{"id":3307,"date":"2025-03-19T19:20:55","date_gmt":"2025-03-19T16:20:55","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=3307"},"modified":"2025-03-19T19:20:57","modified_gmt":"2025-03-19T16:20:57","slug":"regenxbio-reports-positive-biomarker-data-from-affinity-duchenne-trial-of-rgx-202-gene-therapy","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/regenxbio-reports-positive-biomarker-data-from-affinity-duchenne-trial-of-rgx-202-gene-therapy\/","title":{"rendered":"Regenxbio, Rgx-202 Gen Terapisinin Affinity Duchenne Denemesinden Pozitif Biyobelirte\u00e7 Verisi Bildirdi"},"content":{"rendered":"<p class=\"wp-block-paragraph\">REGENXBIO, Duchenne kas distrofisi (Duchenne) i\u00e7in farkl\u0131la\u015ft\u0131r\u0131lm\u0131\u015f bir ara\u015ft\u0131rma gen terapisi olan RGX-202&#039;nin AFFINITY DUCHENNE\u00ae denemesinin Faz I\/II b\u00f6l\u00fcm\u00fcndeki iki ek hastadan yeni, olumlu ara veriler bildirdi. Sonu\u00e7lar 2025 Kas Distrofisi Derne\u011fi (MDA) Klinik ve Bilimsel Konferans\u0131&#039;nda sunuldu.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Rgx-202&#039;den elde edilen temel bulgular \u015funlard\u0131r:<\/strong><\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>RGX-202&#039;nin pivot dozunu alan iki hastadan al\u0131nan yeni biyobelirte\u00e7 verileri MDA&#039;da sunuldu ve t\u00fcm ya\u015flarda RGX-202 mikrodistrofinin tutarl\u0131, g\u00fc\u00e7l\u00fc ekspresyonunu ve transd\u00fcksiyonunu desteklemeye devam ediyor.<\/li>\n<\/ul>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Dozlama s\u0131ras\u0131nda 3 ya\u015f\u0131nda olan bir hastada, mikrodistrofin ekspresyonunun kontrolle kar\u015f\u0131la\u015ft\u0131r\u0131ld\u0131\u011f\u0131nda 122.3% oldu\u011fu \u00f6l\u00e7\u00fcld\u00fc. 4 ya\u015f\u0131ndan k\u00fc\u00e7\u00fck hastalar\u0131n gen terapisine eri\u015fimi yoktur ve REGENXBIO, ABD&#039;de bu ya\u015f grubundaki hastalar\u0131 i\u015fe alan tek gen terapisi sponsorudur<\/li>\n<\/ul>\n\n\n\n<ul class=\"wp-block-list\">\n<li>7 ya\u015f\u0131nda bir hastada RGX-202 mikrodistrofin ekspresyonunun kontrol ile kar\u015f\u0131la\u015ft\u0131r\u0131ld\u0131\u011f\u0131nda 31.5% oldu\u011fu \u00f6l\u00e7\u00fcld\u00fc.<\/li>\n<\/ul>\n\n\n\n<ul class=\"wp-block-list\">\n<li>T\u00fcm hastalarda RGX-202&#039;nin sarkolemmaya uygun \u015fekilde lokalize oldu\u011fu g\u00f6r\u00fcld\u00fc ve bu durum CT-Domain ile farkl\u0131la\u015fan yap\u0131n\u0131n kas\u0131 uygun \u015fekilde hedef ald\u0131\u011f\u0131n\u0131 g\u00f6sterdi.<\/li>\n<\/ul>\n\n\n\n<ul class=\"wp-block-list\">\n<li>RGX-202 mikrodistrofin ekspresyonunun 8 ya\u015f ve \u00fczeri ayakta tedavi g\u00f6ren hastalardaki sonu\u00e7lar\u0131, onayl\u0131 veya ara\u015ft\u0131rma a\u015famas\u0131ndaki gen tedavileri aras\u0131nda bildirilen en y\u00fcksek mikrodistrofin d\u00fczeyleridir.<\/li>\n<\/ul>\n\n\n\n<ul class=\"wp-block-list\">\n<li>H\u0131zland\u0131r\u0131lm\u0131\u015f onay yolunu kullanarak bir Biyolojik Lisans Ba\u015fvurusunu (BLA) desteklemek i\u00e7in, AFFINITY DUCHENNE&#039;in pivotal faz\u0131ndaki birincil son nokta, 12. haftada RGX-202 mikrodistrofin ekspresyonu \u226510% olan kat\u0131l\u0131mc\u0131lar\u0131n oran\u0131d\u0131r.<\/li>\n<\/ul>\n\n\n\n<ul class=\"wp-block-list\">\n<li>RGX-202 ayr\u0131ca onayl\u0131 veya ara\u015ft\u0131rma a\u015famas\u0131ndaki gen terapileri aras\u0131nda qPCR ile \u00f6l\u00e7\u00fclen en y\u00fcksek bildirilen vekt\u00f6r genom kopyalar\u0131n\u0131 (4,9-55,4) g\u00f6stermeye devam ediyor.<\/li>\n<\/ul>","protected":false},"excerpt":{"rendered":"<p>REGENXBIO, Duchenne kas distrofisi (Duchenne) i\u00e7in farkl\u0131la\u015ft\u0131r\u0131lm\u0131\u015f bir ara\u015ft\u0131rma gen tedavisi olan RGX-202&#039;nin AFFINITY DUCHENNE\u00ae denemesinin Faz I\/II b\u00f6l\u00fcm\u00fcndeki iki ek hastadan yeni, olumlu ara veriler bildirdi. Sonu\u00e7lar, 2025 Kas Distrofisi Derne\u011fi (MDA) Klinik ve Bilimsel Konferans\u0131&#039;nda sunuldu. Rgx-202&#039;nin temel bulgular\u0131 \u015funlard\u0131r:<\/p>","protected":false},"author":1,"featured_media":3308,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[61,297,107,108],"class_list":["post-3307","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-gene-therapy","tag-muscular-dystrophy-association","tag-regenxbio","tag-rgx-202"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/3307","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=3307"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/3307\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/3308"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=3307"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=3307"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=3307"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}