{"id":4242,"date":"2025-07-29T10:23:25","date_gmt":"2025-07-29T07:23:25","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=4242"},"modified":"2025-07-30T13:54:29","modified_gmt":"2025-07-30T10:54:29","slug":"genethon-gnt0004-phase-3-pivotal-clinical-trial-begins","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/genethon-gnt0004-phase-3-pivotal-clinical-trial-begins\/","title":{"rendered":"Genethon, Duchenne Kas Distrofisi i\u00e7in D\u00fc\u015f\u00fck Dozlu Mikrodistrofin Gen Terapisi GNT0004&#039;\u00fcn Avrupa&#039;da \u00d6nemli Bir Denemesini Ba\u015flatacak"},"content":{"rendered":"<p class=\"wp-block-paragraph\"><strong>Genethon, Duchenne kas distrofisi (DMD) i\u00e7in gen tedavisi GNT0004&#039;\u00fcn Fransa ve \u0130ngiltere&#039;de \u00f6nemli Faz 3 klinik \u00e7al\u0131\u015fmalar\u0131na ba\u015flamak \u00fczere d\u00fczenleyici otoriteler MHRA ve EMA&#039;den onay ald\u0131.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Genethon CEO&#039;su Frederic Revah, &quot;Bu denemelere devam edebildi\u011fimiz i\u00e7in \u00e7ok mutluyuz ve GNT0004&#039;\u00fc terap\u00f6tik bir \u00e7\u00f6z\u00fcm bekleyen gen\u00e7 hastalar ve aileleri i\u00e7in piyasaya s\u00fcrmeye kararl\u0131y\u0131z. Bu, 2021&#039;de ba\u015flayan ve Faz 1\/2\/3 \u00e7al\u0131\u015fmam\u0131z\u0131n Faz 1\/2 b\u00f6l\u00fcm\u00fcnde tedavi edilen ilk \u00e7ocuklarda son derece \u00fcmit verici sonu\u00e7lar g\u00f6steren DMD gen tedavisi program\u0131m\u0131z i\u00e7in belirleyici bir ad\u0131m.&quot; dedi.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Genethon GNT0004 Faz 3 Pivotal Denemesi<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>A\u011fustos ve Eyl\u00fcl aylar\u0131nda \u0130ngiltere ve Fransa&#039;da ba\u015flat\u0131lacak \u00e7ift k\u00f6r ara\u015ft\u0131rmaya, y\u00fcr\u00fcme yetene\u011fini koruyan 6-10 ya\u015f aras\u0131 64 DMD&#039;li erkek \u00e7ocuk kat\u0131lacak.<\/li>\n<\/ul>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Faz 3 deneme onay\u0131, Duchenne kas distrofisi (DMD) i\u00e7in di\u011fer gen tedavilerinde kullan\u0131lan dozlardan daha d\u00fc\u015f\u00fck olan 3\u00d710\u00b9\u00b3 vg\/kg mikrodistrofin dozunun g\u00fcvenli\u011fini ve etkinli\u011fini g\u00f6steren Faz 1\/2 sonu\u00e7lar\u0131na dayanmaktad\u0131r.<\/li>\n<\/ul>\n\n\n\n<h2 class=\"wp-block-heading\">CPK&#039;da \u00d6nemli Kal\u0131c\u0131 Azalma<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Avrupa&#039;daki Faz 3 onaylar\u0131, GNT0004&#039;\u00fcn iyi tolerans g\u00f6sterdi\u011fini ve mikrodistrofin ekspresyonu, kreatin fosfokinaz (CPK) azalmas\u0131 ve motor fonksiyon a\u00e7\u0131s\u0131ndan etkili oldu\u011funu g\u00f6steren Faz 1\/2 \u00e7al\u0131\u015fmalar\u0131n\u0131n sonu\u00e7lar\u0131na dayanmaktad\u0131r. Hastalar, motor fonksiyonlar\u0131nda uzun s\u00fcreli iyile\u015fme veya stabilizasyon ve kas hasar\u0131n\u0131n \u00f6nemli bir belirteci olan CPK&#039;da \u00f6nemli ve kal\u0131c\u0131 bir azalma g\u00f6stermi\u015ftir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Daha fazla bilgi edin: <a href=\"https:\/\/dmdwarrior.com\/tr\/genethon-reports-results-of-gnt0004-gene-therapy-clinical-trial-permanent-decrease-in-ck-levels\/\">CK D\u00fczeylerinde 75%&#039;den Fazla Kal\u0131c\u0131 Azalma<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Faz 3 \u00e7ift k\u00f6r \u00e7al\u0131\u015fmalar, ge\u00e7ici imm\u00fcnolojik profilaktik tedaviyle ili\u015fkili bir AAV8 vekt\u00f6r\u00fcnde distrofin kodlayan genin k\u0131salt\u0131lm\u0131\u015f (3\u00d710\u00b9\u00b3 vg\/kg mikrodistrofin) ancak i\u015flevsel bir versiyonu olan optimize edilmi\u015f hMD1 transgeni i\u00e7eren GNT0004&#039;\u00fcn tek bir intraven\u00f6z enjeksiyonu kullan\u0131larak Birle\u015fik Krall\u0131k ve Fransa&#039;da A\u011fustos ve Eyl\u00fcl aylar\u0131nda ba\u015flayacakt\u0131r. Vekt\u00f6r, bu dokulara \u00f6zg\u00fc bir Spc5-12 promot\u00f6r dizisi sayesinde kendini kas dokusunda ve kalpte ifade edecek \u015fekilde tasarlanm\u0131\u015ft\u0131r. Y\u00fcr\u00fcme yetene\u011fini koruyan 6-10 ya\u015f aras\u0131 DMD&#039;li toplam 64 erkek \u00e7ocuk \u00e7al\u0131\u015fmaya dahil edilecektir.<\/p>\n\n\n\n<figure class=\"wp-block-embed is-type-video is-provider-youtube wp-block-embed-youtube wp-embed-aspect-16-9 wp-has-aspect-ratio\"><div class=\"wp-block-embed__wrapper\">\n<div class=\"youtube-embed\" data-video_id=\"T0zZN7c2cSA\"><iframe title=\"Duchenne Miyopatisi: Konuya \u00f6zg\u00fc bir terapi \u00f6zelli\u011fi | AFM-T\u00e9lethon\" width=\"696\" height=\"392\" src=\"https:\/\/www.youtube.com\/embed\/T0zZN7c2cSA?feature=oembed&#038;enablejsapi=1\" frameborder=\"0\" allow=\"accelerometer; autoplay; clipboard-write; encrypted-media; gyroscope; picture-in-picture; web-share\" referrerpolicy=\"strict-origin-when-cross-origin\" allowfullscreen><\/iframe><\/div>\n<\/div><\/figure>\n\n\n\n<p class=\"wp-block-paragraph\">9 ya\u015f\u0131ndaki Sacha, G\u00e9n\u00e9thon&#039;un kaslardaki eksik protein \u00fcretimini geri kazand\u0131rmay\u0131 ama\u00e7layan mikrodistrofin tedavisini i\u00e7eren denemenin ilk a\u015famas\u0131 kapsam\u0131nda geli\u015ftirilen tedaviyi alan \u00e7ocuklardan biri. Sacha bug\u00fcn ko\u015fuyor, t\u0131rman\u0131yor, z\u0131pl\u0131yor... Tedavi olmadan yapamayaca\u011f\u0131 hareketler! <a href=\"https:\/\/www.afm-telethon.fr\/fr\/evenement-telethon\/le-combat-du-telethon\/les-familles-ambassadrices\/sacha-la-maladie-detruit-mes\" target=\"_blank\" rel=\"noopener\">Devam\u0131n\u0131 oku<\/a>.<\/p>","protected":false},"excerpt":{"rendered":"<p>Genethon has received approvals from regulatory authorities, MHRA and EMA, to begin pivotal Phase 3 clinical trials in France and the UK of its gene therapy, GNT0004, for Duchenne muscular dystrophy (DMD). Genethon CEO Frederic Revah observed, \u201cWe are delighted to be able to continue these trials and are determined to bring GNT0004 to market [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":4246,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[27],"tags":[137,342,105,106,220],"class_list":["post-4242","post","type-post","status-publish","format-standard","has-post-thumbnail","category-research","tag-clinical-trial","tag-frederic-revah","tag-genethon","tag-gnt-0004","tag-gnt0004"],"subtitle":"Genethon GNT0004 Pivotal Klinik Ara\u015ft\u0131rmas\u0131 Faz 3 \u00e7ift k\u00f6r denemeleri, GNT0004'\u00fcn tek bir intraven\u00f6z enjeksiyonu kullan\u0131larak A\u011fustos ve Eyl\u00fcl aylar\u0131nda \u0130ngiltere ve Fransa'da ba\u015flayacak.","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/4242","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=4242"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/4242\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/4246"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=4242"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=4242"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=4242"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}