{"id":3088,"date":"2025-03-07T12:49:51","date_gmt":"2025-03-07T09:49:51","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=3088"},"modified":"2025-03-08T13:35:34","modified_gmt":"2025-03-08T10:35:34","slug":"solid-biosciences-plans-meeting-with-fda-in-mid-2025-for-sgt-003-gene-therapy","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/solid-biosciences-plans-meeting-with-fda-in-mid-2025-for-sgt-003-gene-therapy\/","title":{"rendered":"Solid Biosciences, SGT-003 i\u00e7in Olas\u0131 H\u0131zland\u0131r\u0131lm\u0131\u015f Onay Yollar\u0131n\u0131 Tart\u0131\u015fmak \u00dczere 2025 Ortas\u0131nda FDA ile Bir Toplant\u0131 Talep Etmeyi Planl\u0131yor"},"content":{"rendered":"<p class=\"wp-block-paragraph\">N\u00f6rom\u00fcsk\u00fcler ve kalp hastal\u0131klar\u0131 i\u00e7in hassas, yeni nesil genetik ila\u00e7lar geli\u015ftiren bir ya\u015fam bilimleri \u015firketi olan Solid Biosciences, 31 Aral\u0131k 2024&#039;te sona eren d\u00f6rd\u00fcnc\u00fc \u00e7eyrek ve tam y\u0131l i\u00e7in mali sonu\u00e7lar\u0131n\u0131 bildirdi ve bir i\u015f g\u00fcncellemesi sa\u011flad\u0131.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Solid Ba\u015fkan\u0131 ve CEO&#039;su Bo Cumbo \u015funlar\u0131 s\u00f6yledi: &quot;Son 18 ayda Duchenne ve FA geli\u015ftirme programlar\u0131m\u0131z\u0131n titizlikle y\u00fcr\u00fct\u00fclmesi Solid&#039;i d\u00f6n\u00fc\u015ft\u00fcrd\u00fc ve bize 2025 ve sonras\u0131na do\u011fru muazzam bir ivme kazand\u0131rd\u0131. Yeni nesil Duchenne kas distrofisi (Duchenne) program\u0131m\u0131z SGT-003&#039;\u00fcn Faz 1\/2 INSPIRE DUCHENNE denemesinin ilk \u00fc\u00e7 kat\u0131l\u0131mc\u0131s\u0131ndan gelen ilk 90 g\u00fcnl\u00fck veriler, erken olmas\u0131na ra\u011fmen g\u00fcven verici bir g\u00fcvenlik deneyimi ve kas b\u00fct\u00fcnl\u00fc\u011f\u00fc ve sa\u011fl\u0131\u011f\u0131n\u0131n biyobelirte\u00e7lerinde umut verici geli\u015fmeler g\u00f6sterdi. Bu erken g\u00f6stergelerin SGT-003&#039;\u00fcn Duchenne&#039;i tedavi etmek i\u00e7in s\u0131n\u0131f\u0131n\u0131n en iyisi bir gen tedavisi aday\u0131 olma potansiyelini destekledi\u011fine inan\u0131yoruz. Duchenne hasta topluluklar\u0131nda bu veriler etraf\u0131ndaki heyecan\u0131 anl\u0131yoruz ve SGT-003&#039;\u00fc m\u00fcmk\u00fcn olan en k\u0131sa s\u00fcrede klini\u011fe getirmek i\u00e7in ama\u00e7 ve aciliyetle hareket etmeye kararl\u0131y\u0131z.&quot; (<a href=\"https:\/\/dmdwarrior.com\/tr\/solid-biosciences-reports-positive-initial-clinical-data-from-next-generation-duchenne-gene-therapy-candidate-sgt-003\/\" target=\"_blank\" rel=\"noreferrer noopener\">Devam\u0131n\u0131 oku<\/a>)<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">SGT-003&#039;ten Pozitif \u0130lk Klinik Veriler<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u00c7ekirdek ba\u015f\u0131na y\u00fcksek vekt\u00f6r genom kopyalar\u0131, g\u00fc\u00e7l\u00fc mikrodistrofin ekspresyonu ve kas b\u00fct\u00fcnl\u00fc\u011f\u00fcn\u00fcn ek \u00f6l\u00e7\u00fcmlerinde erken geli\u015fmeler g\u00f6zlemlendi, bunlar \u015funlar\u0131 i\u00e7erir:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u00c7ekirdek ba\u015f\u0131na ortalama vekt\u00f6r kopyas\u0131: 18,7 (N=3),<\/li>\n\n\n\n<li>Ortalama mikrodistrofin ekspresyonu: Western blot ile \u00f6l\u00e7\u00fclen 110% (N=3),<\/li>\n\n\n\n<li>Ortalama mikrodistrofin ekspresyonu: 108% (N=3), k\u00fctle spektrometrisi ile \u00f6l\u00e7\u00fcld\u00fc\u011f\u00fc gibi,<\/li>\n\n\n\n<li>Ortalama distrofin pozitif lif y\u00fczdesi: 78% (N=3), imm\u00fcnofloresanla \u00f6l\u00e7\u00fclm\u00fc\u015ft\u00fcr,<\/li>\n\n\n\n<li>Ortalama beta sarkoglikan y\u00fczde pozitif lifler: 70% (N=3),<\/li>\n\n\n\n<li>Ortalama nNOS (n\u00f6ronal nitrik oksit sentaz) pozitif lif y\u00fczdesi: 42% (N=3),<\/li>\n\n\n\n<li>7 ek kas b\u00fct\u00fcnl\u00fc\u011f\u00fc biyobelirtecinde (N=3) iyile\u015ftirmeler ve<\/li>\n\n\n\n<li>Ba\u015flang\u0131\u00e7 seviyesine g\u00f6re 180. g\u00fcnde sol ventrik\u00fcl ejeksiyon fraksiyonunda (LVEF) 8%&#039;lik erken ortalama iyile\u015fme (N=2).<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">Daha fazla bilgi edin: <a href=\"https:\/\/dmdwarrior.com\/tr\/faq-solid-biosciences-sgt-003-gene-therapy\/\" target=\"_blank\" rel=\"noreferrer noopener\">Solid Biosciences SGT-003 Gen Terapisi Hakk\u0131nda S\u0131k\u00e7a Sorulan Sorular<\/a><\/p>\n\n\n\n<h2 class=\"wp-block-heading\">SGT-003 i\u00e7in olas\u0131 h\u0131zland\u0131r\u0131lm\u0131\u015f onay yollar\u0131n\u0131 g\u00f6r\u00fc\u015fmek \u00fczere FDA ile toplant\u0131<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>SGT-003, 11 \u015eubat 2025 veri kesme tarihi itibariyle doz uygulanan 6 kat\u0131l\u0131mc\u0131da iyi tolere edilmi\u015f olup, ciddi yan etki (SAE), \u015f\u00fcpheli beklenmeyen ciddi yan etki (SUSAR) veya akut karaci\u011fer hasar\u0131 AE&#039;si g\u00f6zlenmemi\u015ftir.<\/li>\n\n\n\n<li>Denemeye kat\u0131l\u0131mlar devam ediyor ve \u015eirket, 2025 y\u0131l\u0131n\u0131n ikinci \u00e7eyre\u011finin ba\u015flar\u0131nda toplam 10&#039;dan fazla kat\u0131l\u0131mc\u0131ya, 2025 y\u0131l\u0131n\u0131n d\u00f6rd\u00fcnc\u00fc \u00e7eyre\u011finde ise yakla\u015f\u0131k 20 toplam kat\u0131l\u0131mc\u0131ya doz vermeyi bekliyor.<\/li>\n\n\n\n<li><strong>\u015eirket, SGT-003 i\u00e7in potansiyel h\u0131zland\u0131r\u0131lm\u0131\u015f onay yollar\u0131n\u0131 g\u00f6r\u00fc\u015fmek \u00fczere FDA ile 2025 y\u0131l\u0131n\u0131n ortalar\u0131nda bir toplant\u0131 talep etmeyi planl\u0131yor.<\/strong><\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Daha fazla bilgi edin:<\/strong>&nbsp;<a href=\"https:\/\/dmdwarrior.com\/tr\/new-gene-therapies-for-duchenne-muscular-dystrophy\/\" target=\"_blank\" rel=\"noreferrer noopener\">Duchenne Musk\u00fcler Distrofisi \u0130\u00e7in Umut Veren Yeni Gen Terapileri<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>Solid Biosciences, a life sciences company developing precision, next generation, genetic medicines for neuromuscular and cardiac diseases, reported financial results for the fourth quarter and full year ended December 31, 2024, and provided a business update. Bo Cumbo, President and CEO of Solid, commented: &#8220;The diligent execution of our Duchenne and FA development programs over [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":3090,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[88,61,110,170,109],"class_list":["post-3088","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-fda","tag-gene-therapy","tag-sgt-003","tag-sgt-003-gene-therapy","tag-solid-biosciences"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/3088","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=3088"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/3088\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/3090"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=3088"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=3088"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=3088"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}