{"id":8843,"date":"2026-10-04T10:08:04","date_gmt":"2026-10-04T07:08:04","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=8843"},"modified":"2026-10-04T10:08:07","modified_gmt":"2026-10-04T07:08:07","slug":"rag-18-duchenne-first-in-human-data-wms-2026","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/rag-18-duchenne-first-in-human-data-wms-2026\/","title":{"rendered":"Ractigen Therapeutics, Duchenne'de RAG-18 \u0130\u00e7in Olumlu \u0130lk \u0130nsan Verilerini A\u00e7\u0131klad\u0131"},"content":{"rendered":"<p class=\"wp-block-paragraph\"><strong>Ractigen Therapeutics, utrofin \u00fcretimini art\u0131rmak i\u00e7in k\u00fc\u00e7\u00fck aktive edici RNA (saRNA) kullanan, Duchenne kas distrofisi (Duchenne) i\u00e7in deneysel bir tedavi olan RAG-18 (NCT07282652) i\u00e7in insanlarda ilk olumlu verileri sunmu\u015ftur.<\/strong> Faz I \u00e7al\u0131\u015fmas\u0131n\u0131n verileri, Japonya&#039;n\u0131n Hiro\u015fima kentinde d\u00fczenlenen D\u00fcnya Kas Toplulu\u011fu&#039;nun 31. Y\u0131ll\u0131k Kongresi&#039;nde (WMS 2026) sunuldu. Sonu\u00e7lar, saRNA&#039;n\u0131n insan iskelet kas\u0131na ula\u015fabilece\u011fine ve do\u011fal olarak olu\u015fan bir proteini aktive edebilece\u011fine dair erken klinik kan\u0131tlar sa\u011fl\u0131yor. Daha Fazla Bilgi Edinin: <a href=\"https:\/\/dmdwarrior.com\/clinical-trials\/rag-18-early-phase-1-clinical-trial-for-duchenne-muscular-dystrophy\/\">NCT07282652<\/a><\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Utrofin Nedir ve Neden \u00d6nemlidir?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Utrofin, yap\u0131sal olarak distrofin ile ili\u015fkili, do\u011fal olarak olu\u015fan bir proteindir. Duchenne kas distrofisinde, i\u015flevsel distrofin eksikli\u011fi kas liflerine zarar verir. Utrofin, ki\u015finin sahip oldu\u011fu spesifik DMD mutasyonundan ba\u011f\u0131ms\u0131z olarak, kas zar\u0131nda distrofinin yerine ge\u00e7ebilir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Bu nedenle, utrofin miktar\u0131n\u0131 art\u0131rmak, hastan\u0131n belirli genetik mutasyonuna ba\u011fl\u0131 olmayan bir tedavi yakla\u015f\u0131m\u0131 sunabilir. RAG-18, bir genin yerine ge\u00e7mesini sa\u011flamak yerine, v\u00fccudun kendi utrofin \u00fcretimini art\u0131rmak \u00fczere tasarlanm\u0131\u015ft\u0131r.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">RAG-18 Nas\u0131l \u00c7al\u0131\u015f\u0131r?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">RAG-18, Ractigen Therapeutics&#039; RNA aktivasyon teknolojisini kullan\u0131r ve \u015firketin Lipid-Konjuge Oligon\u00fckleotid (LiCO\u2122) teknolojisi arac\u0131l\u0131\u011f\u0131yla uygulan\u0131r. Ayl\u0131k intraven\u00f6z inf\u00fczyon \u015feklinde verilir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Bu tedavi, utrofin \u00fcretimini art\u0131rmak i\u00e7in h\u00fccrenin kendi transkripsiyon mekanizmas\u0131n\u0131 kullanacak \u015fekilde tasarlanm\u0131\u015ft\u0131r. Viral vekt\u00f6r veya kal\u0131c\u0131 DNA d\u00fczenlemesi kullanmaz. RAG-18 ayr\u0131ca ABD'den Yetim \u0130la\u00e7 Stat\u00fcs\u00fc ve Nadir Pediatrik Hastal\u0131k Stat\u00fcs\u00fc (FDA) alm\u0131\u015ft\u0131r.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">\u0130lk \u00dc\u00e7 Kat\u0131l\u0131mc\u0131da Ne G\u00f6r\u00fcld\u00fc?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Bildirilen veriler, genetik olarak Duchenne kas distrofisi tan\u0131s\u0131 konmu\u015f, 4-15 ya\u015f aras\u0131, y\u00fcr\u00fcyebilen \u00fc\u00e7 erkek \u00e7ocu\u011fundan olu\u015fan ilk kohorttan elde edilmi\u015ftir. <strong>Hastalar ayl\u0131k 15 mg RAG-18 ald\u0131lar ve 169. g\u00fcne kadar takip edildiler.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Kas biyopsilerinde, kas zar\u0131ndaki utrofin miktar\u0131nda ba\u015flang\u0131\u00e7 de\u011ferine k\u0131yasla 3,5 ila 5,3 kat art\u0131\u015f oldu\u011fu g\u00f6r\u00fcld\u00fc.<\/strong> Ara\u015ft\u0131rmac\u0131lar ayr\u0131ca kas yap\u0131s\u0131nda da de\u011fi\u015fiklikler g\u00f6zlemlediler; bunlar aras\u0131nda kas lifi boyutunda art\u0131\u015f ve kas ya\u011f\u0131nda azalma yer al\u0131yor. Kas MR&#039;\u0131, aktif kas \u00f6demi ve iltihaplanmas\u0131yla ili\u015fkili \u00f6l\u00e7\u00fcmlerde azalma g\u00f6sterdi. Daha Fazlas\u0131n\u0131 Okuyun: <a href=\"https:\/\/dmdwarrior.com\/tr\/ractigen-therapeutics-rag-18-sarna-early-phase-1-clinical-trial-nct07282652-for-duchenne\/\" target=\"_blank\" rel=\"noreferrer noopener\">RAG-18&#039;in G\u00fcvenli\u011fi ve Tolere Edilebilirli\u011fi<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Fonksiyonel sonu\u00e7lar kar\u0131\u015f\u0131k ancak genel olarak olumlu veya istikrarl\u0131 sinyaller g\u00f6sterdi. Bir kat\u0131l\u0131mc\u0131 6 dakikal\u0131k y\u00fcr\u00fcme mesafesini 36,5 metre art\u0131r\u0131rken, bir di\u011feri y\u00fcr\u00fcme mesafesini korudu. \u00dc\u00e7\u00fcnc\u00fc bir kat\u0131l\u0131mc\u0131 ise 62,5 metrelik bir d\u00fc\u015f\u00fc\u015f ya\u015fad\u0131. Akci\u011fer \u00f6l\u00e7\u00fcmleri her \u00fc\u00e7 kat\u0131l\u0131mc\u0131da da olumlu say\u0131sal e\u011filimler g\u00f6sterdi. Kalp fonksiyonu 24 haftal\u0131k takip s\u00fcresi boyunca normal s\u0131n\u0131rlar i\u00e7inde kald\u0131.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">G\u00fcvenlik ve Sonraki Ad\u0131mlar<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">RAG-18, ilk kohortta olumlu bir g\u00fcvenlik ve tolere edilebilirlik profili sergiledi. Doz s\u0131n\u0131rlay\u0131c\u0131 toksisite, ciddi yan etki veya 3. derece veya daha y\u00fcksek \u015fiddette tedaviye ba\u011fl\u0131 yan etki g\u00f6r\u00fclmedi. Bildirilen yan etkiler hafif ve ge\u00e7iciydi.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Ayl\u0131k 30 mg doz kullanan ikinci kohortun kay\u0131tlar\u0131 tamamland\u0131 ve g\u00fcvenlik takibi devam ediyor.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Bu erken bulgular, Duchenne kas distrofisinde RNA aktivasyonunun mekanizmas\u0131na dair klinik kan\u0131t sunmakta ve RAG-18&#039;in insan iskelet kas\u0131nda utrofin seviyesini art\u0131rabilece\u011fini g\u00f6stermektedir.<\/strong> Ancak, fonksiyonel sonu\u00e7lar \u00fc\u00e7 kat\u0131l\u0131mc\u0131 aras\u0131nda farkl\u0131l\u0131k g\u00f6sterdi ve \u00e7al\u0131\u015fma hen\u00fcz erken a\u015famada.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Daha Fazlas\u0131n\u0131 Ke\u015ffedin<\/strong>:\u00a0<a href=\"https:\/\/dmdwarrior.com\/clinical-trials\/map\/\" target=\"_blank\" rel=\"noopener\">Duchenne Klinik Ara\u015ft\u0131rma Yerleri Haritas\u0131<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>Ractigen Therapeutics has presented positive first-in-human data for RAG-18 (NCT07282652), an investigational treatment for Duchenne muscular dystrophy (Duchenne) that uses small activating RNA (saRNA) to increase the production of utrophin. The Phase I study data were presented at the 31st Annual Congress of the World Muscle Society (WMS 2026) in Hiroshima, Japan. The results provide [&hellip;]<\/p>\n","protected":false},"author":11,"featured_media":8845,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[123,466,467,465,925],"class_list":["post-8843","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-china","tag-ractigen-therapeutics","tag-rag-18","tag-sarna","tag-utrophin"],"subtitle":"RAG-18, Duchenne kas distrofisi i\u00e7in potansiyel yeni bir yakla\u015f\u0131m olarak ortaya \u00e7\u0131k\u0131yor. \u0130lk veriler, kaslarda utrofinin artt\u0131\u011f\u0131n\u0131 g\u00f6steriyor ve insanlarda RNA aktivasyonuna dair ilk bak\u0131\u015f\u0131 sunuyor. Peki bu, hastalar i\u00e7in ne anlama geliyor?","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/8843","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\/11"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/comments?post=8843"}],"version-history":[{"count":2,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/8843\/revisions"}],"predecessor-version":[{"id":8846,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/8843\/revisions\/8846"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/8845"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=8843"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=8843"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=8843"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}