{"id":4065,"date":"2025-07-10T11:42:12","date_gmt":"2025-07-10T08:42:12","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=4065"},"modified":"2025-07-10T11:43:34","modified_gmt":"2025-07-10T08:43:34","slug":"jcr-pharmaceuticals-proprietary-just-aav-capsids-may-be-used-in-the-development-of-duchenne-cures","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/jcr-pharmaceuticals-proprietary-just-aav-capsids-may-be-used-in-the-development-of-duchenne-cures\/","title":{"rendered":"JCR Pharmaceuticals Tescilli JUST-AAV Kapsidleri Duchenne Tedavilerinin Geli\u015ftirilmesinde Kullan\u0131labilir"},"content":{"rendered":"<p class=\"wp-block-paragraph\">AstraZeneca&#039;nin nadir hastal\u0131klar yan kurulu\u015fu olan Alexion, Japon firmas\u0131 JCR Pharmaceuticals&#039;den \u00f6zel gen terapisi ara\u00e7lar\u0131n\u0131n lisans\u0131n\u0131 al\u0131yor.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>8 Haziran 2025&#039;te duyurulan anla\u015fma, Alexion&#039;ye, Alexion&#039;nin genomik ila\u00e7 programlar\u0131ndan be\u015fine kadar\u0131nda kullan\u0131lmak \u00fczere JCR&#039;nin JUST-AAV kapsidlerine eri\u015fim sa\u011fl\u0131yor.<\/strong> Kar\u015f\u0131l\u0131\u011f\u0131nda Alexion, JCR&#039;ye a\u00e7\u0131klanmayan bir pe\u015fin \u00fccretin yan\u0131 s\u0131ra ara\u015ft\u0131rma ve geli\u015ftirmeyle ilgili 225 milyon ABD dolar\u0131na kadar kilometre ta\u015f\u0131 \u00f6demeleri ve sat\u0131\u015f kilometre ta\u015flar\u0131 i\u00e7in ek 600 milyon ABD dolar\u0131 \u00f6deyecek. JCR ayr\u0131ca sat\u0131\u015flarla ilgili kademeli telif haklar\u0131 da alacak, ancak ayr\u0131nt\u0131lar verilmedi.<\/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=\"#whar-is-just-aav-capsids\">JUST-AAV Kapsid Nedir?<\/a><\/li><li><a href=\"#just-aav-capsids-may-be-used-for-duchenne-cures\">JUST-AAV Kapsidleri Duchenne Tedavisinde Kullan\u0131labilir<\/a><\/li><li><a href=\"#pre-clinical-trials-of-just-aav-capsids\">JUST-AAV Kapsidlerinin Klinik \u00d6ncesi Denemeleri<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"whar-is-just-aav-capsids\">JUST-AAV Kapsid Nedir?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>JUST-AAV, modifiye edilmi\u015f adeno-ili\u015fkili vir\u00fcs (AAV) vekt\u00f6rlerini kullanan tescilli bir platform teknolojisidir.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Teknoloji, se\u00e7ilmi\u015f dokular, organlar veya kan-beyin bariyerindeki resept\u00f6rlere kar\u015f\u0131 minyat\u00fcrle\u015ftirilmi\u015f antikorlar\u0131n kapsid y\u00fczeyine yerle\u015ftirilmesini ve bu dokulara ve organlara hedefli iletimi art\u0131rmay\u0131 i\u00e7eriyor.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Daha ileri kapsid modifikasyonlar\u0131, hedef d\u0131\u015f\u0131 etkileri en aza indirir ve g\u00fcvenli\u011fi art\u0131r\u0131r. Ad\u0131, \u00e7e\u015fitli hastal\u0131klarda geni\u015f uygulama potansiyelini yans\u0131tan &quot;JCR&quot;, &quot;Organ\u0131n nihai hedefi&quot;, &quot;Hedef d\u0131\u015f\u0131 iletimi \u00f6nleme&quot; ve &quot;D\u00f6n\u00fc\u015ft\u00fcr\u00fcc\u00fc teknoloji&quot; kelimelerinden t\u00fcretilmi\u015ftir.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"just-aav-capsids-may-be-used-for-duchenne-cures\">JUST-AAV Kapsidleri Duchenne Tedavisinde Kullan\u0131labilir<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Alexion&#039;nin kapsidleri hangi endikasyonlar i\u00e7in kullanaca\u011f\u0131 da belirtilmedi. AstraZeneca&#039;nin nadir hastal\u0131k hatt\u0131, miyastenia gravis ve lupus nefriti gibi hastal\u0131klarda orta ila ileri evre varl\u0131klarla doludur.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>JCR, tescilli JUST-AAV kapsidlerinin birka\u00e7 \u00e7e\u015fitte geldi\u011fini, \u00f6zellikle &quot;karaci\u011feri koruyan&quot;, kaslar\u0131 hedef alan ve beyni hedef alan varyantlar\u0131 \u00f6ne \u00e7\u0131kard\u0131\u011f\u0131n\u0131 iddia ediyor.<\/strong> Gen tedavilerinden kaynaklanan karaci\u011fer hasarlar\u0131, bu y\u0131l\u0131n ba\u015flar\u0131nda Duchenne kas distrofisi i\u00e7in Sarepta&#039;n\u0131n gen tedavisi olan Elevidys&#039;yi alan iki hastan\u0131n karaci\u011fer hasar\u0131 nedeniyle \u00f6lmesi nedeniyle son zamanlarda yo\u011fun bir inceleme alt\u0131na al\u0131nd\u0131. \u2013 Devam\u0131n\u0131 Oku: <a href=\"https:\/\/dmdwarrior.com\/tr\/sarepta-reported-elevidys-gene-therapy-for-duchenne-muscular-dystrophy-will-not-be-used-in-non-ambulatory-patients\/\">Elevidys Gen Terapisi, y\u00fcr\u00fcyemeyen hastalarda kullan\u0131lmayacakt\u0131r<\/a> \u2013<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"pre-clinical-trials-of-just-aav-capsids\">JUST-AAV Kapsidlerinin Klinik \u00d6ncesi Denemeleri<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Ara\u015ft\u0131rmac\u0131lar, transferin resept\u00f6r\u00fcne ba\u011flanan minyat\u00fcr bir antikoru AAV kapsidine dahil ederek beyni hedef alan bir AAV vekt\u00f6r\u00fc (beyni hedef alan JUST-AAV) olu\u015fturmay\u0131 ba\u015fard\u0131lar. Ayr\u0131ca, AAV kapsid dizisindeki benzersiz de\u011fi\u015fiklikler, bilinen bir yan etki kayna\u011f\u0131 olan karaci\u011ferdeki AAV vekt\u00f6r\u00fc birikimini \u00f6nemli \u00f6l\u00e7\u00fcde azaltt\u0131.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Fare \u00e7al\u0131\u015fmalar\u0131nda, JUST-AAV vekt\u00f6r\u00fc, AAV9&#039;a k\u0131yasla beyinde 77 kat daha fazla ye\u015fil floresan protein (GFP) ekspresyonu sa\u011flarken, 99%&#039;nin karaci\u011fere olan tropizmini azaltt\u0131. Beyin hedeflemesini ve BBB ge\u00e7irgenli\u011fini daha da art\u0131rmak i\u00e7in ara\u015ft\u0131rmac\u0131lar, transferin resept\u00f6r\u00fc d\u0131\u015f\u0131ndaki resept\u00f6rlere ba\u011flanan ek molek\u00fclleri AAV kapsidine dahil ettiler.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Maymun \u00e7al\u0131\u015fmalar\u0131nda, bu bispesifik vekt\u00f6r\u00fcn beyne gen iletimi verimlili\u011finin AAV9&#039;a k\u0131yasla birka\u00e7 kat daha fazla artt\u0131\u011f\u0131, karaci\u011fer ve dorsal k\u00f6k ganglionlar\u0131 gibi potansiyel olarak sorunlu dokulardaki enfeksiyonun ise 90%&#039;den daha fazla azald\u0131\u011f\u0131 g\u00f6sterildi.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">JUST-AAV&#039;nin n\u00f6ronal seroid lipof\u00fcsinozisli bir fare modeline uygulanmas\u0131, n\u00f6betler gibi semptomlar\u0131n ortadan kalkmas\u0131n\u0131 ve i\u015flevsel bir iyile\u015fmeye kadar ya\u015fam s\u00fcresinin uzamas\u0131n\u0131 sa\u011flam\u0131\u015ft\u0131r. Bu sonu\u00e7lar, yeni geli\u015ftirilen JUST-AAV teknolojisinin, geleneksel gen iletim vekt\u00f6rlerine k\u0131yasla daha g\u00fcvenli ve daha etkili bir gen terapisi potansiyeli sundu\u011funu g\u00f6stermektedir. \u2013 Daha Fazla Bilgi Edinin: <a href=\"https:\/\/jcrpharm.com\/updatedjcr-pharmaceuticals-presents-preclinical-gene-therapy-data-at-asgct-28th-annual-meeting\/\" target=\"_blank\" rel=\"noreferrer noopener\">JCR Pharmaceuticals klinik \u00f6ncesi gen tedavisi verilerini sunuyor<\/a> \u2013<\/p>","protected":false},"excerpt":{"rendered":"<p>Alexion, AstraZeneca\u2019s rare disease subsidiary, is licensing specialized gene therapy vehicles from the Japanese firm JCR Pharmaceuticals. The deal, announced on 8th June 2025, gives Alexion access to JCR\u2019s JUST-AAV capsids to use in up to five of Alexion\u2019s genomic medicines programs. In exchange, Alexion will pay JCR an undisclosed upfront fee plus up to [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":4069,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[386,387,388,385],"class_list":["post-4065","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-alexion","tag-astrazeneca","tag-genomic-medicines","tag-jcr-pharmaceuticals"],"subtitle":"JCR Pharmaceuticals, genomik ila\u00e7lar geli\u015ftirmek amac\u0131yla JCR'nin yeni, tescilli JUST-AAV kapsidleri i\u00e7in Alexion, AstraZeneca Nadir Hastal\u0131k (\u201cAlexion\u201d) ile bir lisans anla\u015fmas\u0131 imzalad\u0131\u011f\u0131n\u0131 duyurdu.","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/4065","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=4065"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/4065\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/4069"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=4065"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=4065"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=4065"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}