{"id":4428,"date":"2025-09-07T13:07:26","date_gmt":"2025-09-07T10:07:26","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=4428"},"modified":"2025-11-03T20:20:56","modified_gmt":"2025-11-03T17:20:56","slug":"gnr-097-duchenne-gene-therapy-phase-1-2-study-started-by-generium","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/gnr-097-duchenne-gene-therapy-phase-1-2-study-started-by-generium\/","title":{"rendered":"GNR-097: Duchenne Gen Terapisi Faz 1\/2 \u00c7al\u0131\u015fmas\u0131 Generium Taraf\u0131ndan Ba\u015flat\u0131ld\u0131"},"content":{"rendered":"<p class=\"wp-block-paragraph\"><strong>Rus yenilik\u00e7i biyoteknoloji \u015firketi Generium, ilerleyici Duchenne kas distrofisi (DMD) tedavisi i\u00e7in GNR-097 gen terapisi ilac\u0131n\u0131n klinik denemesini y\u00fcr\u00fctmek \u00fczere onay ald\u0131.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u00c7ok merkezli, tek k\u00f6r, randomize plasebo kontroll\u00fc \u00e7al\u0131\u015fma, progresif Duchenne kas distrofisi olan pediatrik hastalara gen tedavisi ilac\u0131n\u0131n tek bir intraven\u00f6z uygulamas\u0131n\u0131 i\u00e7ermektedir. Bu Faz 1\/2 \u00e7al\u0131\u015fmas\u0131, GNR-097 ilac\u0131n\u0131n tolere edilebilirli\u011fini, g\u00fcvenli\u011fini ve etkinli\u011fini de\u011ferlendirmeyi ama\u00e7lamaktad\u0131r.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">GNR-097 Duchenne Gen Terapisi<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>GNR-097, kesilmi\u015f distrofin genini kodlayan serotip 9&#039;un rekombinant adeno-ili\u015fkili viral vekt\u00f6r\u00fcd\u00fcr.<\/strong><\/p>\n\n\n\n<h2 class=\"wp-block-heading\">GENERIUM CEO&#039;su Daniil Talyansky:<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Gen terapisi, k\u00fcresel ila\u00e7 ve t\u0131p bilimindeki en gen\u00e7 ve \u00e7\u0131\u011f\u0131r a\u00e7an aland\u0131r. Rus bilim insanlar\u0131n\u0131n benzersiz uzmanl\u0131\u011f\u0131, mikrodistrofin genini kodlayan rekombinant adeno-ili\u015fkili viral vekt\u00f6r\u00fcn geli\u015ftirilmesini sa\u011flam\u0131\u015ft\u0131r. \u015eu anda b\u00fcy\u00fck umut vadeden bu orijinal ilac\u0131n klinik deneylerine ba\u015fl\u0131yoruz.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">GNR-097 Gen Terapisinin Klinik \u00c7al\u0131\u015fmas\u0131<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Bu yenilik\u00e7i ilac\u0131n denemeleri uluslararas\u0131 olup Rusya Federasyonu ve Belarus Cumhuriyeti&#039;nde ger\u00e7ekle\u015ftirilecektir. Rusya&#039;da \u00e7al\u0131\u015fma be\u015f t\u0131p merkezinde ger\u00e7ekle\u015ftirilecektir: Akademisyen Yu.E. Veltishchev ad\u0131na Pediatrik ve Pediatrik Cerrahi Klinik Ara\u015ft\u0131rma Enstit\u00fcs\u00fc, Pirogov Rusya Ulusal T\u0131p Ara\u015ft\u0131rma \u00dcniversitesi, St. Petersburg Devlet Pediatri \u00dcniversitesi, Yekaterinburg B\u00f6lge \u00c7ocuk Klinik Hastanesi ve Ulusal \u00c7ocuk Sa\u011fl\u0131\u011f\u0131 T\u0131bbi Ara\u015ft\u0131rma Merkezi. Belarus&#039;ta ise denemeler Cumhuriyet\u00e7i Bilim ve Uygulama Merkezi &quot;Anne ve \u00c7ocuk&quot;ta ger\u00e7ekle\u015ftirilecektir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Devam\u0131n\u0131 oku<\/strong>:\u00a0<a href=\"https:\/\/dmdwarrior.com\/tr\/dmd-therapies\/\" target=\"_blank\" rel=\"noreferrer noopener\">Duchenne \u0130\u00e7in Klinik Ara\u015ft\u0131rmalar (T\u00fcm Ara\u015ft\u0131rmalar\u0131n Listesi)<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>Generium is a Russian innovative biotechnology company has received approval to conduct a clinical trial of GNR-097 gene therapy drug for the treatment of progressive Duchenne muscular dystrophy (DMD). The multicenter, single-blind, randomized placebo-controlled trial involves a single intravenous administration of the gene therapy drug to pediatric patients with progressive Duchenne muscular dystrophy. This Phase [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":4430,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[27],"tags":[137,415,61,241,414,135],"class_list":["post-4428","post","type-post","status-publish","format-standard","has-post-thumbnail","category-research","tag-clinical-trial","tag-daniil-talyansky","tag-gene-therapy","tag-generium","tag-gnr-097","tag-russia"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/4428","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/comments?post=4428"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/4428\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/4430"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=4428"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=4428"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=4428"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}