{"id":6742,"date":"2026-03-11T21:55:54","date_gmt":"2026-03-11T18:55:54","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=6742"},"modified":"2026-03-11T21:59:30","modified_gmt":"2026-03-11T18:59:30","slug":"regenxbio-phase-1-2-affinity-duchenne-trial-of-rgx-202","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/regenxbio-phase-1-2-affinity-duchenne-trial-of-rgx-202\/","title":{"rendered":"Regenxbio, Rgx-202&#039;nin Faz 1\/2 Affinity Duchenne \u00c7al\u0131\u015fmas\u0131ndan Yeni Olumlu Ara Veriler Bildirdi"},"content":{"rendered":"<p class=\"wp-block-paragraph\"><strong>REGENXBIO, Duchenne kas distrofisi i\u00e7in potansiyel olarak s\u0131n\u0131f\u0131n\u0131n en iyisi gen terapisi olan RGX-202&#039;nin Faz I\/II AFFINITY DUCHENNE \u00e7al\u0131\u015fmas\u0131ndan elde edilen yeni olumlu ara verileri a\u00e7\u0131klad\u0131.<\/strong> Ara\u015ft\u0131rmay\u0131 y\u00fcr\u00fcten Stanford T\u0131p Fak\u00fcltesi N\u00f6roloji Klinik Profes\u00f6r\u00fc Dr. Carolina Tesi-Rocha, yeni fonksiyonel, g\u00fcvenlik, biyobelirte\u00e7 ve kardiyak MRI \u00f6l\u00e7\u00fcmlerini i\u00e7eren bu verileri Kas Distrofisi Derne\u011fi (MDA) Klinik ve Bilimsel Konferans\u0131&#039;nda sunuyor. Daha fazla bilgi edinin:\u00a0<a href=\"https:\/\/dmdwarrior.com\/tr\/biomarkers-in-duchenne-muscular-dystrophy\/\" target=\"_blank\" rel=\"noreferrer noopener\">Biyobelirte\u00e7ler<\/a><\/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=\"#affinity-duchenne-phase-i-ii-interim-data-updates-data-cut-january-5-2026\">AFFINITY DUCHENNE Faz I\/II Ara Veri G\u00fcncellemeleri (veri kesim tarihi: 5 Ocak 2026)<\/a><ul><li><a href=\"#functional-data\">Fonksiyonel Veriler<\/a><\/li><li><a href=\"#cardiac-function\">Kalp Fonksiyonu<\/a><\/li><li><a href=\"#biomarker-data\">Biyobelirte\u00e7 Verileri<\/a><\/li><li><a href=\"#safety-and-tolerability-data\">G\u00fcvenlik ve Tolere Edilebilirlik Verileri<\/a><\/li><li><a href=\"#affinity-duchenne-trial\">AFFINITY DUCHENNE Denemesi<\/a><\/li><\/ul><\/li><\/ul><\/nav><\/div>\n\n\n\n<p class=\"wp-block-paragraph\">\u201cREGENXBIO Ba\u015f T\u0131bbi Sorumlusu Dr. Steve Pakola, \u201dBug\u00fcnk\u00fc yeni Faz I\/II ara verileri, fonksiyon \u00fczerinde devam eden olumlu etkiyi, istikrarl\u0131 kalp sa\u011fl\u0131\u011f\u0131n\u0131 ve olumlu bir g\u00fcvenlik profilini g\u00f6stererek, RGX-202&#039;nin Duchenne i\u00e7in farkl\u0131la\u015ft\u0131r\u0131lm\u0131\u015f bir gen terapisi se\u00e7ene\u011fi olma potansiyelini vurgulamaktad\u0131r\u201c dedi. \u201dG\u00fcvenli\u011fe y\u00f6nelik proaktif, kapsaml\u0131 yakla\u015f\u0131m\u0131m\u0131z ve yeni mikrodistrofin yap\u0131m\u0131z, tedavi sonras\u0131 iki y\u0131la kadar s\u00fcrd\u00fcr\u00fclen g\u00fcvenlik ve kal\u0131c\u0131 fonksiyonel sonu\u00e7larla desteklenmektedir. 2. \u00e7eyre\u011fin ba\u015flar\u0131nda en \u00f6nemli verilerimizin a\u00e7\u0131klanmas\u0131na yakla\u015f\u0131rken, bu kapsaml\u0131 yeni veri seti ve RGX-202&#039;yi hastalar i\u00e7in potansiyel olarak anlaml\u0131 bir tedavi se\u00e7ene\u011fi olarak ilerletme f\u0131rsat\u0131 bizi \u00e7ok cesaretlendiriyor.\u201d<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u201cDr. Tesi-Rocha, \u201dDuchenne, \u00e7ocuklar\u0131 zamanla kas g\u00fcc\u00fcnden ve ba\u011f\u0131ms\u0131zl\u0131klar\u0131ndan mahrum b\u0131rakan y\u0131k\u0131c\u0131, dejeneratif bir hastal\u0131kt\u0131r ve RGX-202&#039;nin olumlu g\u00fcvenlik ve cesaret verici etkinlik profilinin devam etti\u011fini g\u00f6rmekten memnuniyet duyuyorum,\u201c dedi. \u2019Biyomarker sonu\u00e7lar\u0131, fonksiyonel iyile\u015fme, kardiyak stabilite ve karaci\u011fer g\u00fcvenli\u011fi de dahil olmak \u00fczere bu olumlu Faz I\/II verileri, RGX-202&#039;nin Duchenne&#039;de sa\u011fl\u0131\u011f\u0131n temel \u00f6l\u00e7\u00fctleri \u00fczerindeki potansiyel etkisine dair daha net bir tablo sunmaktad\u0131r.\u201d<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"affinity-duchenne-phase-i-ii-interim-data-updates-data-cut-january-5-2026\">AFFINITY DUCHENNE Faz I\/II Ara Veri G\u00fcncellemeleri (veri kesim tarihi: 5 Ocak 2026)<\/h2>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"functional-data\">Fonksiyonel Veriler<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">Yakla\u015f\u0131k 6 ila 12 ya\u015flar\u0131 aras\u0131ndaki ve temel doz seviyesinde (2\u00d71014 GC\/kg) tedavi edilen yedi kat\u0131l\u0131mc\u0131dan elde edilen ara fonksiyonel sonu\u00e7larda, RGX-202&#039;nin bir y\u0131l sonra Kuzey Y\u0131ld\u0131z\u0131 Ambulatuvar De\u011ferlendirmesi (NSAA) ve t\u00fcm zamanl\u0131 fonksiyon testlerinde (Ayakta Durma S\u00fcresi, 10 Metre Y\u00fcr\u00fcme-Ko\u015fma, T\u0131rmanma S\u00fcresi) hastal\u0131k seyrini olumlu y\u00f6nde etkiledi\u011fine dair kan\u0131tlar g\u00f6stermeye devam etti\u011fi g\u00f6r\u00fclm\u00fc\u015ft\u00fcr.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Fonksiyonel sonu\u00e7lar, tedavi olmaks\u0131z\u0131n beklenen hastal\u0131k ilerlemesini tahmin etmek i\u00e7in cTAP hastal\u0131k ilerleme modeli ve kaba e\u015fle\u015ftirme ve e\u011filim puan\u0131 a\u011f\u0131rl\u0131kland\u0131rmas\u0131 kullan\u0131larak yap\u0131lan d\u0131\u015f kontrol kar\u015f\u0131la\u015ft\u0131rmalar\u0131 da dahil olmak \u00fczere birden fazla do\u011frulanm\u0131\u015f y\u00f6ntem kullan\u0131larak analiz edildi. E\u011filim puan\u0131 a\u011f\u0131rl\u0131kland\u0131rmas\u0131, temel \u00e7al\u0131\u015fma i\u00e7in SAP&#039;de belirtilen birincil analiz y\u00f6ntemidir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Temel doz alan kat\u0131l\u0131mc\u0131lar, e\u011filim puan\u0131 a\u011f\u0131rl\u0131kland\u0131rmas\u0131 kullan\u0131larak yap\u0131lan kar\u015f\u0131la\u015ft\u0131rmada, t\u00fcm zamanl\u0131 fonksiyon testlerinde ve NSAA&#039;da d\u0131\u015f kontrol grubuna g\u00f6re daha iyi performans sergilediler. 95% g\u00fcven aral\u0131\u011f\u0131, RGX-202 lehine olumlu sonu\u00e7lar g\u00f6stermektedir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">NSAA \u00e7al\u0131\u015fmas\u0131nda, kritik doz alan kat\u0131l\u0131mc\u0131lar beklenen hastal\u0131k seyrini ve d\u0131\u015f kontrol gruplar\u0131n\u0131 a\u015ft\u0131. \u00d6zellikle, yedi kat\u0131l\u0131mc\u0131dan be\u015fi dozlama s\u0131ras\u0131nda 8 ya\u015f ve \u00fczerindeydi; bu ya\u015fta fonksiyonel gerileme bekleniyordu. Bir y\u0131l sonra, kat\u0131l\u0131mc\u0131lar (n=7) cTAP&#039;a k\u0131yasla ortalama +4,9 puanl\u0131k bir iyile\u015fme g\u00f6sterdi. Daha ya\u015fl\u0131 kat\u0131l\u0131mc\u0131lar (n=5) ise cTAP&#039;a k\u0131yasla ortalama +5,2 puanl\u0131k bir iyile\u015fme g\u00f6sterdi.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Ek olarak, doz seviyesi 1&#039;deki (1\u00d71014 GC\/kg) kat\u0131l\u0131mc\u0131lar (n=3), beklenen hastal\u0131k seyrini a\u015farak iki y\u0131l i\u00e7inde cTAP&#039;ye k\u0131yasla ortalama +5,6 puanl\u0131k bir iyile\u015fme g\u00f6sterdi.<\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"cardiac-function\">Kalp Fonksiyonu<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">Temel doz uygulanan kat\u0131l\u0131mc\u0131lar, ortalama sol ventrik\u00fcl ejeksiyon fraksiyonu, global \u00e7evresel gerilim ve ge\u00e7 gadolinyum kontrast\u0131 ile de\u011ferlendirilen fibrozis dahil olmak \u00fczere MRI u\u00e7 noktalar\u0131yla \u00f6l\u00e7\u00fcld\u00fc\u011f\u00fc \u00fczere, tedavi sonras\u0131 bir y\u0131l i\u00e7inde kardiyak stabilite sergilediler.<\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"biomarker-data\">Biyobelirte\u00e7 Verileri<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">Faz I\/II \u00e7al\u0131\u015fmas\u0131ndan elde edilen biyobelirte\u00e7 verileri, RGX-202 mikrodistrofinin tutarl\u0131, y\u00fcksek ekspresyonunu ve transd\u00fcksiyonunu desteklemeye devam etmektedir (n=13). Dozlama s\u0131ras\u0131nda 3,6 ya\u015f\u0131nda olan ek bir hastadan elde edilen yeni veriler, 12. haftada 51,2% mikrodistrofin ekspresyon seviyesine sahip oldu\u011funu g\u00f6stermi\u015ftir. AFFINITY DUCHENNE&#039;nin kilit faz\u0131ndaki birincil sonlan\u0131m noktas\u0131, 12. haftada RGX-202 mikrodistrofin ekspresyonu &gt;10% olan kat\u0131l\u0131mc\u0131lar\u0131n oran\u0131d\u0131r.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">RGX-202, sarkolemmaya uygun \u015fekilde lokalize edildi ve bu da C-Terminal (CT) alan\u0131n\u0131n dahil edilmesiyle farkl\u0131la\u015ft\u0131r\u0131lm\u0131\u015f yap\u0131n\u0131n kas\u0131 uygun \u015fekilde hedefledi\u011fini g\u00f6sterdi.<\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"safety-and-tolerability-data\">G\u00fcvenlik ve Tolere Edilebilirlik Verileri<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">Faz I\/II&#039;deki t\u00fcm temel doz kat\u0131l\u0131mc\u0131lar\u0131ndan (dozlama s\u0131ras\u0131nda 1 ila &lt;12 ya\u015f aras\u0131) elde edilen yeni ara g\u00fcvenlik verileri, birden fazla \u00f6l\u00e7\u00fcmde karaci\u011fer hasar\u0131na dair hi\u00e7bir kan\u0131t g\u00f6stermemektedir. Duchenne&#039;de karaci\u011fer iltihab\u0131n\u0131n bilinen belirte\u00e7leri olan ortalama gama-glutamil transferaz (GGT) ve toplam bilirubin, tedaviden sonraki iki y\u0131la kadar normalin \u00fcst s\u0131n\u0131r\u0131n\u0131 a\u015fmam\u0131\u015ft\u0131r. Tedaviden bir y\u0131l sonra kreatin kinazda ortalama bir azalma g\u00f6zlemlenmi\u015ftir (n=8) ve bu azalma, ALT (n=7), AST (n=7) ve LDH&#039;da (n=8) ortalama azalmalarla desteklenmi\u015ftir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">RGX-202, Faz I\/II \u00e7al\u0131\u015fmas\u0131nda veri kesim tarihi itibariyle (n=13) ciddi yan etki (SAE) veya \u00f6zel ilgi alan\u0131 olu\u015fturan yan etki (AESI) olmaks\u0131z\u0131n iyi tolere edilmi\u015ftir. Yayg\u0131n ila\u00e7la ili\u015fkili yan etkiler aras\u0131nda kusma, yorgunluk ve bulant\u0131 yer almaktad\u0131r. Bunlar\u0131n t\u00fcm\u00fc, gen terapisi uygulamas\u0131yla tipik olarak beklenen yan etkilerdir. Farkl\u0131la\u015ft\u0131r\u0131lm\u0131\u015f bir yap\u0131 ve 80% tam kapsidlerin \u00fczerinde end\u00fcstri lideri \u00fcr\u00fcn safl\u0131k seviyeleriyle birle\u015ftirilmi\u015f proaktif, k\u0131sa s\u00fcreli bir imm\u00fcn mod\u00fclasyon rejimi, RGX-202 i\u00e7in olumlu bir g\u00fcvenlik profiline katk\u0131da bulunmu\u015f olabilir.<\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"affinity-duchenne-trial\">AFFINITY DUCHENNE Denemesi<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">REGENXBIO, 2026 y\u0131l\u0131n\u0131n ikinci \u00e7eyre\u011finin ba\u015flar\u0131nda \u00f6nemli verileri payla\u015fmay\u0131 ve 2026 y\u0131l\u0131n\u0131n ortalar\u0131nda FDA ile BLA \u00f6ncesi bir toplant\u0131 talep etmeyi bekliyor. \u015eirket, RGX-202&#039;nin do\u011frulay\u0131c\u0131 denemesine 1 ya\u015f ve \u00fczeri yakla\u015f\u0131k 30 kat\u0131l\u0131mc\u0131 kaydetmeye devam ediyor ve BLA ba\u015fvurusu s\u0131ras\u0131nda bu denemenin b\u00fcy\u00fck \u00e7o\u011funlu\u011funun tamamlanm\u0131\u015f olmas\u0131n\u0131 bekliyor.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Bu Sayfay\u0131 Takip Edin<\/strong>&nbsp;&gt;&gt;&gt;&nbsp;<a href=\"https:\/\/dmdwarrior.com\/tr\/dmd-therapies\/\" target=\"_blank\" rel=\"noreferrer noopener\">Duchenne i\u00e7in T\u00fcm Klinik Ara\u015ft\u0131rmalar<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>REGENXBIO announced new positive interim data from the Phase I\/II AFFINITY DUCHENNE trial of RGX-202, a potential best-in-class gene therapy for Duchenne muscular dystrophy. Trial investigator Carolina Tesi-Rocha, M.D., Clinical Professor, Neurology, Stanford School of Medicine, Stanford Children&#8217;s Health, is presenting this data, including new functional, safety, biomarker, and cardiac MRI measures, at the Muscular [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":6744,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[525,61,107,108],"class_list":["post-6742","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-affinity-duchenne","tag-gene-therapy","tag-regenxbio","tag-rgx-202"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/6742","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=6742"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/6742\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/6744"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=6742"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=6742"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=6742"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}