ea5n huy\u1ebft h\u1ecdc nhi khoa v\u00e0 l\u1ed9 tr\u00ecnh \u0111i\u1ec1u tr\u1ecb t\u1ebf b\u00e0o g\u1ed1c CRISPR\" loading=\"eager\" \/><figcaption class=\"op-briefing-image-caption\">H\u00ecnh \u1ea3nh do ONEPRESS t\u1ea1o: minh h\u1ecda t\u01b0 v\u1ea5n huy\u1ebft h\u1ecdc nhi khoa v\u00e0 l\u1ed9 tr\u00ecnh t\u1ebf b\u00e0o g\u1ed1c t\u1ef1 th\u00e2n ch\u1ec9nh s\u1eeda CRISPR. \u0110\u00e2y kh\u00f4ng ph\u1ea3i c\u1ea3nh \u0111i\u1ec1u tr\u1ecb th\u1eadt hay qu\u1ea3ng b\u00e1 s\u1ea3n ph\u1ea9m.<\/figcaption><\/figure>\n<h2>Li\u1ec7u ph\u00e1p l\u00e0 g\u00ec<\/h2>\n<p>Casgevy d\u00f9ng hematopoietic stem cells c\u1ee7a ch\u00ednh b\u1ec7nh nh\u00e2n, ch\u1ec9nh s\u1eeda b\u1eb1ng CRISPR\/Cas9 r\u1ed3i truy\u1ec1n l\u1ea1i m\u1ed9t l\u1ea7n qua \u0111\u01b0\u1eddng t\u0129nh m\u1ea1ch. FDA n\u00f3i c\u00e1c t\u1ebf b\u00e0o \u0111\u00e3 ch\u1ec9nh s\u1eeda s\u1ebd b\u00e1m v\u00e0o t\u1ee7y x\u01b0\u01a1ng. V\u00ec v\u1eady \u0111\u00e2y kh\u00f4ng ph\u1ea3i vi\u00ean thu\u1ed1c hay m\u0169i ti\u00eam ngo\u1ea1i tr\u00fa th\u00f4ng th\u01b0\u1eddng; qu\u00e1 tr\u00ecnh g\u1ed3m l\u1ea5y t\u1ebf b\u00e0o, conditioning, l\u1eadp k\u1ebf ho\u1ea1ch ch\u0103m s\u00f3c chuy\u00ean s\u00e2u v\u00e0 theo d\u00f5i d\u00e0i h\u1ea1n.<\/p>\n<h2>Gi\u1edbi h\u1ea1n v\u00e0 l\u01b0u \u00fd<\/h2>\n<p>\u0110\u00e2y l\u00e0 quy\u1ebft \u0111\u1ecbnh c\u1ee7a Hoa K\u1ef3, tu\u1ed5i v\u00e0 ch\u1ec9 \u0111\u1ecbnh c\u00f3 th\u1ec3 kh\u00e1c \u1edf n\u01b0\u1edbc kh\u00e1c. N\u00f3 c\u0169ng kh\u00f4ng c\u00f3 ngh\u0129a m\u1ecdi tr\u1ebb m\u1eafc SCD ho\u1eb7c TDT \u0111\u1ec1u \u0111\u1ee7 \u0111i\u1ec1u ki\u1ec7n. Gia \u0111\u00ecnh c\u1ea7n \u0111\u00e1nh gi\u00e1 chuy\u00ean khoa v\u1ec1 m\u1ee9c \u0111\u1ed9 b\u1ec7nh, \u0111i\u1ec1u tr\u1ecb tr\u01b0\u1edbc \u0111\u00f3, r\u1ee7i ro nhi\u1ec5m tr\u00f9ng v\u00e0 gh\u00e9p, t\u01b0 v\u1ea5n b\u1ea3o t\u1ed3n kh\u1ea3 n\u0103ng sinh s\u1ea3n v\u00e0 k\u1ebf ho\u1ea1ch theo d\u00f5i. T\u00e0i li\u1ec7u s\u1ea3n ph\u1ea9m v\u00e0 th\u01b0 ph\u00ea duy\u1ec7t c\u1ee7a FDA l\u00e0 \u0111i\u1ec3m kh\u1edfi \u0111\u1ea7u.<\/p>\n<h2>Ngu\u1ed3n ki\u1ec3m tra<\/h2>\n<p>Th\u00f4ng c\u00e1o FDA x\u00e1c nh\u1eadn ng\u00e0y ph\u00ea duy\u1ec7t, m\u1edf r\u1ed9ng \u0111\u1ed9 tu\u1ed5i v\u00e0 ph\u1ea1m vi gene therapy \u0111\u1ea7u ti\u00ean cho SCD tr\u1ebb nh\u1ecf. Trang Casgevy c\u1ee7a FDA cho bi\u1ebft ch\u1ec9 \u0111\u1ecbnh STN 125787, package insert v\u00e0 th\u01b0 ph\u00ea duy\u1ec7t. Th\u00f4ng b\u00e1o Vertex gi\u00fap \u0111\u1ed1i chi\u1ebfu c\u00e1ch nh\u00e0 s\u1ea3n xu\u1ea5t m\u00f4 t\u1ea3 ch\u1ec9 \u0111\u1ecbnh m\u1edf r\u1ed9ng.<\/p>\n<h2>Ngu\u1ed3n ch\u00ednh th\u1ee9c<\/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: ng\u00e0y ph\u00ea duy\u1ec7t, m\u1edf r\u1ed9ng tu\u1ed5i v\u00e0 ph\u1ea1m vi gene therapy SCD nhi khoa<\/a><\/p>\n<p><a href=\"https:\/\/www.fda.gov\/vaccines-blood-biologics\/casgevy\" target=\"_blank\" rel=\"noopener noreferrer\">Trang FDA Casgevy: ch\u1ec9 \u0111\u1ecbnh STN 125787 v\u00e0 t\u00e0i li\u1ec7u ph\u00ea duy\u1ec7t<\/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: gi\u1ea3i th\u00edch c\u1ee7a nh\u00e0 s\u1ea3n xu\u1ea5t v\u1ec1 ch\u1ec9 \u0111\u1ecbnh m\u1edf r\u1ed9ng<\/a><\/p>\n","protected":false},"excerpt":{"rendered":"<p>FDA ban h\u00e0nh ph\u00ea duy\u1ec7t b\u1ed5 sung ng\u00e0y 1\/7\/2026 cho Casgevy (exagamglogene autotemcel) \u1edf m\u1ed9t s\u1ed1 b\u1ec7nh nh\u00e2n t\u1eeb 2 tu\u1ed5i tr\u1edf l\u00ean m\u1eafc sickle cell disease ho\u1eb7c transfusion-dependent beta thalassemia. Thay \u0111\u1ed5i ch\u00ednh l\u00e0 ch\u1ec9 \u0111\u1ecbnh t\u1ea1i M\u1ef9 c\u1ee7a li\u1ec7u ph\u00e1p gene CRISPR m\u1edf xu\u1ed1ng nh\u00f3m tr\u1ebb nh\u1ecf h\u01a1n, kh\u00f4ng ph\u1ea3i \u0111i\u1ec1u tr\u1ecb \u0111\u01a1n gi\u1ea3n m\u00e0 m\u1ecdi b\u1ec7nh nh\u00e2n nh\u1eadn ngay.<\/p>\n","protected":false},"featured_media":0,"template":"","meta":[],"class_list":["post-743","briefing","type-briefing","status-publish","hentry"],"_links":{"self":[{"href":"https:\/\/onepress.co.kr\/index.php\/wp-json\/wp\/v2\/briefing\/743","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=743"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}