{"id":1402,"date":"2024-04-03T17:30:39","date_gmt":"2024-04-03T15:30:39","guid":{"rendered":"https:\/\/remeds.org\/?p=1402"},"modified":"2025-09-10T08:53:27","modified_gmt":"2025-09-10T06:53:27","slug":"huidagene-moving-towards-clinical-trials","status":"publish","type":"post","link":"https:\/\/remeds.org\/tr\/2024\/04\/03\/huidagene-moving-towards-clinical-trials\/","title":{"rendered":"HuidaGene: Klinik \u00e7al\u0131\u015fmalara do\u011fru ilerliyor"},"content":{"rendered":"

\u015eubat 2024\u2019te, DupMECP2 Ba\u015fkan\u0131 David Covini, \u015eanghay\u2019daki yenilik\u00e7i biyoteknoloji \u015firketi HuidaGene Therapeutics\u2019i ziyaret etti. Bu ziyaret, MECP2 duplikasyon sendromu (MDS) i\u00e7in bir tedavinin gelece\u011fine dair heyecan verici bir bak\u0131\u015f sa\u011flad\u0131.<\/p>\n\n\n\n

HuidaGene CEO\u2019su Dr. Alvin Luk\u2019un liderli\u011finde, \u015firket MDS\u2019den etkilenen \u00e7ocuklar\u0131n ya\u015famlar\u0131n\u0131 k\u00f6kten de\u011fi\u015ftirebilecek \u00e7\u0131\u011f\u0131r a\u00e7\u0131c\u0131 bir CRISPR teknolojisi geli\u015ftirme konusunda \u00f6n saflarda yer al\u0131yor. <\/p>\n\n\n\n

Ziyaret, son teknoloji tesislere ve b\u00f6ylesine yenilik\u00e7i ara\u015ft\u0131rmalar\u0131 te\u015fvik eden yarat\u0131c\u0131 ortama ilk elden tan\u0131kl\u0131k etmek i\u00e7in inan\u0131lmaz bir f\u0131rsat oldu. Ofis alan\u0131nda dinamik ve yarat\u0131c\u0131 bir \u00e7al\u0131\u015fma ortam\u0131n\u0131 te\u015fvik eden bir t\u0131rmanma duvar\u0131 bile bulunuyor.<\/p>\n\n\n\n

\"\"<\/figure>\n\n\n\n

Alvin ve MDS T\u0131bbi Direkt\u00f6r\u00fc Dr. Gloria Tang, MECP2 Duplikasyon Sendromu i\u00e7in HG204 ad\u0131 verilen CRISPR-Cas13 RNA d\u00fczenleme teknolojisinin ayr\u0131nt\u0131lar\u0131n\u0131 sundular. \u0130nsan d\u0131\u015f\u0131 primatlarda y\u00fcr\u00fct\u00fclen toksisite \u00e7al\u0131\u015fmalar\u0131ndan elde edilen umut verici veriler ve \u00f6n sonu\u00e7lar, heyecan verici bir geli\u015fmeye yol a\u00e7t\u0131. \u015eirketin MDS klinik \u00e7al\u0131\u015fmas\u0131, ba\u015flang\u0131\u00e7ta 2024\u2019\u00fcn ikinci yar\u0131s\u0131nda ba\u015flat\u0131lmas\u0131 planlan\u0131rken, halihaz\u0131rda iki hastaneden etik kurul onay\u0131 ald\u0131 ve y\u0131l bitmeden MDS hastalar\u0131n\u0131n \u00e7al\u0131\u015fmaya d\u00e2hil edilmesi planlan\u0131yor.<\/p>\n\n\n\n

Bu, MDS toplulu\u011fu i\u00e7in harika bir haber. Yakla\u015fan HG204 MDS klinik \u00e7al\u0131\u015fmas\u0131, \u201cHERO\u201d ad\u0131yla an\u0131lacak ve ba\u015flang\u0131\u00e7ta 1 y\u0131l boyunca yak\u0131ndan izlenecek en az 4 hasta tedavi edilecek. Bu \u00e7al\u0131\u015fman\u0131n ilk faz\u0131 \u00c7in\u2019de y\u00fcr\u00fct\u00fclecek. \u00c7in\u2019de daha fazla hasta kayd\u0131, m\u00fcdahaleli \u00e7al\u0131\u015fman\u0131n ilk \u00f6n sonu\u00e7lar\u0131na ba\u011fl\u0131 olacak. <\/p>\n\n\n\n

HuidaGene, d\u00fczenleyici onay konusunda \u015fimdiden \u00f6nemli ilerlemeler kaydetti; MECP2 duplikasyon sendromunun tedavisi i\u00e7in HG204\u2019e iki FDA tan\u0131m\u0131 (Nadir Pediatrik Hastal\u0131k Tan\u0131m\u0131 (RPDD) ve Yetim \u0130la\u00e7 Tan\u0131m\u0131 (ODD)) ald\u0131. Ayr\u0131ca ABD ve Avrupa\u2019da y\u00fcr\u00fct\u00fclecek daha ileri \u00e7al\u0131\u015fmalar i\u00e7in FDA ve EMA ile temasa ge\u00e7ti.<\/p>\n\n\n\n

Alvin ve Gloria ile y\u00fcz y\u00fcze g\u00f6r\u00fc\u015fme f\u0131rsat\u0131 bulmak bizim i\u00e7in bir ayr\u0131cal\u0131kt\u0131. Bu yolculu\u011fun bir par\u00e7as\u0131 olmaktan b\u00fcy\u00fck heyecan duyuyoruz ve potansiyel olarak hayat de\u011fi\u015ftiren bu teknolojinin ilerleyi\u015fi hakk\u0131nda toplulu\u011fumuzu bilgilendirmek i\u00e7in sab\u0131rs\u0131zlan\u0131yoruz.<\/p>","protected":false},"excerpt":{"rendered":"

In February 2024, David Covini, President of DupMECP2, visited the innovative biotech company HuidaGene Therapeutics in Shanghai. The visit provided an exciting glimpse into the future for a treatment for MECP2 duplication syndrome (MDS). Led by Dr. Alvin Luk, CEO at HuidaGene, the Company is at the forefront of developing a groundbreaking CRISPR technology that […]<\/p>\n","protected":false},"author":73,"featured_media":3480,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"_jf_save_progress":"","footnotes":""},"categories":[12,10,9],"tags":[],"class_list":["post-1402","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-crispr","category-cure","category-research"],"_links":{"self":[{"href":"https:\/\/remeds.org\/tr\/wp-json\/wp\/v2\/posts\/1402","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/remeds.org\/tr\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/remeds.org\/tr\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/remeds.org\/tr\/wp-json\/wp\/v2\/users\/73"}],"replies":[{"embeddable":true,"href":"https:\/\/remeds.org\/tr\/wp-json\/wp\/v2\/comments?post=1402"}],"version-history":[{"count":4,"href":"https:\/\/remeds.org\/tr\/wp-json\/wp\/v2\/posts\/1402\/revisions"}],"predecessor-version":[{"id":3561,"href":"https:\/\/remeds.org\/tr\/wp-json\/wp\/v2\/posts\/1402\/revisions\/3561"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/remeds.org\/tr\/wp-json\/wp\/v2\/media\/3480"}],"wp:attachment":[{"href":"https:\/\/remeds.org\/tr\/wp-json\/wp\/v2\/media?parent=1402"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/remeds.org\/tr\/wp-json\/wp\/v2\/categories?post=1402"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/remeds.org\/tr\/wp-json\/wp\/v2\/tags?post=1402"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}