{"id":9290,"date":"2025-12-16T15:52:06","date_gmt":"2025-12-16T08:52:06","guid":{"rendered":"https:\/\/biogroupvietnam.com\/?p=9290"},"modified":"2026-02-24T16:40:22","modified_gmt":"2026-02-24T09:40:22","slug":"fda-phe-duyet-waskyra-lieu-phap-gen-dau-tien-dieu-tri-wiskott-aldrich","status":"publish","type":"post","link":"https:\/\/biogroupvietnam.com\/en\/news\/fda-phe-duyet-waskyra-lieu-phap-gen-dau-tien-dieu-tri-wiskott-aldrich\/","title":{"rendered":"FDA ph\u00ea duy\u1ec7t Waskyra nh\u01b0 li\u1ec7u ph\u00e1p gen \u0111\u1ea7u ti\u00ean \u0111i\u1ec1u tr\u1ecb h\u1ed9i ch\u1ee9ng Wiskott\u2013Aldrich (WAS)"},"content":{"rendered":"\n<p>Ng\u00e0y 9\/12\/2025 \u0111\u00e1nh d\u1ea5u m\u1ed9t c\u1ed9t m\u1ed1c l\u1ecbch s\u1eed trong l\u0129nh v\u1ef1c li\u1ec7u ph\u00e1p gen: C\u1ee5c Qu\u1ea3n l\u00fd Th\u1ef1c ph\u1ea9m v\u00e0 D\u01b0\u1ee3c ph\u1ea9m Hoa K\u1ef3 (FDA) \u0111\u00e3 ch\u00ednh th\u1ee9c ph\u00ea duy\u1ec7t etuvetidigene autotemcel (Waskyra) \u2013 li\u1ec7u ph\u00e1p gen \u0111\u1ea7u ti\u00ean \u0111\u01b0\u1ee3c ch\u1ea5p thu\u1eadn \u0111\u1ec3 \u0111i\u1ec1u tr\u1ecb H\u1ed9i ch\u1ee9ng Wiskott-Aldrich (WAS).&nbsp;<\/p>\n\n\n\n<p>Waskyra, do Fondazione Telethon ETS ph\u00e1t tri\u1ec3n, \u0111\u01b0\u1ee3c ch\u1ec9 \u0111\u1ecbnh cho tr\u1ebb em t\u1eeb 6 th\u00e1ng tu\u1ed5i tr\u1edf l\u00ean v\u00e0 ng\u01b0\u1eddi l\u1edbn m\u1eafc \u0111\u1ed9t bi\u1ebfn gen WAS, nh\u1eefng ng\u01b0\u1eddi ph\u00f9 h\u1ee3p \u0111\u1ec3 gh\u00e9p t\u1ebf b\u00e0o g\u1ed1c t\u1ea1o m\u00e1u (HSCT) nh\u01b0ng kh\u00f4ng c\u00f3 ng\u01b0\u1eddi hi\u1ebfn ph\u00f9 h\u1ee3p HLA.&nbsp;<\/p>\n\n\n\n<p>S\u1ef1 ph\u00ea duy\u1ec7t n\u00e0y m\u1edf ra m\u1ed9t k\u1ef7 nguy\u00ean m\u1edbi cho c\u1ed9ng \u0111\u1ed3ng b\u1ec7nh WAS \u2013 m\u1ed9t r\u1ed1i lo\u1ea1n mi\u1ec5n d\u1ecbch hi\u1ebfm g\u1eb7p, di truy\u1ec1n l\u1eb7n li\u00ean k\u1ebft nhi\u1ec5m s\u1eafc th\u1ec3 X, v\u1ed1n ch\u1ec9 c\u00f3 c\u00e1c l\u1ef1a ch\u1ecdn \u0111i\u1ec1u tr\u1ecb r\u1ea5t h\u1ea1n ch\u1ebf.\u00a0<\/p>\n\n\n<div class=\"wp-block-image\">\n<figure class=\"aligncenter size-large\"><img fetchpriority=\"high\" decoding=\"async\" width=\"1024\" height=\"576\" src=\"https:\/\/biogroupvietnam.com\/wp-content\/uploads\/2025\/12\/FDA-phe-duyet-waskyra-lieu-phap-gen-dau-tien-dieu-tri-wiskott-aldrich-1-1024x576.webp\" alt=\"FDA ph\u00ea duy\u1ec7t Waskyra nh\u01b0 li\u1ec7u ph\u00e1p gen \u0111\u1ea7u ti\u00ean \u0111i\u1ec1u tr\u1ecb h\u1ed9i ch\u1ee9ng Wiskott\u2013Aldrich (WAS)\" class=\"wp-image-9307\" srcset=\"https:\/\/biogroupvietnam.com\/wp-content\/uploads\/2025\/12\/FDA-phe-duyet-waskyra-lieu-phap-gen-dau-tien-dieu-tri-wiskott-aldrich-1-1024x576.webp 1024w, https:\/\/biogroupvietnam.com\/wp-content\/uploads\/2025\/12\/FDA-phe-duyet-waskyra-lieu-phap-gen-dau-tien-dieu-tri-wiskott-aldrich-1-300x169.webp 300w, https:\/\/biogroupvietnam.com\/wp-content\/uploads\/2025\/12\/FDA-phe-duyet-waskyra-lieu-phap-gen-dau-tien-dieu-tri-wiskott-aldrich-1-768x432.webp 768w, https:\/\/biogroupvietnam.com\/wp-content\/uploads\/2025\/12\/FDA-phe-duyet-waskyra-lieu-phap-gen-dau-tien-dieu-tri-wiskott-aldrich-1-600x338.webp 600w, https:\/\/biogroupvietnam.com\/wp-content\/uploads\/2025\/12\/FDA-phe-duyet-waskyra-lieu-phap-gen-dau-tien-dieu-tri-wiskott-aldrich-1-90x51.webp 90w, https:\/\/biogroupvietnam.com\/wp-content\/uploads\/2025\/12\/FDA-phe-duyet-waskyra-lieu-phap-gen-dau-tien-dieu-tri-wiskott-aldrich-1.webp 1200w\" sizes=\"(max-width: 1024px) 100vw, 1024px\" \/><figcaption class=\"wp-element-caption\"><em>FDA ph\u00ea duy\u1ec7t Waskyra nh\u01b0 li\u1ec7u ph\u00e1p gen \u0111\u1ea7u ti\u00ean \u0111i\u1ec1u tr\u1ecb h\u1ed9i ch\u1ee9ng Wiskott\u2013Aldrich (WAS) (Ngu\u1ed3n: precisionformedicine.com)<\/em><\/figcaption><\/figure><\/div>\n\n\n<div id=\"ez-toc-container\" class=\"ez-toc-v2_0_80 counter-hierarchy ez-toc-counter ez-toc-grey ez-toc-container-direction\">\n<div class=\"ez-toc-title-container\">\n<p class=\"ez-toc-title\" style=\"cursor:inherit\">Contents<\/p>\n<span class=\"ez-toc-title-toggle\"><a href=\"#\" class=\"ez-toc-pull-right ez-toc-btn ez-toc-btn-xs ez-toc-btn-default ez-toc-toggle\" aria-label=\"Toggle Table of Content\"><span class=\"ez-toc-js-icon-con\"><span class=\"\"><span class=\"eztoc-hide\" style=\"display:none;\">Toggle<\/span><span class=\"ez-toc-icon-toggle-span\"><svg style=\"fill: #999;color:#999\" xmlns=\"http:\/\/www.w3.org\/2000\/svg\" class=\"list-377408\" width=\"20px\" height=\"20px\" viewBox=\"0 0 24 24\" fill=\"none\"><path d=\"M6 6H4v2h2V6zm14 0H8v2h12V6zM4 11h2v2H4v-2zm16 0H8v2h12v-2zM4 16h2v2H4v-2zm16 0H8v2h12v-2z\" fill=\"currentColor\"><\/path><\/svg><svg style=\"fill: #999;color:#999\" class=\"arrow-unsorted-368013\" xmlns=\"http:\/\/www.w3.org\/2000\/svg\" width=\"10px\" height=\"10px\" viewBox=\"0 0 24 24\" version=\"1.2\" baseProfile=\"tiny\"><path d=\"M18.2 9.3l-6.2-6.3-6.2 6.3c-.2.2-.3.4-.3.7s.1.5.3.7c.2.2.4.3.7.3h11c.3 0 .5-.1.7-.3.2-.2.3-.5.3-.7s-.1-.5-.3-.7zM5.8 14.7l6.2 6.3 6.2-6.3c.2-.2.3-.5.3-.7s-.1-.5-.3-.7c-.2-.2-.4-.3-.7-.3h-11c-.3 0-.5.1-.7.3-.2.2-.3.5-.3.7s.1.5.3.7z\"\/><\/svg><\/span><\/span><\/span><\/a><\/span><\/div>\n<nav><ul class='ez-toc-list ez-toc-list-level-1 ' ><li class='ez-toc-page-1 ez-toc-heading-level-2'><a class=\"ez-toc-link ez-toc-heading-1\" href=\"https:\/\/biogroupvietnam.com\/en\/news\/fda-phe-duyet-waskyra-lieu-phap-gen-dau-tien-dieu-tri-wiskott-aldrich\/#Hoi_chung_Wiskott-Aldrich_Mot_benh_ly_hiem_nhung_nguy_hiem\" >H\u1ed9i ch\u1ee9ng Wiskott-Aldrich: M\u1ed9t b\u1ec7nh l\u00fd hi\u1ebfm nh\u01b0ng nguy hi\u1ec3m&nbsp;<\/a><\/li><li class='ez-toc-page-1 ez-toc-heading-level-2'><a class=\"ez-toc-link ez-toc-heading-2\" href=\"https:\/\/biogroupvietnam.com\/en\/news\/fda-phe-duyet-waskyra-lieu-phap-gen-dau-tien-dieu-tri-wiskott-aldrich\/#Waskyra_%E2%80%93_Buoc_dot_pha_cua_y_hoc_hien_dai\" >Waskyra \u2013 B\u01b0\u1edbc \u0111\u1ed9t ph\u00e1 c\u1ee7a y h\u1ecdc hi\u1ec7n \u0111\u1ea1i&nbsp;<\/a><\/li><li class='ez-toc-page-1 ez-toc-heading-level-2'><a class=\"ez-toc-link ez-toc-heading-3\" href=\"https:\/\/biogroupvietnam.com\/en\/news\/fda-phe-duyet-waskyra-lieu-phap-gen-dau-tien-dieu-tri-wiskott-aldrich\/#Ket_qua_lam_sang_cua_lieu_phap_gen_Waskyra_Hieu_qua_manh_me_va_ben_vung\" >K\u1ebft qu\u1ea3 l\u00e2m s\u00e0ng c\u1ee7a li\u1ec7u ph\u00e1p gen Waskyra : Hi\u1ec7u qu\u1ea3 m\u1ea1nh m\u1ebd v\u00e0 b\u1ec1n v\u1eefng&nbsp;<\/a><\/li><li class='ez-toc-page-1 ez-toc-heading-level-2'><a class=\"ez-toc-link ez-toc-heading-4\" href=\"https:\/\/biogroupvietnam.com\/en\/news\/fda-phe-duyet-waskyra-lieu-phap-gen-dau-tien-dieu-tri-wiskott-aldrich\/#Tinh_linh_hoat_trong_phe_duyet_cua_FDA_Bai_hoc_quan_trong_cho_nganh_lieu_phap_gen\" >T\u00ednh linh ho\u1ea1t trong ph\u00ea duy\u1ec7t c\u1ee7a FDA: B\u00e0i h\u1ecdc quan tr\u1ecdng cho ng\u00e0nh li\u1ec7u ph\u00e1p gen&nbsp;<\/a><\/li><li class='ez-toc-page-1 ez-toc-heading-level-2'><a class=\"ez-toc-link ez-toc-heading-5\" href=\"https:\/\/biogroupvietnam.com\/en\/news\/fda-phe-duyet-waskyra-lieu-phap-gen-dau-tien-dieu-tri-wiskott-aldrich\/#Y_nghia_cua_Waskyra_doi_voi_benh_nhan_WAS_va_cong_dong_y_khoa\" >\u00dd ngh\u0129a c\u1ee7a Waskyra \u0111\u1ed1i v\u1edbi b\u1ec7nh nh\u00e2n WAS v\u00e0 c\u1ed9ng \u0111\u1ed3ng y khoa&nbsp;<\/a><\/li><li class='ez-toc-page-1 ez-toc-heading-level-2'><a class=\"ez-toc-link ez-toc-heading-6\" href=\"https:\/\/biogroupvietnam.com\/en\/news\/fda-phe-duyet-waskyra-lieu-phap-gen-dau-tien-dieu-tri-wiskott-aldrich\/#Nhung_giai_phap_ma_Biogroup_cung_cap_cho_lieu_phap_te_bao_%E2%80%93_lieu_phap_gen\" >Nh\u1eefng gi\u1ea3i ph\u00e1p m\u00e0 Biogroup cung c\u1ea5p cho li\u1ec7u ph\u00e1p t\u1ebf b\u00e0o \u2013 li\u1ec7u ph\u00e1p gen&nbsp;&nbsp;<\/a><\/li><li class='ez-toc-page-1 ez-toc-heading-level-2'><a class=\"ez-toc-link ez-toc-heading-7\" href=\"https:\/\/biogroupvietnam.com\/en\/news\/fda-phe-duyet-waskyra-lieu-phap-gen-dau-tien-dieu-tri-wiskott-aldrich\/#Ket_luan\" >K\u1ebft lu\u1eadn&nbsp;<\/a><\/li><li class='ez-toc-page-1 ez-toc-heading-level-2'><a class=\"ez-toc-link ez-toc-heading-8\" href=\"https:\/\/biogroupvietnam.com\/en\/news\/fda-phe-duyet-waskyra-lieu-phap-gen-dau-tien-dieu-tri-wiskott-aldrich\/#Tai_lieu_tham_khao\" >T\u00e0i li\u1ec7u tham kh\u1ea3o&nbsp;<\/a><\/li><\/ul><\/nav><\/div>\n<h2 class=\"wp-block-heading\"><span class=\"ez-toc-section\" id=\"Hoi_chung_Wiskott-Aldrich_Mot_benh_ly_hiem_nhung_nguy_hiem\"><\/span><strong>H\u1ed9i ch\u1ee9ng Wiskott-Aldrich: M\u1ed9t b\u1ec7nh l\u00fd hi\u1ebfm nh\u01b0ng nguy hi\u1ec3m&nbsp;<\/strong><span class=\"ez-toc-section-end\"><\/span><\/h2>\n\n\n\n<p><strong>H\u1ed9i ch\u1ee9ng Wiskott\u2013Aldrich (WAS)<\/strong> l\u00e0 m\u1ed9t r\u1ed1i lo\u1ea1n di truy\u1ec1n hi\u1ebfm g\u1eb7p, h\u1ea7u nh\u01b0 ch\u1ec9 x\u1ea3y ra \u1edf nam gi\u1edbi, g\u00e2y t\u1ed5n h\u1ea1i nghi\u00eam tr\u1ecdng \u0111\u1ebfn c\u1ea3 h\u1ec7 m\u00e1u v\u00e0 h\u1ec7 mi\u1ec5n d\u1ecbch. C\u0103n b\u1ec7nh b\u1eaft ngu\u1ed3n t\u1eeb c\u00e1c \u0111\u1ed9t bi\u1ebfn tr\u00ean gen m\u00e3 h\u00f3a protein WAS\u2014m\u1ed9t th\u00e0nh ph\u1ea7n thi\u1ebft y\u1ebfu c\u00f3 m\u1eb7t trong t\u1ebf b\u00e0o m\u00e1u v\u00e0 t\u1ebf b\u00e0o mi\u1ec5n d\u1ecbch. Khi thi\u1ebfu protein WAS ch\u1ee9c n\u0103ng, c\u00e1c t\u1ebf b\u00e0o mi\u1ec5n d\u1ecbch v\u00e0 t\u1ebf b\u00e0o m\u00e1u c\u1ee7a b\u1ec7nh nh\u00e2n ph\u00e1t tri\u1ec3n b\u1ea5t th\u01b0\u1eddng, d\u1eabn \u0111\u1ebfn h\u00e0ng lo\u1ea1t bi\u1ebfn ch\u1ee9ng \u0111e d\u1ecda t\u00ednh m\u1ea1ng nh\u01b0 ch\u1ea3y m\u00e1u kh\u00f4ng ki\u1ec3m so\u00e1t, nhi\u1ec5m tr\u00f9ng nghi\u00eam tr\u1ecdng v\u00e0 t\u0103ng nguy c\u01a1 ung th\u01b0.&nbsp;<\/p>\n\n\n<div class=\"wp-block-image\">\n<figure class=\"aligncenter size-full is-resized\"><img decoding=\"async\" width=\"640\" height=\"640\" src=\"https:\/\/biogroupvietnam.com\/wp-content\/uploads\/2025\/12\/hoi-chung-wiskott-aldrich.webp\" alt=\"Minh h\u1ecda b\u1ec7nh nh\u00e2n H\u1ed9i ch\u1ee9ng Wiskott-Aldrich \" class=\"wp-image-9305\" style=\"width:384px;height:auto\" srcset=\"https:\/\/biogroupvietnam.com\/wp-content\/uploads\/2025\/12\/hoi-chung-wiskott-aldrich.webp 640w, https:\/\/biogroupvietnam.com\/wp-content\/uploads\/2025\/12\/hoi-chung-wiskott-aldrich-300x300.webp 300w, https:\/\/biogroupvietnam.com\/wp-content\/uploads\/2025\/12\/hoi-chung-wiskott-aldrich-150x150.webp 150w, https:\/\/biogroupvietnam.com\/wp-content\/uploads\/2025\/12\/hoi-chung-wiskott-aldrich-450x450.webp 450w, https:\/\/biogroupvietnam.com\/wp-content\/uploads\/2025\/12\/hoi-chung-wiskott-aldrich-500x500.webp 500w, https:\/\/biogroupvietnam.com\/wp-content\/uploads\/2025\/12\/hoi-chung-wiskott-aldrich-600x600.webp 600w, https:\/\/biogroupvietnam.com\/wp-content\/uploads\/2025\/12\/hoi-chung-wiskott-aldrich-54x54.webp 54w\" sizes=\"(max-width: 640px) 100vw, 640px\" \/><figcaption class=\"wp-element-caption\"><em>Minh h\u1ecda b\u1ec7nh nh\u00e2n H\u1ed9i ch\u1ee9ng Wiskott-Aldrich (Ngu\u1ed3n: Tr\u1ea1m Y t\u1ebf ph\u01b0\u1eddng Linh T\u00e2y)<\/em><\/figcaption><\/figure><\/div>\n\n\n<p class=\"has-text-align-left\">B\u1ec7nh nh\u00e2n WAS th\u01b0\u1eddng d\u1ec5 b\u1ea7m t\u00edm v\u00e0 ch\u1ea3y m\u00e1u do s\u1ed1 l\u01b0\u1ee3ng ti\u1ec3u c\u1ea7u gi\u1ea3m nghi\u00eam tr\u1ecdng, \u0111\u1ed3ng th\u1eddi ph\u1ea3i \u0111\u1ed1i m\u1eb7t v\u1edbi c\u00e1c \u0111\u1ee3t nhi\u1ec5m tr\u00f9ng t\u00e1i ph\u00e1t c\u00f3 th\u1ec3 ti\u1ebfn tri\u1ec3n th\u00e0nh nhi\u1ec5m tr\u00f9ng huy\u1ebft\u2014m\u1ed9t t\u00ecnh tr\u1ea1ng nguy hi\u1ec3m khi \u0111\u1ed9c t\u1ed1 vi khu\u1ea9n g\u00e2y t\u1ed5n th\u01b0\u01a1ng c\u00e1c c\u01a1 quan quan tr\u1ecdng. Ngo\u00e0i ra, nh\u1eefng b\u1ec7nh nh\u00e2n n\u00e0y c\u00f2n \u0111\u1ed1i di\u1ec7n nguy c\u01a1 cao m\u1eafc u lympho v\u00e0 c\u00e1c lo\u1ea1i ung th\u01b0 kh\u00e1c.\u00a0<\/p>\n\n\n\n<p>Gh\u00e9p t\u1ebf b\u00e0o g\u1ed1c t\u1ea1o m\u00e1u (HSCT) v\u1eabn l\u00e0 ph\u01b0\u01a1ng ph\u00e1p \u0111i\u1ec1u tr\u1ecb t\u1ed1i \u01b0u, nh\u01b0ng t\u1ef7 l\u1ec7 th\u00e0nh c\u00f4ng cao nh\u1ea5t ch\u1ec9 khi b\u1ec7nh nh\u00e2n c\u00f3 ng\u01b0\u1eddi hi\u1ebfn ph\u00f9 h\u1ee3p HLA ho\u00e0n to\u00e0n, \u0111i\u1ec1u ch\u1ec9 x\u1ea3y ra \u1edf thi\u1ec3u s\u1ed1 b\u1ec7nh nh\u00e2n.&nbsp;<\/p>\n\n\n\n<p>V\u00ec th\u1ebf, s\u1ef1 xu\u1ea5t hi\u1ec7n c\u1ee7a Waskyra \u0111\u01b0\u1ee3c v\u00ed nh\u01b0 \u201c\u00e1nh s\u00e1ng hy v\u1ecdng\u201d cho nh\u1eefng b\u1ec7nh nh\u00e2n thi\u1ebfu ngu\u1ed3n hi\u1ebfn ph\u00f9 h\u1ee3p.&nbsp;<\/p>\n\n\n\n<h2 class=\"wp-block-heading\"><span class=\"ez-toc-section\" id=\"Waskyra_%E2%80%93_Buoc_dot_pha_cua_y_hoc_hien_dai\"><\/span><strong>Waskyra \u2013 B\u01b0\u1edbc \u0111\u1ed9t ph\u00e1 c\u1ee7a y h\u1ecdc hi\u1ec7n \u0111\u1ea1i&nbsp;<\/strong><span class=\"ez-toc-section-end\"><\/span><\/h2>\n\n\n\n<p>Theo FDA, Waskyra \u0111\u1ea1i di\u1ec7n cho m\u1ed9t b\u01b0\u1edbc ti\u1ebfn mang t\u00ednh bi\u1ebfn \u0111\u1ed5i trong \u0111i\u1ec1u tr\u1ecb WAS. Waskyra s\u1eed d\u1ee5ng c\u00f4ng ngh\u1ec7 li\u1ec7u ph\u00e1p gen ti\u00ean ti\u1ebfn \u0111\u1ec3 gi\u1ea3i quy\u1ebft nhu c\u1ea7u \u0111i\u1ec1u tr\u1ecb c\u00f2n b\u1ecf ng\u1ecf n\u00e0y. Trong quy tr\u00ecnh \u0111i\u1ec1u tr\u1ecb, c\u00e1c t\u1ebf b\u00e0o g\u1ed1c CD34+ \u0111\u01b0\u1ee3c thu th\u1eadp tr\u1ef1c ti\u1ebfp t\u1eeb m\u00e1u c\u1ee7a ch\u00ednh b\u1ec7nh nh\u00e2n, sau \u0111\u00f3 \u0111\u01b0\u1ee3c ch\u1ec9nh s\u1eeda gen trong ph\u00f2ng th\u00ed nghi\u1ec7m \u0111\u1ec3 t\u1ea1o ra protein WAS ch\u1ee9c n\u0103ng. Sau khi b\u1ec7nh nh\u00e2n tr\u1ea3i qua ph\u00e1c \u0111\u1ed3 \u0111i\u1ec1u h\u00f2a (conditioning) chu\u1ea9n b\u1ecb, c\u00e1c t\u1ebf b\u00e0o \u0111\u00e3 \u0111\u01b0\u1ee3c ch\u1ec9nh s\u1eeda s\u1ebd \u0111\u01b0\u1ee3c truy\u1ec1n tr\u1edf l\u1ea1i c\u01a1 th\u1ec3 qua \u0111\u01b0\u1eddng t\u0129nh m\u1ea1ch.&nbsp;<\/p>\n\n\n\n<p>Nh\u1eefng t\u1ebf b\u00e0o n\u00e0y sau \u0111\u00f3 di chuy\u1ec3n t\u1edbi t\u1ee7y x\u01b0\u01a1ng, n\u01a1i ch\u00fang \u0111\u1ecbnh c\u01b0 v\u00e0 b\u1eaft \u0111\u1ea7u t\u1ea1o ra c\u00e1c t\u1ebf b\u00e0o m\u00e1u v\u00e0 t\u1ebf b\u00e0o mi\u1ec5n d\u1ecbch kh\u1ecfe m\u1ea1nh c\u00f3 kh\u1ea3 n\u0103ng s\u1ea3n xu\u1ea5t protein WAS b\u00ecnh th\u01b0\u1eddng. \u0110\u00e1ng ch\u00fa \u00fd, Waskyra ch\u1ec9 c\u1ea7n truy\u1ec1n m\u1ed9t l\u1ea7n duy nh\u1ea5t, mang l\u1ea1i s\u1ef1 thu\u1eadn ti\u1ec7n v\u01b0\u1ee3t tr\u1ed9i nh\u01b0 m\u1ed9t ph\u01b0\u01a1ng ph\u00e1p \u0111i\u1ec1u tr\u1ecb m\u1ed9t l\u1ea7n cho k\u1ebft qu\u1ea3 l\u00e2u d\u00e0i.&nbsp;<\/p>\n\n\n\n<p>\u0110\u00e2y l\u00e0 m\u1ed9t d\u1ea1ng li\u1ec7u ph\u00e1p gen t\u1ef1 th\u00e2n (autologous gene therapy), lo\u1ea1i b\u1ecf r\u1ee7i ro th\u1ea3i gh\u00e9p ho\u1eb7c b\u1ec7nh gh\u00e9p ch\u1ed1ng ch\u1ee7 (GVHD).&nbsp;<\/p>\n\n\n\n<h2 class=\"wp-block-heading\"><span class=\"ez-toc-section\" id=\"Ket_qua_lam_sang_cua_lieu_phap_gen_Waskyra_Hieu_qua_manh_me_va_ben_vung\"><\/span><strong>K\u1ebft qu\u1ea3 l\u00e2m s\u00e0ng c\u1ee7a li\u1ec7u ph\u00e1p gen Waskyra : Hi\u1ec7u qu\u1ea3 m\u1ea1nh m\u1ebd v\u00e0 b\u1ec1n v\u1eefng&nbsp;<\/strong><span class=\"ez-toc-section-end\"><\/span><\/h2>\n\n\n\n<p>Waskyra \u0111\u01b0\u1ee3c \u0111\u00e1nh gi\u00e1 tr\u00ean 27 b\u1ec7nh nh\u00e2n trong:&nbsp;<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>2 nghi\u00ean c\u1ee9u m\u1edf nh\u00e3n, \u0111\u01a1n nh\u00e1nh, \u0111a trung t\u00e2m&nbsp;<\/li>\n\n\n\n<li>1 ch\u01b0\u01a1ng tr\u00ecnh expanded access&nbsp;<\/li>\n<\/ul>\n\n\n\n<p>K\u1ebft qu\u1ea3 r\u1ea5t \u1ea5n t\u01b0\u1ee3ng:&nbsp;<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Gi\u1ea3m 93% nhi\u1ec5m tr\u00f9ng n\u1eb7ng&nbsp;<\/li>\n\n\n\n<li>Trong giai \u0111o\u1ea1n 6\u201318 th\u00e1ng sau truy\u1ec1n t\u1ebf b\u00e0o, t\u1ef7 l\u1ec7 nhi\u1ec5m tr\u00f9ng n\u1eb7ng gi\u1ea3m g\u1ea7n ho\u00e0n to\u00e0n so v\u1edbi 12 th\u00e1ng tr\u01b0\u1edbc \u0111i\u1ec1u tr\u1ecb.&nbsp;<\/li>\n\n\n\n<li>Gi\u1ea3m 60% ch\u1ea3y m\u00e1u m\u1ee9c \u0111\u1ed9 trung b\u00ecnh\u2013n\u1eb7ng&nbsp;<\/li>\n\n\n\n<li>Trong n\u0103m \u0111\u1ea7u sau \u0111i\u1ec1u tr\u1ecb, ph\u1ea7n l\u1edbn b\u1ec7nh nh\u00e2n kh\u00f4ng c\u00f2n g\u1eb7p c\u00e1c bi\u1ebfn c\u1ed1 xu\u1ea5t huy\u1ebft nguy hi\u1ec3m.&nbsp;<\/li>\n\n\n\n<li>Hi\u1ec7u qu\u1ea3 duy tr\u00ec t\u1edbi 4 n\u0103m&nbsp;<\/li>\n\n\n\n<li>\u0110a s\u1ed1 b\u1ec7nh nh\u00e2n v\u1eabn kh\u00f4ng c\u00f3 bi\u1ebfn c\u1ed1 ch\u1ea3y m\u00e1u m\u1ee9c \u0111\u1ed9 trung b\u00ecnh\u2013n\u1eb7ng, cho th\u1ea5y t\u00e1c d\u1ee5ng \u1ed5n \u0111\u1ecbnh v\u00e0 d\u00e0i h\u1ea1n.&nbsp;<\/li>\n\n\n\n<li>\u0110\u1ed9 an to\u00e0n ch\u1ea5p nh\u1eadn \u0111\u01b0\u1ee3c&nbsp;<\/li>\n<\/ul>\n\n\n\n<p>C\u00e1c t\u00e1c d\u1ee5ng ph\u1ee5 ph\u1ed5 bi\u1ebfn g\u1ed3m:&nbsp;<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>S\u1ed1t gi\u1ea3m b\u1ea1ch c\u1ea7u&nbsp;<\/li>\n\n\n\n<li>Nhi\u1ec5m tr\u00f9ng \u0111\u01b0\u1eddng h\u00f4 h\u1ea5p&nbsp;<\/li>\n\n\n\n<li>T\u1ed5n th\u01b0\u01a1ng gan&nbsp;<\/li>\n\n\n\n<li>N\u00f4n, ti\u00eau ch\u1ea3y&nbsp;<\/li>\n\n\n\n<li>Nhi\u1ec5m tr\u00f9ng li\u00ean quan catheter&nbsp;<\/li>\n<\/ul>\n\n\n\n<p>Kh\u00f4ng xu\u1ea5t hi\u1ec7n t\u00edn hi\u1ec7u an to\u00e0n b\u1ea5t th\u01b0\u1eddng, v\u00e0 l\u1ee3i \u00edch v\u01b0\u1ee3t tr\u1ed9i so v\u1edbi nguy c\u01a1 c\u1ee7a b\u1ec7nh WAS \u2013 m\u1ed9t b\u1ec7nh l\u00fd c\u00f3 th\u1ec3 \u0111e d\u1ecda t\u00ednh m\u1ea1ng.&nbsp;<\/p>\n\n\n\n<h2 class=\"wp-block-heading\"><span class=\"ez-toc-section\" id=\"Tinh_linh_hoat_trong_phe_duyet_cua_FDA_Bai_hoc_quan_trong_cho_nganh_lieu_phap_gen\"><\/span><strong>T\u00ednh linh ho\u1ea1t trong ph\u00ea duy\u1ec7t c\u1ee7a FDA: B\u00e0i h\u1ecdc quan tr\u1ecdng cho ng\u00e0nh li\u1ec7u ph\u00e1p gen&nbsp;<\/strong><span class=\"ez-toc-section-end\"><\/span><\/h2>\n\n\n\n<p>Do WAS l\u00e0 b\u1ec7nh hi\u1ebfm, FDA \u0111\u00e3 \u00e1p d\u1ee5ng c\u00e1ch ti\u1ebfp c\u1eadn linh ho\u1ea1t \u0111\u1ec3 \u0111\u1ea9y nhanh qu\u00e1 tr\u00ecnh ph\u00ea duy\u1ec7t nh\u01b0ng v\u1eabn \u0111\u1ea3m b\u1ea3o t\u00ednh khoa h\u1ecdc. Trong \u0111\u00f3, 04 \u0111i\u1ec3m linh ho\u1ea1t ch\u00ednh:&nbsp;<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Hi\u1ebfm b\u1ec7nh \u2013 ch\u1ea5p nh\u1eadn d\u1eef li\u1ec7u t\u1eeb expanded access: Thay v\u00ec ch\u1ec9 d\u1ef1a v\u00e0o RCT (r\u1ea5t kh\u00f3 th\u1ef1c hi\u1ec7n), FDA cho ph\u00e9p d\u00f9ng d\u1eef li\u1ec7u h\u1ed7 tr\u1ee3 t\u1eeb b\u1ec7nh nh\u00e2n \u0111\u01b0\u1ee3c ti\u1ebfp c\u1eadn s\u1edbm.&nbsp;<\/li>\n\n\n\n<li>Thi\u1ebft k\u1ebf th\u1eed nghi\u1ec7m \u0111\u01a1n nh\u00e1nh: Kh\u00f4ng y\u00eau c\u1ea7u nh\u00f3m \u0111\u1ed1i ch\u1ee9ng do t\u00ednh c\u1ea5p b\u00e1ch v\u00e0 s\u1ed1 l\u01b0\u1ee3ng b\u1ec7nh nh\u00e2n th\u1ea5p.&nbsp;<\/li>\n\n\n\n<li>C\u01a1 ch\u1ebf t\u00e1c d\u1ee5ng r\u00f5 r\u00e0ng: D\u1ef1a tr\u00ean sinh h\u1ecdc ph\u00e2n t\u1eed c\u1ee7a gen WAS v\u00e0 vector virus \u0111\u00e3 \u0111\u01b0\u1ee3c ch\u1ee9ng minh \u1edf c\u00e1c s\u1ea3n ph\u1ea9m t\u01b0\u01a1ng t\u1ef1.&nbsp;<\/li>\n\n\n\n<li>Linh ho\u1ea1t trong h\u1ed3 s\u01a1 CMC: Ch\u1ea5p nh\u1eadn d\u1eef li\u1ec7u so s\u00e1nh t\u1eeb m\u1ed9t li\u1ec7u ph\u00e1p gen \u0111\u00e3 \u0111\u01b0\u1ee3c ph\u00ea duy\u1ec7t tr\u01b0\u1edbc \u0111\u00f3 s\u1eed d\u1ee5ng c\u00f4ng ngh\u1ec7 t\u01b0\u01a1ng \u0111\u1ed3ng.&nbsp;<\/li>\n<\/ul>\n\n\n\n<p>\u0110i\u1ec1u n\u00e0y cho th\u1ea5y FDA \u0111ang \u1ee7ng h\u1ed9 m\u1ea1nh m\u1ebd ng\u00e0nh li\u1ec7u ph\u00e1p gen, \u0111\u1eb7c bi\u1ec7t trong \u0111i\u1ec1u tr\u1ecb b\u1ec7nh hi\u1ebfm.&nbsp;<\/p>\n\n\n\n<h2 class=\"wp-block-heading\"><span class=\"ez-toc-section\" id=\"Y_nghia_cua_Waskyra_doi_voi_benh_nhan_WAS_va_cong_dong_y_khoa\"><\/span><strong>\u00dd ngh\u0129a c\u1ee7a Waskyra \u0111\u1ed1i v\u1edbi b\u1ec7nh nh\u00e2n WAS v\u00e0 c\u1ed9ng \u0111\u1ed3ng y khoa&nbsp;<\/strong><span class=\"ez-toc-section-end\"><\/span><\/h2>\n\n\n\n<p>Vi\u1ec7c Waskyra \u0111\u01b0\u1ee3c ph\u00ea duy\u1ec7t mang \u00fd ngh\u0129a \u0111\u1eb7c bi\u1ec7t quan tr\u1ecdng \u0111\u1ed1i v\u1edbi b\u1ec7nh nh\u00e2n m\u1eafc h\u1ed9i ch\u1ee9ng Wiskott\u2013Aldrich v\u00e0 c\u1ed9ng \u0111\u1ed3ng y khoa. L\u1ea7n \u0111\u1ea7u ti\u00ean, ng\u01b0\u1eddi b\u1ec7nh c\u00f3 m\u1ed9t li\u1ec7u ph\u00e1p gen \u0111\u01b0\u1ee3c c\u00f4ng nh\u1eadn ch\u00ednh th\u1ee9c, m\u1edf ra c\u01a1 h\u1ed9i \u0111i\u1ec1u tr\u1ecb mang t\u00ednh b\u01b0\u1edbc ngo\u1eb7t gi\u00fap h\u1ecd kh\u00f4ng c\u00f2n ph\u1ee5 thu\u1ed9c v\u00e0o c\u00e1c bi\u1ec7n ph\u00e1p h\u1ed7 tr\u1ee3 su\u1ed1t \u0111\u1eddi, kh\u00f4ng ph\u1ea3i ti\u1ebfp t\u1ee5c t\u00ecm ki\u1ebfm ng\u01b0\u1eddi hi\u1ebfn t\u1ebf b\u00e0o g\u1ed1c ph\u00f9 h\u1ee3p hay \u0111\u1ed1i m\u1eb7t v\u1edbi nguy c\u01a1 th\u1ea3i gh\u00e9p v\u00e0 bi\u1ebfn ch\u1ee9ng GVHD nh\u01b0 tr\u01b0\u1edbc \u0111\u00e2y. Nhi\u1ec1u b\u1ec7nh nh\u00e2n v\u00e0 gia \u0111\u00ecnh m\u00f4 t\u1ea3 cu\u1ed9c s\u1ed1ng tr\u01b0\u1edbc \u0111i\u1ec1u tr\u1ecb nh\u01b0 \u201cm\u1ed9t chu\u1ed7i ng\u00e0y \u0111\u1ea7y lo l\u1eafng v\u00e0 b\u1ea5t an\u201d, b\u1edfi h\u1ecd lu\u00f4n ph\u1ea3i s\u1ed1ng trong n\u1ed7i s\u1ee3 tr\u01b0\u1edbc nguy c\u01a1 ch\u1ea3y m\u00e1u, nhi\u1ec5m tr\u00f9ng v\u00e0 c\u00e1c bi\u1ebfn ch\u1ee9ng nguy hi\u1ec3m kh\u00e1c. S\u1ef1 xu\u1ea5t hi\u1ec7n c\u1ee7a Waskyra mang l\u1ea1i hy v\u1ecdng th\u1ef1c s\u1ef1, gi\u00fap h\u1ecd c\u00f3 th\u1ec3 quay tr\u1edf l\u1ea1i nh\u1eefng sinh ho\u1ea1t b\u00ecnh th\u01b0\u1eddng nh\u01b0 \u0111\u1ebfn tr\u01b0\u1eddng, tham gia th\u1ec3 thao hay \u0111\u01a1n gi\u1ea3n l\u00e0 t\u1eadn h\u01b0\u1edfng m\u1ed9t cu\u1ed9c s\u1ed1ng \u00edt r\u1ee7i ro h\u01a1n.&nbsp;<\/p>\n\n\n\n<p>Kh\u00f4ng ch\u1ec9 c\u1ea3i thi\u1ec7n ch\u1ea5t l\u01b0\u1ee3ng s\u1ed1ng cho b\u1ec7nh nh\u00e2n, Waskyra c\u00f2n \u0111\u00e1nh d\u1ea5u b\u01b0\u1edbc ti\u1ebfn quan tr\u1ecdng trong l\u0129nh v\u1ef1c li\u1ec7u ph\u00e1p t\u1ebf b\u00e0o \u2013 gen. \u0110\u00e2y l\u00e0 li\u1ec7u ph\u00e1p \u0111\u1ea7u ti\u00ean do m\u1ed9t t\u1ed5 ch\u1ee9c phi l\u1ee3i nhu\u1eadn ph\u00e1t tri\u1ec3n v\u00e0 \u0111\u01b0\u1ee3c FDA ph\u00ea duy\u1ec7t, cho th\u1ea5y r\u1eb1ng nh\u1eefng t\u1ed5 ch\u1ee9c nghi\u00ean c\u1ee9u, kh\u00f4ng ch\u1ec9 c\u00e1c c\u00f4ng ty d\u01b0\u1ee3c l\u1edbn, c\u0169ng c\u00f3 th\u1ec3 \u0111\u00f3ng vai tr\u00f2 then ch\u1ed1t trong vi\u1ec7c t\u1ea1o ra c\u00e1c li\u1ec7u ph\u00e1p ti\u00ean phong. Th\u00e0nh c\u00f4ng n\u00e0y m\u1edf \u0111\u01b0\u1eddng cho nhi\u1ec1u nghi\u00ean c\u1ee9u v\u00e0 ch\u01b0\u01a1ng tr\u00ecnh ph\u00e1t tri\u1ec3n li\u1ec7u ph\u00e1p gen ti\u1ebfp theo, \u0111\u1eb7c bi\u1ec7t trong l\u0129nh v\u1ef1c b\u1ec7nh hi\u1ebfm, n\u01a1i nhu c\u1ea7u \u0111i\u1ec1u tr\u1ecb lu\u00f4n v\u01b0\u1ee3t xa l\u1ef1a ch\u1ecdn hi\u1ec7n c\u00f3. \u0110\u1ed3ng th\u1eddi, Waskyra c\u0169ng th\u00fac \u0111\u1ea9y c\u00e1c b\u1ec7nh vi\u1ec7n v\u00e0 trung t\u00e2m y t\u1ebf \u0111\u1ea7u t\u01b0 ph\u00e1t tri\u1ec3n m\u00f4 h\u00ecnh \u0111i\u1ec1u tr\u1ecb d\u1ef1a tr\u00ean t\u1ebf b\u00e0o g\u1ed1c t\u1ef1 th\u00e2n \u0111\u01b0\u1ee3c ch\u1ec9nh s\u1eeda gen, g\u00f3p ph\u1ea7n h\u00ecnh th\u00e0nh n\u1ec1n t\u1ea3ng v\u1eefng ch\u1eafc cho th\u1ebf h\u1ec7 li\u1ec7u ph\u00e1p y h\u1ecdc ch\u00ednh x\u00e1c trong t\u01b0\u01a1ng lai.&nbsp;<\/p>\n\n\n\n<h2 class=\"wp-block-heading\"><span class=\"ez-toc-section\" id=\"Nhung_giai_phap_ma_Biogroup_cung_cap_cho_lieu_phap_te_bao_%E2%80%93_lieu_phap_gen\"><\/span><strong>Nh\u1eefng gi\u1ea3i ph\u00e1p m\u00e0 Biogroup cung c\u1ea5p cho li\u1ec7u ph\u00e1p t\u1ebf b\u00e0o \u2013 li\u1ec7u ph\u00e1p gen&nbsp;&nbsp;<\/strong><span class=\"ez-toc-section-end\"><\/span><\/h2>\n\n\n\n<p>\u0110\u00e2y kh\u00f4ng ch\u1ec9 l\u00e0 th\u00e0nh c\u00f4ng c\u1ee7a m\u1ed9t s\u1ea3n ph\u1ea9m, m\u00e0 c\u00f2n l\u00e0 th\u00e0nh c\u00f4ng c\u1ee7a c\u1ea3 h\u1ec7 sinh th\u00e1i ph\u00e1t tri\u1ec3n li\u1ec7u ph\u00e1p gen, g\u00f3p ph\u1ea7n m\u1edf ra t\u01b0\u01a1ng lai \u0111i\u1ec1u tr\u1ecb c\u00e1 th\u1ec3 h\u00f3a d\u1ef1a tr\u00ean ch\u1ec9nh s\u1eeda gen.&nbsp;<\/p>\n\n\n<div class=\"wp-block-image\">\n<figure class=\"aligncenter size-full is-resized\"><img decoding=\"async\" width=\"800\" height=\"615\" src=\"https:\/\/biogroupvietnam.com\/wp-content\/uploads\/2025\/12\/stemspan-AOF-moi-truong-nuoi-cay-animal-origin-free.webp\" alt=\"StemSpan\u2122 AOF - M\u00f4i tr\u01b0\u1eddng nu\u00f4i c\u1ea5y Animal Origin-Free\" class=\"wp-image-9304\" style=\"width:542px;height:auto\" srcset=\"https:\/\/biogroupvietnam.com\/wp-content\/uploads\/2025\/12\/stemspan-AOF-moi-truong-nuoi-cay-animal-origin-free.webp 800w, https:\/\/biogroupvietnam.com\/wp-content\/uploads\/2025\/12\/stemspan-AOF-moi-truong-nuoi-cay-animal-origin-free-300x231.webp 300w, https:\/\/biogroupvietnam.com\/wp-content\/uploads\/2025\/12\/stemspan-AOF-moi-truong-nuoi-cay-animal-origin-free-768x590.webp 768w, https:\/\/biogroupvietnam.com\/wp-content\/uploads\/2025\/12\/stemspan-AOF-moi-truong-nuoi-cay-animal-origin-free-600x461.webp 600w, https:\/\/biogroupvietnam.com\/wp-content\/uploads\/2025\/12\/stemspan-AOF-moi-truong-nuoi-cay-animal-origin-free-70x54.webp 70w\" sizes=\"(max-width: 800px) 100vw, 800px\" \/><figcaption class=\"wp-element-caption\"><em>StemSpan\u2122 AOF &#8211; M\u00f4i tr\u01b0\u1eddng nu\u00f4i c\u1ea5y Animal Origin-Free<\/em> <em>(Ngu\u1ed3n: STEMCELL Technologies)<\/em><\/figcaption><\/figure><\/div>\n\n\n<p>B\u00ean c\u1ea1nh \u00fd ngh\u0129a khoa h\u1ecdc v\u00e0 l\u00e2m s\u00e0ng, s\u1ef1 ph\u00e1t tri\u1ec3n c\u1ee7a c\u00e1c li\u1ec7u ph\u00e1p gen nh\u01b0 Waskyra c\u0169ng m\u1edf ra c\u01a1 h\u1ed9i \u0111\u1ec3 c\u00e1c n\u1ec1n t\u1ea3ng c\u00f4ng ngh\u1ec7 hi\u1ec7n c\u00f3 t\u1ea1i Vi\u1ec7t Nam tham gia s\u00e2u h\u01a1n v\u00e0o l\u0129nh v\u1ef1c y h\u1ecdc t\u00e1i t\u1ea1o v\u00e0 \u0111i\u1ec1u tr\u1ecb b\u1ec7nh hi\u1ebfm. T\u1ea1i Vi\u1ec7t Nam, Biogroup hi\u1ec7n \u0111ang ph\u00e2n ph\u1ed1i nhi\u1ec1u gi\u1ea3i ph\u00e1p then ch\u1ed1t c\u00f3 th\u1ec3 h\u1ed7 tr\u1ee3 tr\u1ef1c ti\u1ebfp cho c\u00e1c quy tr\u00ecnh nghi\u00ean c\u1ee9u v\u00e0 ph\u00e1t tri\u1ec3n li\u1ec7u ph\u00e1p t\u1ebf b\u00e0o \u2013 gen t\u01b0\u01a1ng t\u1ef1 Waskyra.&nbsp;&nbsp;<\/p>\n\n\n\n<p><strong>StemSpan\u2122 AOF<\/strong> l\u00e0 m\u00f4i tr\u01b0\u1eddng nu\u00f4i c\u1ea5y <strong>Animal Origin-Free<\/strong> l\u00fd t\u01b0\u1edfng cho m\u1edf r\u1ed9ng t\u1ebf b\u00e0o g\u1ed1c t\u1ea1o m\u00e1u CD34\u207a trong c\u00e1c quy tr\u00ecnh ch\u1ec9nh s\u1eeda gen. V\u1edbi th\u00e0nh ph\u1ea7n ho\u00e0n to\u00e0n t\u1ed5ng h\u1ee3p v\u00e0 protein t\u00e1i t\u1ed5 h\u1ee3p, s\u1ea3n ph\u1ea9m \u0111\u1ea3m b\u1ea3o <strong>\u0111\u1ed9 tinh khi\u1ebft, t\u00ednh nh\u1ea5t qu\u00e1n v\u00e0 an to\u00e0n cao<\/strong>, \u0111\u00e1p \u1ee9ng y\u00eau c\u1ea7u cGMP cho \u1ee9ng d\u1ee5ng l\u00e2m s\u00e0ng. StemSpan AOF \u0111\u00e3 ch\u1ee9ng minh kh\u1ea3 n\u0103ng <strong>m\u1edf r\u1ed9ng v\u01b0\u1ee3t tr\u1ed9i<\/strong> c\u00e1c qu\u1ea7n th\u1ec3 HSC quan tr\u1ecdng, duy tr\u00ec kh\u1ea3 n\u0103ng t\u00e1i t\u1ea1o ng\u1eafn h\u1ea1n v\u00e0 d\u00e0i h\u1ea1n, l\u00e0 l\u1ef1a ch\u1ecdn t\u1ed1i \u01b0u cho s\u1ea3n xu\u1ea5t t\u1ebf b\u00e0o ph\u1ee5c v\u1ee5 li\u1ec7u ph\u00e1p gen nh\u01b0 Waskyra.&nbsp;<\/p>\n\n\n\n<p><em>Xem th\u00eam th\u00f4ng tin chi ti\u1ebft StemSpan AOF: <a href=\"https:\/\/www.stemcell.com\/stemspan-aof.html\">Link<\/a>\u00a0<\/em><\/p>\n\n\n\n<p>Trong qu\u00e1 tr\u00ecnh thu th\u1eadp v\u00e0 m\u1edf r\u1ed9ng t\u1ebf b\u00e0o g\u1ed1c t\u1ea1o m\u00e1u CD34+, c\u00e1c ph\u00f2ng th\u00ed nghi\u1ec7m c\u00f3 th\u1ec3 s\u1eed d\u1ee5ng m\u00f4i tr\u01b0\u1eddng t\u0103ng sinh t\u1ebf b\u00e0o g\u1ed1c t\u1ea1o m\u00e1u StemSpan\u2122 AOF \u2013 m\u1ed9t m\u00f4i tr\u01b0\u1eddng ho\u00e0n to\u00e0n kh\u00f4ng c\u00f3 ngu\u1ed3n g\u1ed1c \u0111\u1ed9ng v\u1eadt, \u0111\u01b0\u1ee3c thi\u1ebft k\u1ebf t\u1ed1i \u01b0u \u0111\u1ec3 duy tr\u00ec kh\u1ea3 n\u0103ng sinh tr\u01b0\u1edfng v\u00e0 ch\u1ee9c n\u0103ng c\u1ee7a HSCs trong \u0111i\u1ec1u ki\u1ec7n GMP. \u0110\u00e2y l\u00e0 m\u1ed9t trong nh\u1eefng ti\u00eau chu\u1ea9n quan tr\u1ecdng khi nu\u00f4i c\u1ea5y t\u1ebf b\u00e0o ph\u1ee5c v\u1ee5 ch\u1ec9nh s\u1eeda gen.&nbsp;<\/p>\n\n\n\n<p><strong>H\u1ec7 th\u1ed1ng chuy\u1ec3n gen CellPore\u2122<\/strong> gi\u1ea3i quy\u1ebft c\u00e1c r\u00e0o c\u1ea3n c\u1ee7a c\u00e1c ph\u01b0\u01a1ng ph\u00e1p chuy\u1ec3n gen truy\u1ec1n th\u1ed1ng\u2014v\u1ed1n th\u01b0\u1eddng g\u1eb7p c\u00e1c v\u1ea5n \u0111\u1ec1 nh\u01b0 hi\u1ec7u qu\u1ea3 th\u1ea5p, \u0111\u1ed9c t\u00ednh cao, g\u00e2y t\u1ed5n th\u01b0\u01a1ng t\u1ebf b\u00e0o v\u00e0 l\u00e0m m\u1ea5t \u0111\u1eb7c t\u00ednh t\u1ebf b\u00e0o g\u1ed1c. CellPore\u2122 cho ph\u00e9p ch\u1ec9nh s\u1eeda gen m\u1ea1nh m\u1ebd tr\u00ean HSPC CD34+ ng\u01b0\u1eddi trong khi v\u1eabn duy tr\u00ec ki\u1ec3u h\u00ecnh v\u00e0 ch\u1ee9c n\u0103ng t\u1ebf b\u00e0o. C\u00f4ng ngh\u1ec7 s\u1eed d\u1ee5ng \u00e1p l\u1ef1c \u0111\u1ec3 \u0111\u01b0a \u0111a d\u1ea1ng v\u1eadt li\u1ec7u v\u00e0o c\u00e1c t\u1ebf b\u00e0o \u201ckh\u00f3 chuy\u1ec3n n\u1ea1p\u201d v\u1edbi m\u1ee9c t\u00e1c \u0111\u1ed9ng t\u1ed1i thi\u1ec3u, mang l\u1ea1i kh\u1ea3 n\u0103ng s\u1ed1ng s\u00f3t v\u00e0 hi\u1ec7u qu\u1ea3 \u0111\u01b0a v\u1eadt li\u1ec7u v\u01b0\u1ee3t tr\u1ed9i. \u0110\u1eb7c bi\u1ec7t, h\u1ec7 th\u1ed1ng ch\u1ec9 c\u1ea7n \u0111i\u1ec1u ch\u1ec9nh quy tr\u00ecnh r\u1ea5t \u00edt, gi\u00fap t\u00edch h\u1ee3p d\u1ec5 d\u00e0ng v\u00e0o c\u00e1c workflow hi\u1ec7n c\u00f3.&nbsp;<\/p>\n\n\n<div class=\"wp-block-image\">\n<figure class=\"aligncenter size-large\"><img loading=\"lazy\" decoding=\"async\" width=\"1024\" height=\"302\" src=\"https:\/\/biogroupvietnam.com\/wp-content\/uploads\/2025\/12\/workflow-chinh-sua-gen-te-bao-goc-tao-mau-CD34-su-dung-he-thong-cellpore-1024x302.png\" alt=\"Workflow ch\u1ec9nh s\u1eeda gen t\u1ebf b\u00e0o g\u1ed1c t\u1ea1o m\u00e1u CD34+ s\u1eed d\u1ee5ng h\u1ec7 th\u1ed1ng CellPore\u00a0\" class=\"wp-image-9308\" srcset=\"https:\/\/biogroupvietnam.com\/wp-content\/uploads\/2025\/12\/workflow-chinh-sua-gen-te-bao-goc-tao-mau-CD34-su-dung-he-thong-cellpore-1024x302.png 1024w, https:\/\/biogroupvietnam.com\/wp-content\/uploads\/2025\/12\/workflow-chinh-sua-gen-te-bao-goc-tao-mau-CD34-su-dung-he-thong-cellpore-300x89.png 300w, https:\/\/biogroupvietnam.com\/wp-content\/uploads\/2025\/12\/workflow-chinh-sua-gen-te-bao-goc-tao-mau-CD34-su-dung-he-thong-cellpore-768x227.png 768w, https:\/\/biogroupvietnam.com\/wp-content\/uploads\/2025\/12\/workflow-chinh-sua-gen-te-bao-goc-tao-mau-CD34-su-dung-he-thong-cellpore-600x177.png 600w, https:\/\/biogroupvietnam.com\/wp-content\/uploads\/2025\/12\/workflow-chinh-sua-gen-te-bao-goc-tao-mau-CD34-su-dung-he-thong-cellpore-90x27.png 90w, https:\/\/biogroupvietnam.com\/wp-content\/uploads\/2025\/12\/workflow-chinh-sua-gen-te-bao-goc-tao-mau-CD34-su-dung-he-thong-cellpore.png 1111w\" sizes=\"(max-width: 1024px) 100vw, 1024px\" \/><figcaption class=\"wp-element-caption\"><em>Workflow ch\u1ec9nh s\u1eeda gen t\u1ebf b\u00e0o g\u1ed1c t\u1ea1o m\u00e1u CD34+ s\u1eed d\u1ee5ng h\u1ec7 th\u1ed1ng CellPore\u00a0(Ngu\u1ed3n: STEMCELL Technologies)<\/em><\/figcaption><\/figure><\/div>\n\n\n<p><em>Xem th\u00eam chi ti\u1ebft t\u1ea1i: <a href=\"https:\/\/cdn.stemcell.com\/media\/files\/brochure\/BR27268-Efficient_Gene_Editing_of_CD34_HSPCs_with_CellPore.pdf\">Effient gene editing of CD34+ HSPCs<\/a>\u00a0<\/em><\/p>\n\n\n<div class=\"wp-block-image\">\n<figure class=\"aligncenter size-large is-resized\"><img loading=\"lazy\" decoding=\"async\" width=\"1024\" height=\"1024\" src=\"https:\/\/biogroupvietnam.com\/wp-content\/uploads\/2025\/12\/ADAMII-CD34-2-1024x1024.jpg\" alt=\"\" class=\"wp-image-9312\" style=\"width:361px;height:auto\" srcset=\"https:\/\/biogroupvietnam.com\/wp-content\/uploads\/2025\/12\/ADAMII-CD34-2-1024x1024.jpg 1024w, https:\/\/biogroupvietnam.com\/wp-content\/uploads\/2025\/12\/ADAMII-CD34-2-300x300.jpg 300w, https:\/\/biogroupvietnam.com\/wp-content\/uploads\/2025\/12\/ADAMII-CD34-2-150x150.jpg 150w, https:\/\/biogroupvietnam.com\/wp-content\/uploads\/2025\/12\/ADAMII-CD34-2-768x768.jpg 768w, https:\/\/biogroupvietnam.com\/wp-content\/uploads\/2025\/12\/ADAMII-CD34-2-450x450.jpg 450w, https:\/\/biogroupvietnam.com\/wp-content\/uploads\/2025\/12\/ADAMII-CD34-2-500x500.jpg 500w, https:\/\/biogroupvietnam.com\/wp-content\/uploads\/2025\/12\/ADAMII-CD34-2-600x600.jpg 600w, https:\/\/biogroupvietnam.com\/wp-content\/uploads\/2025\/12\/ADAMII-CD34-2-54x54.jpg 54w, https:\/\/biogroupvietnam.com\/wp-content\/uploads\/2025\/12\/ADAMII-CD34-2.jpg 1080w\" sizes=\"(max-width: 1024px) 100vw, 1024px\" \/><figcaption class=\"wp-element-caption\"><em>M\u00e1y \u0111\u1ebfm t\u1ebf b\u00e0o ADAMII CD34+<\/em><\/figcaption><\/figure><\/div>\n\n\n<p><strong>M\u00e1y \u0111\u1ebfm t\u1ebf b\u00e0o ADAMII CD34+<\/strong> l\u00e0 h\u1ec7 th\u1ed1ng ph\u00e2n t\u00edch t\u1ebf b\u00e0o t\u1ef1 \u0111\u1ed9ng d\u1ef1a tr\u00ean h\u00ecnh \u1ea3nh hu\u1ef3nh quang, \u0111\u01b0\u1ee3c thi\u1ebft k\u1ebf \u0111\u1ec3 \u0111\u1ecbnh l\u01b0\u1ee3ng ch\u00ednh x\u00e1c t\u1ebf b\u00e0o CD34+ trong c\u00e1c m\u1eabu t\u1ebf b\u00e0o g\u1ed1c t\u1ea1o m\u00e1u. V\u1edbi c\u00f4ng ngh\u1ec7 vi l\u01b0u v\u00e0 thu\u1eadt to\u00e1n nh\u1eadn d\u1ea1ng h\u00ecnh \u1ea3nh ti\u00ean ti\u1ebfn, ADAMII mang l\u1ea1i \u0111\u1ed9 ch\u00ednh x\u00e1c v\u00e0 \u0111\u1ed9 l\u1eb7p l\u1ea1i cao, \u0111\u00e1p \u1ee9ng y\u00eau c\u1ea7u kh\u1eaft khe trong s\u1ea3n xu\u1ea5t t\u1ebf b\u00e0o cho li\u1ec7u ph\u00e1p gen. Thi\u1ebft b\u1ecb ho\u1ea1t \u0111\u1ed9ng nhanh, quy tr\u00ecnh thao t\u00e1c \u0111\u01a1n gi\u1ea3n, gi\u1ea3m thi\u1ec3u sai s\u1ed1 ng\u01b0\u1eddi v\u1eadn h\u00e0nh v\u00e0 cho ph\u00e9p chu\u1ea9n h\u00f3a k\u1ebft qu\u1ea3 gi\u1eefa c\u00e1c ph\u00f2ng th\u00ed nghi\u1ec7m. Nh\u1edd kh\u1ea3 n\u0103ng ki\u1ec3m so\u00e1t ch\u1ea5t l\u01b0\u1ee3ng CD34+ \u0111\u00e1ng tin c\u1eady, ADAMII l\u00e0 c\u00f4ng c\u1ee5 l\u00fd t\u01b0\u1edfng trong thu nh\u1eadn, m\u1edf r\u1ed9ng v\u00e0 \u0111\u00e1nh gi\u00e1 HSPC ph\u1ee5c v\u1ee5 c\u00e1c quy tr\u00ecnh ch\u1ec9nh s\u1eeda gen v\u00e0 gh\u00e9p t\u1ebf b\u00e0o.&nbsp;<\/p>\n\n\n\n<p><em>Xem th\u00f4ng tin s\u1ea3n ph\u1ea9m: <a href=\"https:\/\/www.nanoentek.com\/theme\/nanont2_en\/shop\/02\/product01_view.php?it_id=1547537314\">ADAMII Fluorescence CD34 Counter<\/a>\u00a0<\/em><\/p>\n\n\n\n<h2 class=\"wp-block-heading\"><span class=\"ez-toc-section\" id=\"Ket_luan\"><\/span><strong>K\u1ebft lu\u1eadn&nbsp;<\/strong><span class=\"ez-toc-section-end\"><\/span><\/h2>\n\n\n\n<p>Vi\u1ec7c FDA ph\u00ea duy\u1ec7t Waskyra \u0111\u00e1nh d\u1ea5u b\u01b0\u1edbc ti\u1ebfn quan tr\u1ecdng trong \u0111i\u1ec1u tr\u1ecb h\u1ed9i ch\u1ee9ng Wiskott-Aldrich v\u00e0 c\u1ee7ng c\u1ed1 v\u1ecb th\u1ebf c\u1ee7a li\u1ec7u ph\u00e1p gen nh\u01b0 m\u1ed9t tr\u1ee5 c\u1ed9t m\u1edbi trong y h\u1ecdc t\u00e1i t\u1ea1o.&nbsp;<\/p>\n\n\n\n<p>Waskyra:&nbsp;<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>L\u00e0 li\u1ec7u ph\u00e1p gen \u0111\u1ea7u ti\u00ean d\u00e0nh cho WAS&nbsp;<\/li>\n\n\n\n<li>Mang l\u1ea1i hi\u1ec7u qu\u1ea3 l\u00e2m s\u00e0ng m\u1ea1nh m\u1ebd v\u00e0 b\u1ec1n v\u1eefng&nbsp;<\/li>\n\n\n\n<li>Lo\u1ea1i b\u1ecf s\u1ef1 ph\u1ee5 thu\u1ed9c v\u00e0o ng\u01b0\u1eddi hi\u1ebfn HLA ph\u00f9 h\u1ee3p&nbsp;<\/li>\n\n\n\n<li>T\u1ea1o hy v\u1ecdng m\u1edbi cho h\u00e0ng ng\u00e0n b\u1ec7nh nh\u00e2n v\u00e0 gia \u0111\u00ecnh tr\u00ean th\u1ebf gi\u1edbi\u00a0<\/li>\n<\/ul>\n\n\n\n<p>\ud83d\udce9&nbsp;<strong>Th\u00f4ng tin li\u00ean h\u1ec7:<\/strong><\/p>\n\n\n\n<p>Biogroup Vietnam chuy\u00ean ph\u00e2n ph\u1ed1i v\u00e0 cung c\u1ea5p d\u1ecbch v\u1ee5 k\u1ef9 thu\u1eadt trong l\u0129nh v\u1ef1c thi\u1ebft b\u1ecb, v\u1eadt t\u01b0, h\u00f3a ch\u1ea5t v\u00e0 gi\u1ea3i ph\u00e1p sinh h\u1ecdc cho c\u00e1c ph\u00f2ng th\u00ed nghi\u1ec7m c\u00f4ng ngh\u1ec7 sinh h\u1ecdc, y sinh v\u00e0 y h\u1ecdc t\u00e1i t\u1ea1o t\u1ea1i Vi\u1ec7t Nam.<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Website:\u00a0<a href=\"https:\/\/biogroupvietnam.com\/vi\/lien-he\/\">https:\/\/biogroupvietnam.com\/vi\/lien-he\/<\/a>\u00a0<\/li>\n\n\n\n<li>Hotline: +84 963 621 421\u00a0<\/li>\n\n\n\n<li>Email: infor@biogroupvietnam.com<\/li>\n<\/ul>\n\n\n\n<h2 class=\"wp-block-heading\"><span class=\"ez-toc-section\" id=\"Tai_lieu_tham_khao\"><\/span><strong>T\u00e0i li\u1ec7u tham kh\u1ea3o<\/strong>&nbsp;<span class=\"ez-toc-section-end\"><\/span><\/h2>\n\n\n\n<p><em>1. <\/em>FDA. (2025, December 9). <em>FDA approves first gene therapy treatment for Wiskott-Aldrich syndrome<\/em>. <em>https:\/\/www.fda.gov\/news-events\/press-announcements\/fda-approves-first-gene-therapy-treatment-wiskott-aldrich-syndrome<\/em><\/p>\n\n\n\n<p><em>2. <\/em>Clare, B. (2025, November 25). <em>Gene therapy revolution: Waskyra offers life-changing hope for Wiskott-Aldrich syndrome patients<\/em>. Patient Worthy.<strong> <\/strong><a href=\"https:\/\/patientworthy.com\/2025\/11\/25\/gene-therapy-revolution-waskyra-offers-life-changing-hope-for-wiskott-aldrich-syndrome-patients\/\"><em>https:\/\/patientworthy.com\/2025\/11\/25\/gene-therapy-revolution-waskyra-offers-life-changing-hope-for-wiskott-aldrich-syndrome-patients\/<\/em><\/a>\u00a0<\/p>\n\n\n\n<p><em>3.<\/em><strong> <\/strong><em>Hoffman, M. (2025, December 10). FDA approves Waskyra as first gene therapy for Wiskott-Aldrich syndrome. CGTlive. <a href=\"https:\/\/www.cgtlive.com\/view\/fda-approves-waskyra-first-gene-therapy-wiskott-aldrich-syndrome\">https:\/\/www.cgtlive.com\/view\/fda-approves-waskyra-first-gene-therapy-wiskott-aldrich-syndrome<\/a><\/em><\/p>\n","protected":false},"excerpt":{"rendered":"<p>Ng\u00e0y 9\/12\/2025 \u0111\u00e1nh d\u1ea5u m\u1ed9t c\u1ed9t m\u1ed1c l\u1ecbch s\u1eed trong l\u0129nh v\u1ef1c li\u1ec7u ph\u00e1p gen: C\u1ee5c Qu\u1ea3n l\u00fd Th\u1ef1c ph\u1ea9m v\u00e0 D\u01b0\u1ee3c ph\u1ea9m Hoa K\u1ef3 (FDA) \u0111\u00e3 ch\u00ednh th\u1ee9c ph\u00ea duy\u1ec7t etuvetidigene autotemcel (Waskyra) \u2013 li\u1ec7u ph\u00e1p gen \u0111\u1ea7u ti\u00ean \u0111\u01b0\u1ee3c ch\u1ea5p thu\u1eadn \u0111\u1ec3 \u0111i\u1ec1u tr\u1ecb H\u1ed9i ch\u1ee9ng Wiskott-Aldrich (WAS).&nbsp; Waskyra, do Fondazione Telethon ETS<\/p>\n<div class=\"klb-readmore post-buttons\"><a class=\"button rounded primary\" href=\"https:\/\/biogroupvietnam.com\/en\/news\/fda-phe-duyet-waskyra-lieu-phap-gen-dau-tien-dieu-tri-wiskott-aldrich\/\">Read More <i class=\"klbth-icon-right-arrow\"><\/i><\/a><\/div>\n","protected":false},"author":1,"featured_media":9307,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"footnotes":""},"categories":[19],"tags":[],"class_list":["post-9290","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-news"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v26.8 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>FDA ph\u00ea duy\u1ec7t Waskyra nh\u01b0 li\u1ec7u ph\u00e1p gen \u0111\u1ea7u ti\u00ean \u0111i\u1ec1u tr\u1ecb h\u1ed9i ch\u1ee9ng Wiskott\u2013Aldrich (WAS)<\/title>\n<meta name=\"description\" content=\"Ng\u00e0y 9\/12\/2025, FDA ch\u00ednh th\u1ee9c ph\u00ea duy\u1ec7t Waskyra \u2013 li\u1ec7u ph\u00e1p gen \u0111\u1ea7u ti\u00ean \u0111\u01b0\u1ee3c ch\u1ea5p thu\u1eadn \u0111\u1ec3 \u0111i\u1ec1u tr\u1ecb H\u1ed9i ch\u1ee9ng Wiskott-Aldrich (WAS), \u0111\u00e1nh d\u1ea5u b\u01b0\u1edbc ngo\u1eb7c quan tr\u1ecdng c\u1ee7a y h\u1ecdc hi\u1ec7n \u0111\u1ea1i.\" \/>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"FDA ph\u00ea duy\u1ec7t Waskyra nh\u01b0 li\u1ec7u ph\u00e1p gen \u0111\u1ea7u ti\u00ean \u0111i\u1ec1u tr\u1ecb h\u1ed9i ch\u1ee9ng Wiskott\u2013Aldrich (WAS)\" \/>\n<meta property=\"og:description\" content=\"Ng\u00e0y 9\/12\/2025, FDA ch\u00ednh th\u1ee9c ph\u00ea duy\u1ec7t Waskyra \u2013 li\u1ec7u ph\u00e1p gen \u0111\u1ea7u ti\u00ean \u0111\u01b0\u1ee3c ch\u1ea5p thu\u1eadn \u0111\u1ec3 \u0111i\u1ec1u tr\u1ecb H\u1ed9i ch\u1ee9ng Wiskott-Aldrich (WAS), \u0111\u00e1nh d\u1ea5u b\u01b0\u1edbc ngo\u1eb7c quan tr\u1ecdng c\u1ee7a y h\u1ecdc hi\u1ec7n \u0111\u1ea1i.\" \/>\n<meta property=\"og:url\" content=\"https:\/\/biogroupvietnam.com\/en\/news\/fda-phe-duyet-waskyra-lieu-phap-gen-dau-tien-dieu-tri-wiskott-aldrich\/\" \/>\n<meta property=\"og:site_name\" content=\"Biogroup Vietnam\" \/>\n<meta property=\"article:published_time\" content=\"2025-12-16T08:52:06+00:00\" \/>\n<meta property=\"article:modified_time\" content=\"2026-02-24T09:40:22+00:00\" \/>\n<meta property=\"og:image\" content=\"https:\/\/biogroupvietnam.com\/wp-content\/uploads\/2025\/12\/FDA-phe-duyet-waskyra-lieu-phap-gen-dau-tien-dieu-tri-wiskott-aldrich-1.webp\" \/>\n\t<meta property=\"og:image:width\" content=\"1200\" \/>\n\t<meta property=\"og:image:height\" content=\"675\" \/>\n\t<meta property=\"og:image:type\" content=\"image\/webp\" \/>\n<meta name=\"author\" content=\"admin\" \/>\n<meta name=\"twitter:card\" content=\"summary_large_image\" \/>\n<meta name=\"twitter:label1\" content=\"Written by\" \/>\n\t<meta name=\"twitter:data1\" content=\"admin\" \/>\n\t<meta name=\"twitter:label2\" content=\"Est. reading time\" \/>\n\t<meta name=\"twitter:data2\" content=\"14 minutes\" \/>\n<script type=\"application\/ld+json\" class=\"yoast-schema-graph\">{\"@context\":\"https:\/\/schema.org\",\"@graph\":[{\"@type\":\"Article\",\"@id\":\"https:\/\/biogroupvietnam.com\/en\/news\/fda-phe-duyet-waskyra-lieu-phap-gen-dau-tien-dieu-tri-wiskott-aldrich\/#article\",\"isPartOf\":{\"@id\":\"https:\/\/biogroupvietnam.com\/en\/news\/fda-phe-duyet-waskyra-lieu-phap-gen-dau-tien-dieu-tri-wiskott-aldrich\/\"},\"author\":{\"name\":\"admin\",\"@id\":\"https:\/\/biogroupvietnam.com\/vi\/#\/schema\/person\/78a1c640012e6e7bc2bdf0d0fbffea95\"},\"headline\":\"FDA ph\u00ea duy\u1ec7t Waskyra nh\u01b0 li\u1ec7u ph\u00e1p gen \u0111\u1ea7u ti\u00ean \u0111i\u1ec1u tr\u1ecb h\u1ed9i ch\u1ee9ng Wiskott\u2013Aldrich (WAS)\",\"datePublished\":\"2025-12-16T08:52:06+00:00\",\"dateModified\":\"2026-02-24T09:40:22+00:00\",\"mainEntityOfPage\":{\"@id\":\"https:\/\/biogroupvietnam.com\/en\/news\/fda-phe-duyet-waskyra-lieu-phap-gen-dau-tien-dieu-tri-wiskott-aldrich\/\"},\"wordCount\":3579,\"commentCount\":0,\"image\":{\"@id\":\"https:\/\/biogroupvietnam.com\/en\/news\/fda-phe-duyet-waskyra-lieu-phap-gen-dau-tien-dieu-tri-wiskott-aldrich\/#primaryimage\"},\"thumbnailUrl\":\"https:\/\/biogroupvietnam.com\/wp-content\/uploads\/2025\/12\/FDA-phe-duyet-waskyra-lieu-phap-gen-dau-tien-dieu-tri-wiskott-aldrich-1.webp\",\"articleSection\":[\"News\"],\"inLanguage\":\"en-US\",\"potentialAction\":[{\"@type\":\"CommentAction\",\"name\":\"Comment\",\"target\":[\"https:\/\/biogroupvietnam.com\/en\/news\/fda-phe-duyet-waskyra-lieu-phap-gen-dau-tien-dieu-tri-wiskott-aldrich\/#respond\"]}]},{\"@type\":\"WebPage\",\"@id\":\"https:\/\/biogroupvietnam.com\/en\/news\/fda-phe-duyet-waskyra-lieu-phap-gen-dau-tien-dieu-tri-wiskott-aldrich\/\",\"url\":\"https:\/\/biogroupvietnam.com\/en\/news\/fda-phe-duyet-waskyra-lieu-phap-gen-dau-tien-dieu-tri-wiskott-aldrich\/\",\"name\":\"FDA 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