{"id":4964,"date":"2025-10-30T15:41:35","date_gmt":"2025-10-30T12:41:35","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=4964"},"modified":"2025-11-03T19:56:20","modified_gmt":"2025-11-03T16:56:20","slug":"regenxbio-announces-completion-pivotal-enrollment-and-initiates","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/regenxbio-announces-completion-pivotal-enrollment-and-initiates\/","title":{"rendered":"REGENXBIO, RGX-202 Pivotal Kayd\u0131n\u0131n Tamamland\u0131\u011f\u0131n\u0131 ve Duchenne Gen Terapisi Program\u0131nda Ticari \u00dcretime Ba\u015flad\u0131\u011f\u0131n\u0131 Duyurdu"},"content":{"rendered":"<p class=\"wp-block-paragraph\">REGENXBIO, Duchenne kas distrofisi tedavisinde kullan\u0131lmak \u00fczere geli\u015ftirilen bir gen tedavisi olan RGX-202&#039;nin AFFINITY DUCHENNE \u00f6nc\u00fc denemesine kat\u0131l\u0131m\u0131n tamamland\u0131\u011f\u0131n\u0131 ve ticari olarak tedarik edilmek \u00fczere tasarlanan ilk partilerin ba\u015far\u0131yla \u00fcretildi\u011fini duyurdu. <strong>Devam\u0131n\u0131 oku<\/strong>: <a href=\"https:\/\/clinicaltrials.gov\/study\/NCT05693142\" target=\"_blank\" rel=\"noreferrer noopener\">NCT05693142<\/a>.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u201cDuchenne toplulu\u011funun, kal\u0131c\u0131, g\u00fcvenli sonu\u00e7lar sa\u011flayan ve bu dejeneratif hastal\u0131\u011f\u0131n seyrini anlaml\u0131 bir \u015fekilde de\u011fi\u015ftirebilecek yeni tedavi se\u00e7eneklerine acilen ihtiyac\u0131 var. <strong>REGENXBIO Ba\u015fkan\u0131 ve \u0130cra Kurulu Ba\u015fkan\u0131 Curran Simpson, &quot;Bu \u00f6nemli deneme kilometre ta\u015f\u0131n\u0131 tamamlamak ve ticari kullan\u0131ma y\u00f6nelik ilk dozlar\u0131m\u0131z\u0131 kendi b\u00fcnyemizde \u00fcretmek, s\u0131n\u0131rl\u0131 se\u00e7eneklere sahip Duchenne hastalar\u0131 i\u00e7in RGX-202&#039;yi s\u0131n\u0131f\u0131n\u0131n en iyisi olabilecek bir gen tedavisi olarak sunmam\u0131za daha da yakla\u015f\u0131yor&quot; dedi.<\/strong> \u201cRGX-202&#039;nin arkas\u0131ndaki farkl\u0131la\u015ft\u0131r\u0131lm\u0131\u015f tedavi yakla\u015f\u0131m\u0131, sekt\u00f6r lideri \u00fcr\u00fcn safl\u0131k seviyelerimiz ve C-Terminal alan\u0131na sahip yeni yap\u0131m\u0131z dahil olmak \u00fczere, Faz I\/II&#039;de g\u00f6r\u00fclen tutarl\u0131 fonksiyonel fayda ile birlikte pozitif g\u00fcvenlik ve etkililik profiliyle sonu\u00e7land\u0131. <strong>Bu son derece cesaret verici sonu\u00e7larla, ticari tedarikimizi geni\u015fletmeye ve gelecek y\u0131l\u0131n ikinci \u00e7eyre\u011finin ba\u015flar\u0131nda \u00f6nemli verileri payla\u015fmaya kararl\u0131y\u0131z.\u201d<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Daha fazla bilgi edin<\/strong>: <a href=\"https:\/\/dmdwarrior.com\/tr\/rgx-202-gene-therapy-phase-3-clinical-trial-nct05693142\/\">RGX-202 Gen Terapisi Faz 3 Klinik Denemesi Ba\u015fl\u0131yor<\/a><\/p>\n\n\n\n<h2 class=\"wp-block-heading\">RGX-202 Hakk\u0131nda Temel Sonu\u00e7lar<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>RGX-202 ile tedavi edilen hastalar, AFFINITY DUCHENNE \u00e7al\u0131\u015fmas\u0131n\u0131n Faz I\/II k\u0131sm\u0131nda do\u011fal seyirle kar\u015f\u0131la\u015ft\u0131r\u0131ld\u0131\u011f\u0131nda tutarl\u0131, g\u00fc\u00e7l\u00fc mikrodistrofin ekspresyonu ve i\u015flevsel iyile\u015fme g\u00f6stermektedir ve bu da h\u0131zland\u0131r\u0131lm\u0131\u015f onay yoluyla olas\u0131 onay\u0131 desteklemektedir.<\/li>\n\n\n\n<li>REGENXBIO, do\u011frulay\u0131c\u0131 denemeye hasta kaydetmeye devam ediyor<\/li>\n\n\n\n<li>Ticari tedarik i\u00e7in tasarlanan ilk partiler, \u015firket i\u00e7i \u00dcretim \u0130novasyon Merkezi&#039;nde \u00fcretildi\n<ul class=\"wp-block-list\">\n<li>Y\u0131lda 2.500&#039;e kadar RGX-202 dozu \u00fcretme kapasitesi<\/li>\n<\/ul>\n<\/li>\n\n\n\n<li>\u00d6nemli verilerin art\u0131k 2026&#039;n\u0131n 2. \u00e7eyre\u011finin ba\u015flar\u0131nda ve BLA&#039;n\u0131n 2026 ortas\u0131nda sunulmas\u0131 bekleniyor<\/li>\n<\/ul>\n\n\n\n<h2 class=\"wp-block-heading\">RGX-202 Etki Mekanizmas\u0131<\/h2>\n\n\n\n<figure class=\"wp-block-embed is-type-video is-provider-vimeo wp-block-embed-vimeo\"><div class=\"wp-block-embed__wrapper\">\n<iframe title=\"Son Hasta videosu 21 May\u0131s 2025 (1080p)\" src=\"https:\/\/player.vimeo.com\/video\/1092247292?dnt=1&amp;app_id=122963\" width=\"696\" height=\"392\" frameborder=\"0\" allow=\"autoplay; fullscreen; picture-in-picture; clipboard-write; encrypted-media; web-share\" referrerpolicy=\"strict-origin-when-cross-origin\"><\/iframe>\n<\/div><\/figure>\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>REGENXBIO announced the completion of enrollment in the AFFINITY DUCHENNE pivotal trial of RGX-202, an investigational gene therapy for the treatment of Duchenne muscular dystrophy, as well as the successful production of the first batches intended for commercial supply. Read More: NCT05693142. &#8220;The Duchenne community urgently needs new treatment options that provide durable, safe outcomes [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":4965,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[61,107,108],"class_list":["post-4964","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-gene-therapy","tag-regenxbio","tag-rgx-202"],"subtitle":"REGENXBIO, Duchenne kas distrofisi i\u00e7in ara\u015ft\u0131rma a\u015famas\u0131ndaki bir gen tedavisi olan RGX-202 i\u00e7in AFFINITY DUCHENNE denemesinin \u00f6nemli bir b\u00f6l\u00fcm\u00fcnde 30 kat\u0131l\u0131mc\u0131n\u0131n kayd\u0131n\u0131 tamamlad\u0131\u011f\u0131n\u0131 duyurdu.","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/4964","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=4964"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/4964\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/4965"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=4964"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=4964"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=4964"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}