{"id":5470,"date":"2026-01-14T10:57:27","date_gmt":"2026-01-14T07:57:27","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=5470"},"modified":"2026-01-14T10:58:30","modified_gmt":"2026-01-14T07:58:30","slug":"solid-biosciences-announces-2026-outlook-for-clinical-trials-of-duchenne-gene-therapy-sgt-003","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/solid-biosciences-announces-2026-outlook-for-clinical-trials-of-duchenne-gene-therapy-sgt-003\/","title":{"rendered":"Solid Biosciences, Duchenne Gen Terapisi SGT-003&#039;\u00fcn Klinik Denemeleri \u0130\u00e7in 2026 Beklentilerini A\u00e7\u0131klad\u0131"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Solid Biosciences taraf\u0131ndan yay\u0131nlanan bas\u0131n b\u00fclteninde \u015fu ifadeler yer ald\u0131: &quot;\u00d6n\u00fcm\u00fczdeki aylarda planlanan \u00e7ok say\u0131da d\u00fczenleyici kurumla etkile\u015fimimizle, SGT-003 i\u00e7in potansiyel h\u0131zland\u0131r\u0131lm\u0131\u015f onay yolunda uzla\u015fmaya varmay\u0131 ve ara\u015ft\u0131rma a\u015famas\u0131ndaki tedavimizi Duchenne toplulu\u011fundan gelen yo\u011fun talebi kar\u015f\u0131lamak \u00fczere piyasaya s\u00fcrmeyi hedefliyoruz.&quot;\u201c <strong>Daha fazla bilgi edin:<\/strong> <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\">ABD&#039;de Duchenne kas distrofisi (Duchenne) i\u00e7in SGT-003<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>9 Ocak 2026 tarihli g\u00fcvenlik son tarihine kadar, SGT-003, devam eden INSPIRE DUCHENNE Faz 1\/2 klinik \u00e7al\u0131\u015fmas\u0131na kat\u0131lan 33 kat\u0131l\u0131mc\u0131da genel olarak iyi tolere edilmi\u015ftir.\n<ul class=\"wp-block-list\">\n<li>SGT-003&#039;\u00fcn g\u00fcvenlik ve tolere edilebilirlik profili, yaln\u0131zca steroid i\u00e7eren profilaktik imm\u00fcnomod\u00fclasyon rejimini desteklemeye devam etmektedir.<\/li>\n\n\n\n<li>Ayakta tedavi g\u00f6ren hastalara doz uygulamas\u0131 Eyl\u00fcl 2025&#039;ten itibaren m\u00fcmk\u00fcn hale geldi.<\/li>\n\n\n\n<li>9 Ocak 2026 tarihine kadar ila\u00e7 kaynakl\u0131 karaci\u011fer hasar\u0131 (DILI), trombotik mikroanjiyopati (TMA), atipik hemolitik \u00fcremik sendrom (aHUS) veya miyokardit vakas\u0131 g\u00f6zlemlenmemi\u015ftir.<\/li>\n\n\n\n<li>Kalp g\u00fcvenli\u011fi izlemesi, sol ventrik\u00fcl ejeksiyon fraksiyonu (LVEF) ile \u00f6l\u00e7\u00fclen kalp hasar\u0131nda azalma ve kalp sistolik fonksiyonunun normalle\u015fmesinin erken sinyallerini g\u00f6stermi\u015ftir (N=14, veri kesim tarihi 29 Eyl\u00fcl 2025).<\/li>\n<\/ul>\n<\/li>\n\n\n\n<li>Dikkat \u00e7ekici mikrodistrofin ekspresyon seviyeleri (N=10, veri kesim tarihi 29 Eyl\u00fcl 2025) ve distrofinle ili\u015fkili protein kompleksinin (DAPC) temel bile\u015fenlerinin uyumlu \u015fekilde yeniden olu\u015fumu g\u00f6zlemlenmi\u015ftir.<\/li>\n\n\n\n<li>Kas b\u00fct\u00fcnl\u00fc\u011f\u00fcne ili\u015fkin \u00e7e\u015fitli biyobelirte\u00e7lerde iyile\u015fmeler g\u00f6zlemlenmi\u015ftir; bu da koordineli bir a\u015fa\u011f\u0131 y\u00f6nl\u00fc etkiyi d\u00fc\u015f\u00fcnd\u00fcrmektedir (N=11-14, veri kesim tarihi 29 Eyl\u00fcl 2025).<\/li>\n\n\n\n<li>\u015eirket, 2026 y\u0131l\u0131n\u0131n ilk yar\u0131s\u0131nda, Faz 3 do\u011frulama denemesi tasar\u0131m\u0131 ve SGT-003 i\u00e7in potansiyel h\u0131zland\u0131r\u0131lm\u0131\u015f onay s\u00fcrecini desteklemek \u00fczere gerekli do\u011frulama kan\u0131tlar\u0131 konusunda ABD G\u0131da ve \u0130la\u00e7 \u0130daresi (FDA) ile birden fazla g\u00f6r\u00fc\u015fme yapmay\u0131 planlamaktad\u0131r; 2026 y\u0131l\u0131n\u0131n ortalar\u0131nda bir g\u00fcncelleme beklenmektedir.<\/li>\n\n\n\n<li>\u015eirket, Faz 1\/2 INSPIRE DUCHENNE \u00e7al\u0131\u015fmas\u0131ndan elde edilen ek verileri 2026 y\u0131l\u0131n\u0131n ortalar\u0131nda a\u00e7\u0131klamay\u0131 bekliyor.<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Daha Fazlas\u0131n\u0131 Ke\u015ffedin:<\/strong> <a href=\"https:\/\/dmdwarrior.com\/tr\/solid-biosciences-sgt-003-gene-therapy-phase-3-clinical-trial-nct07160634\/\" target=\"_blank\" rel=\"noreferrer noopener\">Solid Biosciences, Hareket Halindeki Erkeklerde SGT-003 Gen Terapisinin (IMPACT DUCHENNE) 3. Faz Klinik \u00c7al\u0131\u015fmas\u0131n\u0131 Ba\u015flatt\u0131<\/a><\/p>\n\n\n\n<h2 class=\"wp-block-heading\">SGT-003&#039;\u00fcn ABD d\u0131\u015f\u0131nda klinik denemesi<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>IMPACT DUCHENNE adl\u0131 Faz 3 randomize, \u00e7ift k\u00f6r, plasebo kontroll\u00fc, ABD d\u0131\u015f\u0131 klinik \u00e7al\u0131\u015fmaya ilk kat\u0131l\u0131mc\u0131 kaydedildi ve dozlaman\u0131n 2026 y\u0131l\u0131n\u0131n ilk \u00e7eyre\u011finde ger\u00e7ekle\u015fmesi bekleniyor.<\/li>\n\n\n\n<li>IMPACT DUCHENNE \u00e7al\u0131\u015fmas\u0131n\u0131n Kanada ve Avustralya&#039;da olmak \u00fczere iki aktif klinik ara\u015ft\u0131rma merkezi bulunmaktad\u0131r ve d\u00fczenleyici onaylar\u0131n al\u0131nmas\u0131na ba\u011fl\u0131 olarak, 2026 y\u0131l\u0131n\u0131n ortalar\u0131ndan itibaren Avrupa da dahil olmak \u00fczere ek \u00fclkelere geni\u015fletilmesi planlanmaktad\u0131r.<\/li>\n\n\n\n<li>SGT-003, yeni Birle\u015fik Krall\u0131k Yenilik\u00e7i Lisanslama ve Eri\u015fim Yolu (ILAP) kapsam\u0131nda Yenilik\u00e7i Pasaport Belgesi almaya hak kazand\u0131. Bu belge, piyasaya s\u00fcr\u00fclme s\u00fcresini h\u0131zland\u0131rmay\u0131 ve hastalar\u0131n yeni ila\u00e7lara eri\u015fimini kolayla\u015ft\u0131rmay\u0131 ama\u00e7l\u0131yor ve SGT-003&#039;\u00fc potansiyel olarak Birle\u015fik Krall\u0131k&#039;ta piyasaya s\u00fcr\u00fclen ilk Duchenne gen terapisi haline getiriyor. <a href=\"https:\/\/dmdwarrior.com\/tr\/solid-biosciences-sgt-003-innovation-passport-designation-licensing\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u0130novasyon Pasaportu Belgesi<\/a><\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Devam\u0131n\u0131 oku<\/strong>:&nbsp;<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>In a press release issued by Solid Biosciences, it was stated that &#8220;With multiple regulatory interactions planned in the coming months, we aim to achieve alignment on a potential accelerated approval pathway for SGT-003 and to bring our investigational therapy to market to meet the overwhelming demand from the Duchenne community.\u201d&#8221; Learn More: Frequently Asked [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":5473,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[66,61,110,170,109],"class_list":["post-5470","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-duchenne-muscular-dystrophy","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\/5470","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=5470"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/5470\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/5473"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=5470"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=5470"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=5470"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}