{"id":3027,"date":"2026-08-06T17:27:14","date_gmt":"2026-08-06T14:27:14","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=3027"},"modified":"2026-08-06T17:27:17","modified_gmt":"2026-08-06T14:27:17","slug":"ips-heart-full-length-dystrophin-duchenne","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/ips-heart-full-length-dystrophin-duchenne\/","title":{"rendered":"IPS HEART, Duchenne Musk\u00fcler Distrofisinde \u0130ki Rejeneratif H\u00fccre Terapisi ile Tam Uzunlukta Distrofin \u00dcretimini Geri Kazand\u0131rmay\u0131 Hedefliyor"},"content":{"rendered":"<p class=\"wp-block-paragraph\">IPS HEART, Duchenne kas distrofisi (DMD) i\u00e7in geli\u015ftirdi\u011fi deneysel kalp kas\u0131 tedavisi ISX9-CPC i\u00e7in FDA Nadir Pediatrik Hastal\u0131k Tan\u0131m\u0131&#039;n\u0131 ald\u0131ktan sonra bir ba\u015fka \u00f6nemli d\u00fczenleyici kilometre ta\u015f\u0131n\u0131 duyurdu. <strong>\u015eirket, yeni kas olu\u015fumunu sa\u011flamak ve tam uzunluktaki distrofini geri kazand\u0131rmak i\u00e7in tasarlanm\u0131\u015f deneysel bir iskelet kas\u0131 terapisi olan GIVI-MPCs ile birlikte, hem kalp hem de iskelet kas\u0131 hasar\u0131n\u0131 ele almay\u0131 ama\u00e7layan rejeneratif bir yakla\u015f\u0131m izliyor.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">IPS HEART, hastal\u0131\u011f\u0131n ilerlemesini yava\u015flatmaya odaklanan bir\u00e7ok mevcut tedavi yakla\u015f\u0131m\u0131n\u0131n aksine, rejeneratif t\u0131p platformunun, tam uzunlukta distrofin \u00fcretebilen tamamen yeni insan kas h\u00fccreleri olu\u015fturmak \u00fczere tasarland\u0131\u011f\u0131n\u0131 belirtiyor.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Mevcut DMD Tedavilerinden Farkl\u0131 Bir Strateji<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Mevcut veya deneysel Duchenne tedavilerinin \u00e7o\u011fu (ekzon atlama terapileri ve mikro-distrofin gen terapileri dahil) yaln\u0131zca belirli hasta gruplar\u0131na yard\u0131mc\u0131 olur ve genellikle distrofin proteininin tamam\u0131n\u0131 geri kazand\u0131rmaz.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">IPS HEART, bas\u0131n b\u00fclteninde bu yakla\u015f\u0131mlar\u0131n klinik s\u0131n\u0131rlamalara sahip oldu\u011funu ve tam uzunlukta distrofini geri kazand\u0131ramad\u0131\u011f\u0131n\u0131 belirtirken, ind\u00fcklenmi\u015f pluripotent k\u00f6k h\u00fccre (iPSC) platformunun ise preklinik \u00e7al\u0131\u015fmalarda tam uzunlukta distrofin ifade eden yeni fonksiyonel kas h\u00fccreleri \u00fcretmek \u00fczere tasarland\u0131\u011f\u0131n\u0131 ifade etmektedir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u015eirket iki ayr\u0131 rejeneratif tedavi y\u00f6ntemi geli\u015ftiriyor:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>Kalp kas\u0131 i\u00e7in ISX9-CPC<\/strong><\/li>\n\n\n\n<li><strong>\u0130skelet kas\u0131 i\u00e7in GIVI-MPCs<\/strong><\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">Bu tedavilerin amac\u0131, kas\u0131n bozulmas\u0131n\u0131 sadece yava\u015flatmak de\u011fil, hasar g\u00f6rm\u00fc\u015f kas\u0131 yeniden olu\u015fturmakt\u0131r.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">ISX9-CPC: Kalp Kas\u0131n\u0131n Yeniden Olu\u015fturulmas\u0131<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Duchenne kas distrofisinde kalp hastal\u0131\u011f\u0131 \u00f6nde gelen \u00f6l\u00fcm nedenlerinden biridir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">IPS HEART&#039;a g\u00f6re, ISX9-CPC, kalpteki fibrotik skar dokusunu yeni \u00fcretilen kas\u0131labilen kalp kas\u0131yla de\u011fi\u015ftirmek \u00fczere tasarlanm\u0131\u015ft\u0131r. Klinik \u00f6ncesi \u00e7al\u0131\u015fmalar, tedavi sonras\u0131nda kalp fonksiyonunda iyile\u015fme ve ejeksiyon fraksiyonunda art\u0131\u015f oldu\u011funu g\u00f6stermi\u015ftir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u015eirket ayr\u0131ca resmi internet sitesinde ek preklinik bulgulara da dikkat \u00e7ekerek \u015funlar\u0131 belirtiyor:<\/p>\n\n\n\n<blockquote class=\"wp-block-quote is-layout-flow wp-block-quote-is-layout-flow\">\n<p class=\"wp-block-paragraph\">\u201c&quot;ISX9-CPC ile ilgili olarak, patentli ISX9-CPC&#039;mizin tek bir tedavisinin, kalp krizinden 90 g\u00fcn sonra \u00f6l\u00fc skar dokusunu 70% oran\u0131nda azaltt\u0131\u011f\u0131 ve kontrol grubuna k\u0131yasla kalp fonksiyonunu 60%&#039;nin \u00fczerinde EF iyile\u015fmesiyle geri kazand\u0131rd\u0131\u011f\u0131, in vivo bir kavram kan\u0131t\u0131 niteli\u011findeki hakemli bir \u00e7al\u0131\u015fmada g\u00f6sterilmi\u015ftir.&quot;\u201d<\/p>\n<\/blockquote>\n\n\n\n<p class=\"wp-block-paragraph\">Bu bulgular Duchenne hastalar\u0131ndan ziyade kalp krizi modelleriyle ilgili olsa da, IPS HEART bunlar\u0131 kalp h\u00fccresi tedavisinin rejeneratif potansiyelini destekleyen kan\u0131t olarak sunmaktad\u0131r.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">GIVI-MPCs: Tam Boy Distrofin ile Yeni \u0130skelet Kaslar\u0131 Olu\u015fturma<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u015eirketin ikinci program\u0131 olan GIVI-MPCs, Duchenne kas distrofisinden etkilenen iskelet kaslar\u0131n\u0131n yenilenmesine odaklanmaktad\u0131r.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Bas\u0131n a\u00e7\u0131klamas\u0131na g\u00f6re, GIVI-MPCs, \u00e7e\u015fitli distrofik hayvan modellerinde tam uzunlukta insan distrofinini geri kazand\u0131r\u0131rken yeni insan iskelet kas\u0131 dokusu olu\u015fturmak \u00fczere tasarlanm\u0131\u015ft\u0131r.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">IPS HEART, resmi internet sitesinde bu iddiay\u0131 daha da detayland\u0131rarak \u015funlar\u0131 belirtiyor:<\/p>\n\n\n\n<blockquote class=\"wp-block-quote is-layout-flow wp-block-quote-is-layout-flow\">\n<p class=\"wp-block-paragraph\">\u201cGIVI-MPC ile, mdx farelerinde ve distrofik domuzlarda 100% tam uzunlukta insan distrofini ile birlikte \u00f6nemli yeni insan iskelet kas\u0131 olu\u015fturduk.\u201d<\/p>\n<\/blockquote>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Bu, \u015firketin \u015fu anda \u00f6ne s\u00fcrd\u00fc\u011f\u00fc en g\u00fc\u00e7l\u00fc iddialardan biridir \u00e7\u00fcnk\u00fc tam uzunluktaki distrofin, mevcut gen terapisi yakla\u015f\u0131mlar\u0131yla \u00fcretilen k\u0131salt\u0131lm\u0131\u015f mikro-distrofinin aksine, sa\u011fl\u0131kl\u0131 kaslarda bulunan do\u011fal olarak olu\u015fan proteine \u00e7ok benzemektedir.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Ancak bu sonu\u00e7lar\u0131n klinik \u00f6ncesi nitelikte oldu\u011funu ve hen\u00fcz insan klinik denemelerinde kan\u0131tlanmad\u0131\u011f\u0131n\u0131 belirtmek \u00f6nemlidir.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">\u0130nsanlar \u00dczerinde Klinik Denemelerin 2027'de Ba\u015flanmas\u0131 Planlan\u0131yor<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>IPS HEART, Ara\u015ft\u0131rma Ama\u00e7l\u0131 Yeni \u0130la\u00e7 (IND) ba\u015fvurusunu ABD G\u0131da ve \u0130la\u00e7 \u0130daresi'ne (FDA) sunmay\u0131 ve d\u00fczenleyici onay al\u0131nmas\u0131 halinde GIVI-MPCs'nin insanlar \u00fczerinde ilk klinik denemesine 2027 y\u0131l\u0131nda ba\u015flamay\u0131 planlad\u0131\u011f\u0131n\u0131 a\u00e7\u0131klad\u0131.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">E\u011fer onaylan\u0131rsa, bu, \u015firketin rejeneratif stratejisinin Duchenne kas distrofisi olan ki\u015filerde iskelet kas\u0131n\u0131 ve tam uzunlukta distrofini g\u00fcvenli bir \u015fekilde geri kazand\u0131r\u0131p kazand\u0131ramayaca\u011f\u0131n\u0131 de\u011ferlendirmek i\u00e7in ilk f\u0131rsat olacakt\u0131r.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Bu Neden \u00d6nemli?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Duchenne ara\u015ft\u0131rmalar\u0131ndaki en b\u00fcy\u00fck bilimsel zorluklardan biri, kaybedilen kas dokusunu yerine koyarken distrofin proteininin tamam\u0131n\u0131 geri kazand\u0131rman\u0131n bir yolunu bulmakt\u0131r.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">IPS HEART, rejeneratif h\u00fccre tedavisi platformunun nihayetinde her iki hedefi de ayn\u0131 anda ele alabilece\u011fine inan\u0131yor:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>Hasar g\u00f6rm\u00fc\u015f kalp kas\u0131n\u0131 yenilenmesi<\/strong><\/li>\n\n\n\n<li><strong>Yeni iskelet kas\u0131 olu\u015fturulmas\u0131<\/strong><\/li>\n\n\n\n<li><strong>Distrofinin tam uzunlu\u011funu geri kazand\u0131rmas\u0131<\/strong><\/li>\n\n\n\n<li><strong>Mutasyona \u00f6zg\u00fc tedavilere k\u0131yasla potansiyel olarak daha geni\u015f bir uygulama alan\u0131 sa\u011flayabilir.<\/strong><\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">Bu umut vadeden preklinik bulgular\u0131n anlaml\u0131 klinik faydalara d\u00f6n\u00fc\u015f\u00fcp d\u00f6n\u00fc\u015fmeyece\u011fi, dikkatlice y\u00fcr\u00fct\u00fclen insan denemeleriyle belirlenecektir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u015eimdi Ke\u015ffedin<\/strong>:&nbsp;<a href=\"https:\/\/dmdwarrior.com\/clinical-trials\/\" target=\"_blank\" rel=\"noreferrer noopener\">DMDWarrioR Klinik Ara\u015ft\u0131rmalar Merkezi<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>IPS HEART has announced another important regulatory milestone after receiving FDA Rare Pediatric Disease Designation for ISX9-CPC, its investigational cardiac muscle therapy for Duchenne muscular dystrophy (DMD). Together with GIVI-MPCs, an experimental skeletal muscle therapy designed to generate new muscle and restore full-length dystrophin, the company is pursuing a regenerative approach that aims to address [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":8332,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[817,359,857,856,858,259],"class_list":["post-3027","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-dmd-treatment","tag-full-length-dystrophin","tag-givi-mpcs","tag-isx9-cpc","tag-muscle-regeneration","tag-stem-cell-therapy"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/3027","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=3027"}],"version-history":[{"count":10,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/3027\/revisions"}],"predecessor-version":[{"id":8333,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/3027\/revisions\/8333"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/8332"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=3027"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=3027"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=3027"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}