{"id":2961,"date":"2025-02-18T15:46:12","date_gmt":"2025-02-18T12:46:12","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=2961"},"modified":"2025-03-07T12:57:05","modified_gmt":"2025-03-07T09:57:05","slug":"solid-biosciences-reports-positive-initial-clinical-data-from-next-generation-duchenne-gene-therapy-candidate-sgt-003","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/solid-biosciences-reports-positive-initial-clinical-data-from-next-generation-duchenne-gene-therapy-candidate-sgt-003\/","title":{"rendered":"Solid Biosciences, Yeni Nesil Duchenne Gen Terapisi Aday\u0131 SGT-003&#039;ten Pozitif \u0130lk Klinik Veriler Bildirdi"},"content":{"rendered":"<p class=\"wp-block-paragraph\">N\u00f6rom\u00fcsk\u00fcler ve kardiyak hastal\u0131klar i\u00e7in hassas genetik ila\u00e7lar geli\u015ftiren bir ya\u015fam bilimleri \u015firketi olan Solid Biosciences, bug\u00fcn Duchenne kas distrofisi (Duchenne) tedavisi i\u00e7in tasarlanm\u0131\u015f yeni nesil bir gen terapisi \u00fcr\u00fcn\u00fc aday\u0131 olan SGT-003&#039;\u00fc de\u011ferlendiren Faz 1\/2 INSPIRE DUCHENNE denemesinden olumlu ilk verileri duyurdu. \u0130lk \u00fc\u00e7 kat\u0131l\u0131mc\u0131da bildirilen ge\u00e7ici 90 g\u00fcnl\u00fck biyopsi verileri, western blot ile \u00f6l\u00e7\u00fcld\u00fc\u011f\u00fcnde 110%&#039;lik ortalama bir mikrodistrofin ekspresyonu ve kas sa\u011fl\u0131\u011f\u0131 ve dayan\u0131kl\u0131l\u0131\u011f\u0131n\u0131n g\u00f6stergeleri olan birden fazla biyobelirte\u00e7te iyile\u015fmeler g\u00f6sterdi.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Duchenne Gen Terapisi Aday\u0131 SGT-003&#039;\u00fcn Klinik Verileri<\/h2>\n\n\n\n<ol class=\"wp-block-list\">\n<li>110%&#039;nin (N=3) ortalama mikrodistrofin ekspresyonu ve g\u00f6zlemlenen \u00e7ok say\u0131da ek kas sa\u011fl\u0131\u011f\u0131 biyobelirtecinde \u00f6nemli geli\u015fmeler, SGT-003&#039;\u00fcn yeni nesil, s\u0131n\u0131f\u0131n\u0131n en iyisi Duchenne kas distrofisi gen tedavisi aday\u0131 olma potansiyelini desteklemektedir.<\/li>\n\n\n\n<li>Potansiyel kardiyak faydan\u0131n erken sinyallerinin g\u00f6zlenmesi cesaret vericidir.<\/li>\n\n\n\n<li>SGT-003, 11 \u015eubat 2025 itibariyle doz uygulanan 6 kat\u0131l\u0131mc\u0131da iyi tolere edilmi\u015f olup, ciddi bir yan etki g\u00f6zlenmemi\u015ftir.<\/li>\n\n\n\n<li>Kat\u0131l\u0131mc\u0131 kay\u0131tlar\u0131 devam etmekte olup, 7. kat\u0131l\u0131mc\u0131ya 17 \u015eubat 2025 tarihinde doz uygulanm\u0131\u015ft\u0131r; \u015eirket, 2025&#039;in 4. \u00e7eyre\u011fine kadar toplam yakla\u015f\u0131k 20 kat\u0131l\u0131mc\u0131ya doz uygulamay\u0131 beklemektedir.<\/li>\n<\/ol>","protected":false},"excerpt":{"rendered":"<p>Solid Biosciences, a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today announced positive initial data from the Phase 1\/2 INSPIRE DUCHENNE trial evaluating SGT-003, a next-generation gene therapy product candidate intended for the treatment of Duchenne muscular dystrophy (Duchenne). Interim 90-day biopsy data reported in the first three participants showed [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":2963,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[137,110,170,109],"class_list":["post-2961","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-clinical-trial","tag-sgt-003","tag-sgt-003-gene-therapy","tag-solid-biosciences"],"subtitle":"Klinik a\u015famada bir biyoteknoloji \u015firketi olan Solid Biosciences, Duchenne kas distrofisi i\u00e7in yeni nesil bir gen terapisi olan SGT-003'\u00fc de\u011ferlendiren Faz 1\/2 INSPIRE DUCHENNE \u00e7al\u0131\u015fmas\u0131ndan elde edilen olumlu ilk verileri payla\u015ft\u0131.","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/2961","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=2961"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/2961\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/2963"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=2961"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=2961"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=2961"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}