{"id":8859,"date":"2026-10-14T14:50:29","date_gmt":"2026-10-14T11:50:29","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=8859"},"modified":"2026-10-14T15:34:05","modified_gmt":"2026-10-14T12:34:05","slug":"entrada-therapeutics-entr-601-44-highest-dose-dmd-trial","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/entrada-therapeutics-entr-601-44-highest-dose-dmd-trial\/","title":{"rendered":"Entrada Therapeutics, Duchenne Ekzon 44 Atlama \u00c7al\u0131\u015fmas\u0131nda ENTR-601-44'yi 18 mg\/kg'a Y\u00fckseltiyor"},"content":{"rendered":"<p class=\"wp-block-paragraph\"><strong>Entrada Therapeutics, Duchenne kas distrofisi (DMD) i\u00e7in ekzon 44 atlama \u00e7al\u0131\u015fmas\u0131nda en y\u00fcksek dozu test etme iznini ald\u0131.<\/strong> Ba\u011f\u0131ms\u0131z Veri \u0130zleme Komitesi (DMC), 2. Kohorttaki kat\u0131l\u0131mc\u0131lardan elde edilen mevcut g\u00fcvenlik ve farmakokinetik verilerini inceledikten sonra, ELEVATE-44-201 klinik \u00e7al\u0131\u015fmas\u0131nda 3. Kohorta 18 mg\/kg dozunda ba\u015flanmas\u0131n\u0131 \u00f6nermi\u015ftir.<\/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=\"#entrada-advances-to-the-18-mg-kg-dose\">Entrada, 18 mg\/kg Dozuna Ge\u00e7iyor<\/a><\/li><li><a href=\"#what-is-entr-601-44\">ENTR-601-44 Nedir?<\/a><\/li><li><a href=\"#what-does-the-higher-dose-mean\">Daha y\u00fcksek doz ne anlama geliyor?<\/a><\/li><li><a href=\"#when-will-more-data-be-available\">Daha fazla veri ne zaman kullan\u0131ma sunulacak?<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<h2 id=\"entrada-advances-to-the-18-mg-kg-dose\" class=\"wp-block-heading\">Entrada, 18 mg\/kg Dozuna Ge\u00e7iyor<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">DMC, \u00e7ift k\u00f6r, plasebo kontroll\u00fc, \u00e7oklu artan doz \u00e7al\u0131\u015fmas\u0131n\u0131n 2. Kohortuna kay\u0131tl\u0131 sekiz kat\u0131l\u0131mc\u0131n\u0131n t\u00fcm\u00fcnden elde edilen g\u00fcvenlik ve farmakokinetik (PK) verilerini inceledi. Daha fazla bilgi edinin: <a href=\"https:\/\/dmdwarrior.com\/tr\/duchenne-exon-deletion-tool\/\" target=\"_blank\" rel=\"noreferrer noopener\">DMDWarrior Ekzon Kontrol Arac\u0131<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Bu incelemenin ard\u0131ndan komite, \u00e7al\u0131\u015fmada \u015fimdiye kadar planlanan en y\u00fcksek doz olan 18 mg\/kg ile 3. Kohorta ge\u00e7ilmesini tavsiye etti.<\/strong> Bu, 2. gruptaki 12 mg\/kg ve 1. gruptaki 6 mg\/kg de\u011ferlerine g\u00f6re bir art\u0131\u015f\u0131 temsil etmektedir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">ELEVATE-44-201, ekzon 44 atlamaya uygun oldu\u011fu do\u011frulanm\u0131\u015f DMD gen mutasyonuna sahip Duchenne hastalar\u0131nda ENTR-601-44'yi de\u011ferlendiren bir Faz 1\/2 klinik \u00e7al\u0131\u015fmas\u0131d\u0131r.<\/p>\n\n\n\n<h2 id=\"what-is-entr-601-44\" class=\"wp-block-heading\">ENTR-601-44 Nedir?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">ENTR-601-44, Entrada Therapeutics taraf\u0131ndan, ekzon 44 atlamas\u0131na uygun mutasyonlara sahip Duchenne hastalar\u0131 i\u00e7in geli\u015ftirilen deneysel bir tedavidir. Bu grup, d\u00fcnya genelindeki Duchenne hasta pop\u00fclasyonunun yakla\u015f\u0131k 'ini temsil etmektedir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Bu tedavi, v\u00fccudun do\u011fal olarak olu\u015fan DMD geninin i\u015flevsel distrofin \u00fcretmesine yard\u0131mc\u0131 olmak \u00fczere tasarlanm\u0131\u015f, Endozomal Ka\u00e7\u0131\u015f Arac\u0131 (EEV\u2122) ile konjuge edilmi\u015f bir oligon\u00fckleotid kullan\u0131r.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Distrofin, kas fonksiyonu i\u00e7in \u00f6nemli bir proteindir ve Duchenne kas distrofisi, \u00fcretimini veya i\u015flevini etkileyen DMD genindeki mutasyonlardan kaynaklan\u0131r.<\/p>\n\n\n\n<h2 id=\"what-does-the-higher-dose-mean\" class=\"wp-block-heading\">Daha y\u00fcksek doz ne anlama geliyor?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Entrada Therapeutics Ara\u015ft\u0131rma ve Geli\u015ftirme Ba\u015fkan\u0131 Dr. Natarajan Sethuraman&#039;a g\u00f6re, ENTR-601-44&#039;nin klinik geli\u015fiminde 18 mg\/kg dozuna ula\u015fmak \u00f6nemli bir ad\u0131md\u0131r.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u015eirket, daha y\u00fcksek dozun daha fazla ila\u00e7 maruziyetine, artan distrofin \u00fcretimine ve potansiyel fonksiyonel faydalara yol a\u00e7abilece\u011fini \u00f6ng\u00f6r\u00fcyor. Bununla birlikte, bunlar devam eden \u00e7al\u0131\u015fman\u0131n ara\u015ft\u0131rmay\u0131 ama\u00e7lad\u0131\u011f\u0131 olas\u0131l\u0131klard\u0131r, do\u011frulanm\u0131\u015f klinik sonu\u00e7lar de\u011fildir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Doz art\u0131r\u0131m\u0131, ara\u015ft\u0131rmac\u0131lar\u0131n tedavinin g\u00fcvenli\u011fini, farmakokinetik profilini ve farmakodinamik etkilerini daha iyi anlamalar\u0131na da yard\u0131mc\u0131 olacakt\u0131r.<\/p>\n\n\n\n<h2 id=\"when-will-more-data-be-available\" class=\"wp-block-heading\">Daha fazla veri ne zaman kullan\u0131ma sunulacak?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Entrada Therapeutics \u00e7al\u0131\u015fmas\u0131n\u0131n, a\u00e7\u0131k etiketli d\u00f6nemde bir y\u0131ll\u0131k tedavi sonras\u0131nda 1. Kohort kat\u0131l\u0131mc\u0131lar\u0131ndan elde edilen verileri 2026 y\u0131l\u0131n\u0131n sonlar\u0131nda a\u00e7\u0131klamas\u0131 bekleniyor. <strong>2. Kohorttan elde edilen verilerin 2027 y\u0131l\u0131n\u0131n ilk \u00e7eyre\u011finde a\u00e7\u0131klanmas\u0131 bekleniyor.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Aral\u0131k 2025&#039;te ABD G\u0131da ve \u0130la\u00e7 \u0130daresi (FDA), ENTR-601-44&#039;ye Nadir Pediatrik Hastal\u0131k Tan\u0131m\u0131 verdi.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">En y\u00fcksek doza ge\u00e7i\u015f, ENTR-601-44'nin geli\u015ftirilmesinde bir ba\u015fka ad\u0131m\u0131 i\u015faret ediyor. Gelecekteki klinik veriler, Duchenne kas distrofisi olan ve ekzon 44 atlamas\u0131 i\u00e7in uygun olan ki\u015filer i\u00e7in g\u00fcvenli\u011fini ve potansiyel faydalar\u0131n\u0131 anlamak a\u00e7\u0131s\u0131ndan \u00f6nemli 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 received clearance to test the highest dose in its Exon 44 Skipping Study for Duchenne muscular dystrophy (DMD). The independent Data Monitoring Committee (DMC) has recommended starting Cohort 3 at 18 mg\/kg in the ELEVATE-44-201 clinical trial, following its review of available safety and pharmacokinetic data from participants in Cohort 2. Entrada [&hellip;]<\/p>\n","protected":false},"author":7,"featured_media":8861,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[235,265,234,141,127,246],"class_list":["post-8859","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-elevate-44-201","tag-entr-601-44","tag-entrada-therapeutics","tag-exon-44","tag-exon-44-skipping","tag-exon-44-skipping-therapies"],"subtitle":"Entrada Therapeutics, Duchenne Ekzon 44 Atlama \u00c7al\u0131\u015fmas\u0131'nda ENTR-601-44'nin en y\u00fcksek dozunu test etmek i\u00e7in onay ald\u0131. ELEVATE-44-201 denemesi \u015fimdi 18 mg\/kg'a ilerleyecek. Bu d\u00f6n\u00fcm noktas\u0131, gelecekteki tedavi beklentileri i\u00e7in ne anlama geliyor?","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/8859","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\/7"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/comments?post=8859"}],"version-history":[{"count":5,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/8859\/revisions"}],"predecessor-version":[{"id":8871,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/8859\/revisions\/8871"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/8861"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=8859"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=8859"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=8859"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}