{"id":3883,"date":"2025-05-29T11:06:14","date_gmt":"2025-05-29T08:06:14","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=3883"},"modified":"2025-11-03T20:23:45","modified_gmt":"2025-11-03T17:23:45","slug":"pepgen-announces-it-will-halt-clinical-trials-of-exon-51-skipping-study","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/pepgen-announces-it-will-halt-clinical-trials-of-exon-51-skipping-study\/","title":{"rendered":"PepGen, Ekzon 51 Atlama \u00c7al\u0131\u015fmas\u0131n\u0131n Klinik Denemelerini Durduraca\u011f\u0131n\u0131 Duyurdu"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Oligon\u00fckleotid tedavilerinin yeni neslini ilerleten klinik a\u015famadaki bir biyoteknoloji \u015firketi olan PepGen, bug\u00fcn Duchenne kas distrofisi (DMD) hastalar\u0131nda PGN-EDO51&#039;yi ara\u015ft\u0131ran CONNECT1-EDO51 \u00e7al\u0131\u015fmas\u0131n\u0131n 10 mg\/kg kohortunda \u00f6l\u00e7\u00fclen distrofin protein seviyelerine dayanarak, \u015firketin \u015fu anda Faz 2 klinik geli\u015ftirme a\u015famas\u0131nda olan umut verici miyotonik distrofi tip 1 (DM1) program\u0131n\u0131 ilerletmeye odaklanaca\u011f\u0131n\u0131 duyurdu. \u015eirket, PGN-EDO51&#039;nin geli\u015ftirilmesini g\u00f6n\u00fcll\u00fc olarak durduruyor ve DMD ile ilgili t\u00fcm ara\u015ft\u0131rma ve geli\u015ftirme faaliyetlerini sonland\u0131rmay\u0131 planl\u0131yor.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">CONNECT1 \u00e7al\u0131\u015fmas\u0131n\u0131n 10 mg\/kg kohortunda (n=4), PGN-EDO51 ekzon 51 atlanm\u0131\u015f transkriptleri 4.26%&#039;ye (ortalama 3.5% art\u0131\u015f) y\u00fckseltti; ancak, toplam distrofin sadece normal seviyelerin 0.59%&#039;sine y\u00fckseldi (ortalama 0.36% art\u0131\u015f). PGN-EDO51&#039;nin g\u00fcvenlik profili genel olarak olumlu olmaya devam etti ve tedaviyle ili\u015fkili t\u00fcm yan etkiler hafif nitelikteydi. \u00c7al\u0131\u015fmada ciddi yan etki bildirilmedi.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Devam\u0131n\u0131 oku:<\/strong> <a href=\"https:\/\/dmdwarrior.com\/tr\/next-generation-exon-skipping-therapies-for-duchenne-muscular-dystrophy\/\">Duchenne Musk\u00fcler Distrofi Tedavisi \u0130\u00e7in Geli\u015ftirilen Yeni Nesil Ekzon Atlama Terapileri<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u201cCONNECT1&#039;de 10 mg\/kg doz kohortunda g\u00f6zlemlenen distrofin sonu\u00e7lar\u0131ndan hayal k\u0131r\u0131kl\u0131\u011f\u0131na u\u011frad\u0131k, \u00e7\u00fcnk\u00fc hastalar i\u00e7in mevcut tedavileri daha derin bir \u015fekilde iyile\u015ftirebilece\u011fimizi umuyorduk,\u201d dedi PepGen&#039;nin Ba\u015fkan\u0131 ve CEO&#039;su Dr. James McArthur. \u201cDMD program\u0131m\u0131z\u0131 sonland\u0131r\u0131rken, hastalara, ailelere, bak\u0131c\u0131lara, ara\u015ft\u0131rmac\u0131lara ve \u00e7al\u0131\u015fma personeline bu ara\u015ft\u0131rmaya verdikleri destek ve kat\u0131l\u0131mlar\u0131 i\u00e7in te\u015fekk\u00fcr etmek istiyoruz. Ayr\u0131ca ekibimizin DMD hastalar\u0131 i\u00e7in yeni potansiyel tedavileri ilerletme konusundaki s\u0131k\u0131 \u00e7al\u0131\u015fmas\u0131n\u0131 ve ba\u011fl\u0131l\u0131\u011f\u0131n\u0131 da takdir etmek istiyorum.\u201d<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Devam\u0131n\u0131 oku<\/strong>:\u00a0<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>PepGen, a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies, today announced that based on the levels of dystrophin protein measured in the 10 mg\/kg cohort of its CONNECT1-EDO51 study investigating PGN-EDO51 in Duchenne muscular dystrophy (DMD) patients amenable to exon 51 skipping, the Company will focus on advancing its promising myotonic dystrophy [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":3889,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[242,275,122,193,194,371,273,274],"class_list":["post-3883","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-clinical-trials","tag-connect1-edo51","tag-exon-51","tag-exon-51-skipping","tag-exon-51-treatment","tag-freedom-dm1","tag-pepgen","tag-pgn-edo51"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/3883","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=3883"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/3883\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/3889"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=3883"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=3883"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=3883"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}