{"id":4132,"date":"2025-07-16T18:57:40","date_gmt":"2025-07-16T15:57:40","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=4132"},"modified":"2025-07-16T18:57:41","modified_gmt":"2025-07-16T15:57:41","slug":"precision-biosciences-highlights-new-preclinical-data-pbgene-dmd","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/precision-biosciences-highlights-new-preclinical-data-pbgene-dmd\/","title":{"rendered":"Precision BioSciences, Duchenne Musk\u00fcler Distrofi Tedavisi i\u00e7in PBGENE-DMD&#039;ye \u0130li\u015fkin Yeni Klinik \u00d6ncesi Verileri \u00d6ne \u00c7\u0131kar\u0131yor"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Y\u00fcksek kar\u015f\u0131lanmam\u0131\u015f gereksinime sahip hastal\u0131klar i\u00e7in in vivo gen d\u00fczenleme terapileri geli\u015ftirmek amac\u0131yla yenilik\u00e7i tescilli ARCUS\u00ae platformunu kullanan klinik a\u015famada gen d\u00fczenleme \u015firketi Precision BioSciences, bug\u00fcn Duchenne kas distrofisi (DMD) i\u00e7in \u015eirketin s\u0131n\u0131f\u0131n\u0131n ilk in vivo gen d\u00fczenleme yakla\u015f\u0131m\u0131 olan PBGENE-DMD&#039;nin insan \u00fczerinde ilk klinik ara\u015ft\u0131rmaya do\u011fru h\u0131zla geli\u015ftirilmesini destekleyen bilimsel gerek\u00e7eyi daha da do\u011frulayan g\u00fcncellenmi\u015f ek klinik \u00f6ncesi verileri duyurdu.<\/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=\"#new-preclinical-data-for-pbgene-dmd\">PBGENE-DMD i\u00e7in Yeni Klinik \u00d6ncesi Veriler<\/a><\/li><li><a href=\"#pbgene-dmd-is-the-first-in-vivo-gene-editing-program\">PBGENE-DMD, ilk in vivo gen d\u00fczenleme program\u0131d\u0131r<\/a><\/li><li><a href=\"#pbgene-dmd-between-exons-45-55\">PBGENE-DMD: 45-55 Eksonlar Aras\u0131<\/a><\/li><li><a href=\"#what-is-pbgene-dmd\">PBGENE-DMD nedir?<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"new-preclinical-data-for-pbgene-dmd\">PBGENE-DMD i\u00e7in Yeni Klinik \u00d6ncesi Veriler<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>PBGENE-DMD nihai klinik aday\u0131 i\u00e7in yeni klinik \u00f6ncesi veriler, potansiyel olarak kas uydu h\u00fccrelerinin d\u00fczenlenmesiyle y\u00f6nlendirilen, \u00f6nemli kas tiplerinde distrofin pozitif kas h\u00fccrelerinde bir art\u0131\u015f oldu\u011funu g\u00f6stermektedir.<\/li>\n<\/ul>\n\n\n\n<ul class=\"wp-block-list\">\n<li>PBGENE-DMD, \u00f6zellikle 45-55. eksonlar aras\u0131ndaki &#039;s\u0131cak nokta&#039; b\u00f6lgesindeki distrofin mutasyonlar\u0131ndan etkilenen Duchenne Kas Distrofisi hastalar\u0131n\u0131n 60%&#039;sine kadar olan hastalar i\u00e7in s\u0131n\u0131f\u0131nda ilk olan bir in vivo gen d\u00fczenleme yakla\u015f\u0131m\u0131d\u0131r.<\/li>\n<\/ul>\n\n\n\n<ul class=\"wp-block-list\">\n<li>2025 y\u0131l\u0131nda PBGENE-DMD i\u00e7in Ara\u015ft\u0131rma Ama\u00e7l\u0131 Yeni \u0130la\u00e7 (IND) ve\/veya Klinik Ara\u015ft\u0131rma Ba\u015fvurusu (CTA) sunulmas\u0131 ve 2026 y\u0131l\u0131nda klinik verilerin beklenmesi i\u00e7in hassas hedefleme \u2013<\/li>\n<\/ul>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"pbgene-dmd-is-the-first-in-vivo-gene-editing-program\">PBGENE-DMD, ilk in vivo gen d\u00fczenleme program\u0131d\u0131r<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><br>\u201cDMD hastal\u0131\u011f\u0131 olan bir fare modelinde yapt\u0131\u011f\u0131m\u0131z uzun vadeli dayan\u0131kl\u0131l\u0131k \u00e7al\u0131\u015fmas\u0131nda, \u00fc\u00e7 ila dokuz ay aras\u0131nda quadriceps, gastroknemius (bald\u0131r), kalp ve diyaframda distrofin pozitif kas h\u00fccrelerinde \u00fc\u00e7 kata kadar art\u0131\u015f g\u00f6zlemledik. <strong>Precision BioSciences&#039;de Ba\u015f Bilim Sorumlusu Dr. Cassie Gorsuch, &quot;Gastroknemiusta 85% h\u00fccresine kadar distrofin pozitifti&quot; dedi.<\/strong> &quot;Bu yeni veriler, May\u0131s 2025&#039;te ASGCT&#039;de payla\u015f\u0131lan klinik \u00f6ncesi verilere dayan\u0131yor ve PBGENE-DMD tedavisinin ayn\u0131 \u00fc\u00e7 ve dokuz ayl\u0131k zaman noktalar\u0131nda maksimum kuvvet \u00e7\u0131kt\u0131s\u0131nda \u00f6nemli ve kal\u0131c\u0131 bir iyile\u015fme sa\u011flad\u0131\u011f\u0131n\u0131 g\u00f6steriyor. Distrofin pozitif h\u00fccrelerdeki bu geni\u015f art\u0131\u015f, dokularda tespit edilen distrofin proteinindeki art\u0131\u015fla birlikte, zaman i\u00e7inde g\u00f6zlemlenen ve bu \u00e7al\u0131\u015fmada da g\u00f6zlemlenen d\u00fczenlenmi\u015f uydu h\u00fccrelerine atfedilebilecek geli\u015fmi\u015f kas fonksiyonunu daha da do\u011fruluyor. Bu sonu\u00e7lar\u0131n, mikrodistrofin yakla\u015f\u0131mlar\u0131 i\u00e7in gerekli olan AAV kal\u0131c\u0131l\u0131\u011f\u0131na ihtiya\u00e7 duymadan kal\u0131c\u0131 bir i\u015flevsel fayda sa\u011flama konusunda PBGENE-DMD gen d\u00fczenleme yakla\u015f\u0131m\u0131n\u0131n benzersiz potansiyelini g\u00f6sterdi\u011fine inan\u0131yoruz. Gelecekteki bir bilimsel konferansta eksiksiz veri setini sunmay\u0131 d\u00f6rt g\u00f6zle bekliyoruz. Klinik \u00f6ncesi kan\u0131tlar\u0131n tamam\u0131 g\u00f6z \u00f6n\u00fcne al\u0131nd\u0131\u011f\u0131nda, bu program\u0131 klinik d\u00fczeye ta\u015f\u0131ma konusunda heyecanl\u0131 ve kararl\u0131 olmaya devam ediyoruz.&quot;<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"pbgene-dmd-between-exons-45-55\">PBGENE-DMD: 45-55 Eksonlar Aras\u0131<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u015eu anda, DMD&#039;nin uzun vadeli prognozunu olumlu y\u00f6nde de\u011fi\u015ftirecek \u015fekilde kas fonksiyonunu zamanla \u00f6nemli \u00f6l\u00e7\u00fcde iyile\u015ftiren onayl\u0131 bir tedavi veya geli\u015ftirilme a\u015famas\u0131nda olan bir tedavi bulunmamaktad\u0131r. <strong>PBGENE-DMD, tedavi paradigmas\u0131n\u0131 d\u00f6n\u00fc\u015ft\u00fcrme ve \u00e7o\u011fu hasta i\u00e7in kal\u0131c\u0131 i\u015flevsel iyile\u015fme sa\u011flama potansiyeline sahip ilk in vivo gen d\u00fczenleme program\u0131d\u0131r; etkilenenlerin 60%&#039;sine kadar\u0131, 45-55. eksonlar aras\u0131ndaki &#039;s\u0131cak nokta&#039; b\u00f6lgesinde mutasyon ta\u015f\u0131maktad\u0131r.<\/strong> Precision&#039;\u0131n yakla\u015f\u0131m\u0131, hastan\u0131n kendi DNA dizisini kal\u0131c\u0131 olarak d\u00fczenleyerek, insanlarda i\u015flevsel oldu\u011fu bilinen, do\u011fal olarak \u00fcretilen, neredeyse tam uzunlukta distrofin proteini elde etmek \u00fczere tasarlanm\u0131\u015ft\u0131r. IND&#039;yi destekleyen son toksikoloji \u00e7al\u0131\u015fmalar\u0131 \u015fu anda devam etmekte olup, IND ve\/veya CTA ba\u015fvurular\u0131n\u0131n 2025&#039;te, ilk klinik verilerin ise 2026&#039;da yap\u0131lmas\u0131 hedeflenmektedir. \u015eirket, mevcut nakit ak\u0131\u015f\u0131n\u0131n hem mevcut Faz 1 varl\u0131\u011f\u0131 olan PBGENE-HBV&#039;yi hem de PBGENE-DMD&#039;yi Faz 1 klinik sonu\u00e7lar\u0131na ta\u015f\u0131mak i\u00e7in yeterli olaca\u011f\u0131na inanmaktad\u0131r.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Devam\u0131n\u0131 oku<\/strong>: <a href=\"https:\/\/dmdwarrior.com\/tr\/precision-biosciences-pbgene-dmd-restores-production-of-nearly-full-length-dystrophin-protein\/\">Precision BioSciences PBGENE-DMD, Neredeyse Tam Uzunlukta Distrofin Proteininin \u00dcretimini Geri Kazand\u0131r\u0131r<\/a><\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"what-is-pbgene-dmd\">PBGENE-DMD nedir?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">PBGENE-DMD, Precision&#039;\u0131n DMD tedavisine y\u00f6nelik geli\u015ftirme program\u0131d\u0131r. <strong>Bu yakla\u015f\u0131m, fonksiyonel sonu\u00e7lar\u0131 iyile\u015ftirmek amac\u0131yla v\u00fccuttaki distrofin proteininin neredeyse tam uzunlu\u011funu geri kazand\u0131rmak amac\u0131yla distrofin geninin 45-55. ekzonlar\u0131n\u0131 \u00e7\u0131karmak i\u00e7in tek bir AAV&#039;de tek seferlik uygulama yoluyla verilen iki tamamlay\u0131c\u0131 ARCUS n\u00fckleaz\u0131n\u0131 kullan\u0131r.<\/strong> PBGENE-DMD, DMD hasta pop\u00fclasyonunun 60%&#039;sinden fazlas\u0131n\u0131 hedeflemektedir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Klinik \u00f6ncesi \u00e7al\u0131\u015fmalarda, PBGENE-DMD, DMD&#039;nin ilerlemesinde rol oynayan temel kas tiplerini hedefleme yetene\u011fini g\u00f6sterdi ve insanla\u015ft\u0131r\u0131lm\u0131\u015f bir DMD fare modelinde \u00f6nemli, kal\u0131c\u0131 i\u015flevsel iyile\u015ftirmeler \u00fcretti. PBGENE-DMD, v\u00fccudun kardiyak doku ve \u00e7e\u015fitli temel iskelet kas gruplar\u0131 dahil olmak \u00fczere birden fazla kasta neredeyse tam uzunlukta i\u015flevsel bir distrofin proteini \u00fcretme yetene\u011fini geri kazand\u0131rd\u0131. Ek olarak, PBGENE-DMD, uzun vadeli dayan\u0131kl\u0131l\u0131k ve s\u00fcrekli i\u015flevsel iyile\u015ftirme i\u00e7in kritik oldu\u011funa inan\u0131lan uydu kas k\u00f6k h\u00fccrelerini d\u00fczenledi.<\/p>","protected":false},"excerpt":{"rendered":"<p>Precision BioSciences a clinical stage gene editing company utilizing its novel proprietary ARCUS\u00ae platform to develop in vivo gene editing therapies for diseases with high unmet need, today announced updated additional preclinical data further validating the scientific rationale supporting the rapid development of PBGENE-DMD, the Company\u2019s first-in-class in vivo gene editing approach for Duchenne muscular [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":4134,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[359,72,314,313],"class_list":["post-4132","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-full-length-dystrophin","tag-gene-editing","tag-pbgene-dmd","tag-precision-biosciences"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/4132","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=4132"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/4132\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/4134"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=4132"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=4132"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=4132"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}