{"id":8865,"date":"2026-10-14T15:15:01","date_gmt":"2026-10-14T12:15:01","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=8865"},"modified":"2026-10-14T15:28:15","modified_gmt":"2026-10-14T12:28:15","slug":"entr-601-45-dystrophin-production-duchenne","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/entr-601-45-dystrophin-production-duchenne\/","title":{"rendered":"Entrada Therapeutics ENTR-601-45: Erken D\u00f6nem Ekzon 45 Atlama \u00c7al\u0131\u015fmas\u0131, Distrofin Art\u0131\u015f\u0131n\u0131 G\u00f6steriyor"},"content":{"rendered":"<p class=\"wp-block-paragraph\"><strong>Entrada Therapeutics, Duchenne kas distrofisi (Duchenne) hastalar\u0131nda ENTR-601-45'yi de\u011ferlendiren ekzon 45 atlama \u00e7al\u0131\u015fmas\u0131 (ELEVATE-45-201)'ndan olumlu ilk sonu\u00e7lar a\u00e7\u0131klad\u0131.<\/strong> \u0130lk kat\u0131l\u0131mc\u0131 grubu, olumlu bir g\u00fcvenlik profili ve ilk dozu ald\u0131ktan sonra distrofin \u00fcretiminde k\u00fc\u00e7\u00fck bir art\u0131\u015f g\u00f6sterdi. Bulgular, tedavinin de\u011ferlendirilmesinde erken bir ad\u0131md\u0131r; daha y\u00fcksek dozlar\u0131n daha fazla distrofin \u00fcretip \u00fcretemeyece\u011fini de\u011ferlendirmek i\u00e7in planlamalar yap\u0131lmaktad\u0131r.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Faz 1\/2 \u00e7al\u0131\u015fmas\u0131, ekzon 45 atlamaya uygun oldu\u011fu do\u011frulanm\u0131\u015f DMD gen mutasyonlar\u0131na sahip ayakta tedavi g\u00f6ren kat\u0131l\u0131mc\u0131larda ENTR-601-45'i de\u011ferlendirmektedir.<\/strong><\/p>\n\n\n\n<div class=\"wp-block-rank-math-toc-block\" id=\"rank-math-toc\"><h2>\u0130\u00e7indekiler<\/h2><nav><ul><li><a href=\"#what-did-the-first-cohort-show\">\u0130lk d\u00f6nem kat\u0131l\u0131mc\u0131lar\u0131n\u0131n bulgular\u0131 nelerdi?<\/a><ul><li><a href=\"#dystrophin-production-increased-by-0-10\">Distrofin \u00dcretimi 0.10% Kadar Artt\u0131<\/a><\/li><\/ul><\/li><li><a href=\"#safety-and-tolerability-results\">G\u00fcvenlik ve Tolere Edilebilirlik Sonu\u00e7lar\u0131<\/a><ul><li><a href=\"#drug-exposure-matched-expectations\">\u0130la\u00e7 Maruziyeti Beklentilerle Uyumluydu<\/a><\/li><\/ul><\/li><li><a href=\"#what-comes-next-for-entr-601-45\">ENTR-601-45 i\u00e7in s\u0131rada ne var?<\/a><\/li><li><a href=\"#a-potentially-less-frequent-dosing-schedule\">Potansiyel Olarak Daha Az S\u0131kl\u0131kta Dozlama Program\u0131<\/a><\/li><li><a href=\"#conclusion\">Sonu\u00e7<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<h2 id=\"what-did-the-first-cohort-show\" class=\"wp-block-heading\">\u0130lk d\u00f6nem kat\u0131l\u0131mc\u0131lar\u0131n\u0131n bulgular\u0131 nelerdi?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Birinci grupta sekiz kat\u0131l\u0131mc\u0131 yer ald\u0131: alt\u0131s\u0131 5 mg\/kg dozunda ENTR-601-45 al\u0131rken, ikisi plasebo ald\u0131.<\/strong> \u00c7al\u0131\u015fman\u0131n \u00e7oklu artan doz uygulamas\u0131nda kat\u0131l\u0131mc\u0131lara \u00fc\u00e7 doz a\u015f\u0131 yap\u0131ld\u0131.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Sonu\u00e7lar, \u00e7al\u0131\u015fman\u0131n g\u00fcvenlik ve tolere edilebilirlik a\u00e7\u0131s\u0131ndan birincil hedeflerini kar\u015f\u0131lad\u0131. \u0130kincil hedefler ayr\u0131ca, tedavi alan kat\u0131l\u0131mc\u0131lar aras\u0131nda distrofin \u00fcretiminde ve ekzon atlamas\u0131nda de\u011fi\u015fiklikler oldu\u011funu da g\u00f6sterdi.<\/p>\n\n\n\n<h3 id=\"dystrophin-production-increased-by-0-10\" class=\"wp-block-heading\">Distrofin \u00dcretimi 0.10% Kadar Artt\u0131<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">En \u00f6nemli bulgu, ENTR-601-45 ile tedavi edilen alt\u0131 kat\u0131l\u0131mc\u0131 aras\u0131nda distrofin \u00fcretiminde ba\u015flang\u0131\u00e7 de\u011ferine g\u00f6re ortalama 0,10%&#039;lik bir art\u0131\u015f olmas\u0131yd\u0131.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Buna kar\u015f\u0131l\u0131k, plasebo grubunda distrofin d\u00fczeyinde ba\u015flang\u0131\u00e7 de\u011ferine g\u00f6re ortalama 0,02%&#039;lik bir azalma g\u00f6zlendi.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Tedavi g\u00f6ren grupta, plasebo grubundaki ortalama 0,04%'lik azalmaya k\u0131yasla, ekzon atlamas\u0131nda ortalama 0,15%'lik bir art\u0131\u015f da g\u00f6zlemlendi.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Bu bulgular, ENTR-601-45&#039;nin ama\u00e7lanan biyolojik etkileri \u00fcretti\u011fine dair erken bir g\u00f6sterge sa\u011flamaktad\u0131r. Bununla birlikte, bu ba\u015flang\u0131\u00e7 dozunda g\u00f6zlemlenen distrofin \u00fcretimindeki art\u0131\u015f k\u00fc\u00e7\u00fckt\u00fcr ve daha y\u00fcksek dozlar\u0131n etkilerini anlamak i\u00e7in daha fazla \u00e7al\u0131\u015fmaya ihtiya\u00e7 vard\u0131r.<\/p>\n\n\n\n<h2 id=\"safety-and-tolerability-results\" class=\"wp-block-heading\">G\u00fcvenlik ve Tolere Edilebilirlik Sonu\u00e7lar\u0131<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">ENTR-601-45, 5 mg\/kg&#039;l\u0131k ba\u015flang\u0131\u00e7 dozunda olumlu bir g\u00fcvenlik ve tolere edilebilirlik profili g\u00f6stermi\u015ftir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Ba\u015f a\u011fr\u0131s\u0131, iki kat\u0131l\u0131mc\u0131da be\u015f kez bildirilen en yayg\u0131n tedaviyle ili\u015fkili yan etkiydi. Bir kat\u0131l\u0131mc\u0131da kusma ve ate\u015f olmak \u00fczere iki ciddi tedaviyle ili\u015fkili yan etki g\u00f6r\u00fcld\u00fc. Her iki olay da daha sonra d\u00fczeldi.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u00c7al\u0131\u015fmaya kat\u0131lanlardan hi\u00e7biri \u00e7al\u0131\u015fmay\u0131 b\u0131rakmad\u0131 ve hepsi a\u00e7\u0131k etiketli uzatma \u00e7al\u0131\u015fmas\u0131na kaydoldu.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u015eirket ayr\u0131ca d\u00fc\u015f\u00fck magnezyum seviyeleri veya b\u00f6brek g\u00fcvenli\u011fi ile ilgili herhangi bir endi\u015fe bildirmedi. Tedavi g\u00f6ren kat\u0131l\u0131mc\u0131larda tahmini glomer\u00fcler filtrasyon h\u0131z\u0131 (eGFR) ve sistatin C dahil olmak \u00fczere b\u00f6brek belirte\u00e7leri normal aral\u0131klardayd\u0131. Magnezyum seviyeleri de normal aral\u0131ktayd\u0131.<\/p>\n\n\n\n<h3 id=\"drug-exposure-matched-expectations\" class=\"wp-block-heading\">\u0130la\u00e7 Maruziyeti Beklentilerle Uyumluydu<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">Kanda \u00f6l\u00e7\u00fclen ENTR-601-45 d\u00fczeyleri, gen\u00e7 insan d\u0131\u015f\u0131 primatlar \u00fczerinde yap\u0131lan \u00f6nceki \u00e7al\u0131\u015fmalara dayal\u0131 tahminlerle tutarl\u0131yd\u0131.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u015eirketin a\u00e7\u0131klamas\u0131na g\u00f6re, bu sonu\u00e7lar daha y\u00fcksek dozlar\u0131n daha fazla ila\u00e7 maruziyetine ve artan distrofin \u00fcretimine yol a\u00e7abilece\u011fi y\u00f6n\u00fcndeki beklentilerini destekliyor.<\/p>\n\n\n\n<h2 id=\"what-comes-next-for-entr-601-45\" class=\"wp-block-heading\">ENTR-601-45 i\u00e7in s\u0131rada ne var?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u015eirket, ENTR-601-45&#039;nin distrofin \u00fcretiminde daha \u00f6nemli bir art\u0131\u015f sa\u011flay\u0131p sa\u011flayamayaca\u011f\u0131n\u0131 ve potansiyel olarak i\u015flevsel faydalar sa\u011flay\u0131p sa\u011flayamayaca\u011f\u0131n\u0131 belirlemek i\u00e7in daha y\u00fcksek dozlar\u0131n kullan\u0131lmas\u0131n\u0131 bekliyor.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>10 mg\/kg dozun de\u011ferlendirildi\u011fi ikinci kohortun kay\u0131t i\u015flemleri tamamland\u0131. Entrada Therapeutics, bu kohorttan elde edilen verilerin 2027 y\u0131l\u0131n\u0131n ilk yar\u0131s\u0131nda a\u00e7\u0131klanmas\u0131n\u0131 bekliyor.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">15 mg\/kg&#039;a kadar olan dozlar\u0131 de\u011ferlendirmek \u00fczere \u00fc\u00e7\u00fcnc\u00fc bir kohort planlanmaktad\u0131r.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Birinci gruptaki kat\u0131l\u0131mc\u0131lar ayr\u0131ca \u00e7al\u0131\u015fman\u0131n a\u00e7\u0131k etiketli Faz 2 a\u015famas\u0131na da ge\u00e7tiler ve burada 5 mg\/kg dozunda alt\u0131 doz ENTR-601-45 alacaklar.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u0130lk kohortta \u00fc\u00e7 dozdan sonra g\u00f6zlemlenebilir bir fonksiyonel fayda beklenmedi veya bildirilmedi. Tedavinin fonksiyonel etkisi, gelecekteki kohortlarda ve a\u00e7\u0131k etiketli \u00e7al\u0131\u015fmalarda daha y\u00fcksek dozlarda ve daha uzun tedavi s\u00fcreleri boyunca de\u011ferlendirilecektir. Daha Fazla Bilgi Edinin: <a href=\"https:\/\/dmdwarrior.com\/tr\/duchenne-exon-deletion-tool\/\" target=\"_blank\" rel=\"noopener\">DMDWarrior Ekzon Kontrol Arac\u0131<\/a><\/p>\n\n\n\n<h2 id=\"a-potentially-less-frequent-dosing-schedule\" class=\"wp-block-heading\">Potansiyel Olarak Daha Az S\u0131kl\u0131kta Dozlama Program\u0131<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">ENTR-601-45, alt\u0131 haftada bir uygulanmak \u00fczere tasarlanm\u0131\u015ft\u0131r. Bu uygulama program\u0131, haftal\u0131k tedavi program\u0131ndaki 50&#039;den fazla uygulamaya k\u0131yasla y\u0131lda yakla\u015f\u0131k dokuz uygulama anlam\u0131na gelebilir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Ara\u015ft\u0131r\u0131lan tedavinin \u00f6nemli bir y\u00f6n\u00fc, daha seyrek dozlama potansiyelidir. Bununla birlikte, uzun vadeli g\u00fcvenli\u011fi, etkinli\u011fi ve fonksiyonel faydalar\u0131 daha fazla klinik \u00e7al\u0131\u015fma ile belirlenmelidir.<\/p>\n\n\n\n<h2 id=\"conclusion\" class=\"wp-block-heading\">Sonu\u00e7<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">ELEVATE-45-201 \u00e7al\u0131\u015fmas\u0131n\u0131n ilk kohortu, ba\u015flang\u0131\u00e7taki g\u00fcvenlik ve tolere edilebilirlik hedeflerine ula\u015ft\u0131 ve 5 mg\/kg dozunda ENTR-601-45 ile distrofin \u00fcretiminde ortalama 0,10%'lik bir art\u0131\u015f g\u00f6sterdi. Ekzon atlamas\u0131 da ba\u015flang\u0131\u00e7 de\u011ferine g\u00f6re artt\u0131.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Bunlar, klinik veya fonksiyonel faydan\u0131n kesin kan\u0131t\u0131 de\u011fil, erken d\u00f6nem biyolojik bulgulard\u0131r. Bir sonraki \u00f6nemli d\u00f6n\u00fcm noktas\u0131, 2027 y\u0131l\u0131n\u0131n ilk yar\u0131s\u0131nda beklenilen 10 mg\/kg&#039;l\u0131k grupla ba\u015flayarak, daha y\u00fcksek dozlardan elde edilecek sonu\u00e7lar olacakt\u0131r.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Daha Fazlas\u0131n\u0131 Ke\u015ffedin<\/strong>:&nbsp;<a href=\"https:\/\/dmdwarrior.com\/clinical-trials\/map\/\" target=\"_blank\" rel=\"noopener\">Duchenne Klinik Ara\u015ft\u0131rma Yerleri Haritas\u0131<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>Entrada Therapeutics has announced positive initial results from its Exon 45 Skipping Study, ELEVATE-45-201, evaluating ENTR-601-45 in people with Duchenne muscular dystrophy (Duchenne). The first group of participants showed a favorable safety profile and a small increase in dystrophin production after receiving the initial dose. The findings are an early step in evaluating the treatment, [&hellip;]<\/p>\n","protected":false},"author":6,"featured_media":8867,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[320,321,233,234,317,318,319],"class_list":["post-8865","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-elevate-45-201","tag-entr-601-45","tag-entrada","tag-entrada-therapeutics","tag-exon-45","tag-exon-45-skipping","tag-exon-45-skipping-therapy"],"subtitle":"Entrada Therapeutics, Duchenne hastal\u0131\u011f\u0131nda ENTR-601-45'nin erken sonu\u00e7lar\u0131n\u0131 rapor etmektedir. ELEVATE-45-201 \u00e7al\u0131\u015fmas\u0131, distrofin \u00fcretiminde ortalama 0,10%'lik bir art\u0131\u015f g\u00f6sterdi ve daha b\u00fcy\u00fck etkileri ara\u015ft\u0131rmak i\u00e7in daha y\u00fcksek dozlar planlanmaktad\u0131r.","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/8865","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\/6"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/comments?post=8865"}],"version-history":[{"count":3,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/8865\/revisions"}],"predecessor-version":[{"id":8870,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/8865\/revisions\/8870"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/8867"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=8865"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=8865"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=8865"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}