{"id":4466,"date":"2025-09-10T14:49:23","date_gmt":"2025-09-10T11:49:23","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=4466"},"modified":"2025-09-10T15:50:17","modified_gmt":"2025-09-10T12:50:17","slug":"avidity-biosciences-del-zota-increase-dystrophin-phase-1-2-clinical-trial","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/avidity-biosciences-del-zota-increase-dystrophin-phase-1-2-clinical-trial\/","title":{"rendered":"Del-zota (Ekzon 44 Atlama Terapisi), Faz 1\/2 Denemesinde Temel Fonksiyonel Son Noktalarda Hastal\u0131\u011f\u0131n \u0130lerlemesinin Tersine D\u00f6nd\u00fc\u011f\u00fcn\u00fc G\u00f6sterdi"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Avidity Biosciences, EXPLORE44 ve EXPLORE44-OLE \u00e7al\u0131\u015fmalar\u0131nda bir y\u0131l boyunca s\u00fcrekli del-zota tedavisi g\u00f6ren kat\u0131l\u0131mc\u0131lardan elde edilen olumlu yeni verileri duyurdu. Bu veriler, hastal\u0131k ilerlemesinin tersine d\u00f6nd\u00fc\u011f\u00fcn\u00fc ve birden fazla fonksiyonel \u00f6l\u00e7\u00fcmde ba\u015flang\u0131\u00e7 ve do\u011fal seyirle kar\u015f\u0131la\u015ft\u0131r\u0131ld\u0131\u011f\u0131nda benzeri g\u00f6r\u00fclmemi\u015f bir iyile\u015fme oldu\u011funu g\u00f6sterdi. EXPLORE44 program\u0131ndan elde edilen ek veriler, yakla\u015fan bilimsel kongrelerde sunulacakt\u0131r. \u2013 Devam\u0131n\u0131 Oku: <a href=\"https:\/\/dmdwarrior.com\/tr\/fda-designates-avidity-biosciences-delpacibart-zotadirsen-del-zota-as-a-breakthrough-therapy-for-the-treatment-of-dmd-in-individuals-with-mutations-that-allow-for-exon-44-skipping\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u00c7\u0131\u011f\u0131r A\u00e7an Terapi: Ekson 44 Atlama<\/a> \u2013<\/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=\"#del-zota-exon-44-skipping-therapy\">Del-zota: Ekzon 44 Atlama Terapisi<\/a><\/li><li><a href=\"#del-zota-phase-1-2-clinical-trial\">Del-zota: Faz 1\/2 Klinik Ara\u015ft\u0131rmas\u0131<\/a><\/li><li><a href=\"#biologics-license-application-bla-to-fda\">FDA&#039;ye Biyolojik Lisans Ba\u015fvurusu (BLA)<\/a><\/li><li><a href=\"#explore-44-exon-44-skipping-program\">EXPLORE44: Ekzon 44 Atlama Program\u0131<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"del-zota-exon-44-skipping-therapy\">Del-zota: Ekzon 44 Atlama Terapisi<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">DMD, do\u011fu\u015ftan distrofin proteininin eksikli\u011fi nedeniyle \u00e7ok k\u00fc\u00e7\u00fck ya\u015flarda ba\u015flayan ilerleyici kas hasar\u0131 ve g\u00fc\u00e7s\u00fczl\u00fc\u011f\u00fc ile karakterize nadir bir genetik rahats\u0131zl\u0131kt\u0131r. <strong>Del-zota, distrofin mRNA's\u0131n\u0131n 44. ekzonunu \u00f6zel olarak atlamak ve i\u015flevsel, neredeyse tam uzunlukta distrofin \u00fcretimini sa\u011flamak i\u00e7in iskelet kas\u0131 ve kalp dokusuna fosforodiamidat morfolino oligomerleri (PMO'lar) iletmek \u00fczere tasarlanm\u0131\u015ft\u0131r.<\/strong> Neredeyse tam uzunluktaki distrofin, \u00f6nemli i\u015flevsel alanlar\u0131 korur ve DMD44 ile ya\u015fayan insanlar i\u00e7in geli\u015fmi\u015f kas korumas\u0131 sa\u011flayabilir.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"del-zota-phase-1-2-clinical-trial\">Del-zota: Faz 1\/2 Klinik Ara\u015ft\u0131rmas\u0131<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Yakla\u015f\u0131k bir y\u0131lda, Yerden Kalkma S\u00fcresi (TTR), 4 Merdiven \u00c7\u0131kma (4SC), \u00dcst Uzuv Performans\u0131 (PUL) ve 10 Metrelik Y\u00fcr\u00fcme\/Ko\u015fu Testi (10mWRT) dahil olmak \u00fczere birden fazla i\u015flevsel \u00f6l\u00e7\u00fcmde ba\u015flang\u0131\u00e7 ve do\u011fal ge\u00e7mi\u015fe k\u0131yasla benzeri g\u00f6r\u00fclmemi\u015f iyile\u015fme<\/li>\n<\/ul>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>Kreatin kinaz\u0131nda (CK) benzeri g\u00f6r\u00fclmemi\u015f h\u0131zl\u0131 d\u00fc\u015f\u00fc\u015f, 16 ayl\u0131k takip boyunca neredeyse normal seviyelere indi ve distrofin \u00fcretiminde normalin 25%&#039;lik art\u0131\u015f\u0131, kas lifi korumas\u0131n\u0131n s\u00fcrd\u00fcr\u00fcld\u00fc\u011f\u00fcn\u00fc yans\u0131tmaktad\u0131r<\/strong><\/li>\n<\/ul>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Avidity, h\u0131zland\u0131r\u0131lm\u0131\u015f onay i\u00e7in 2025 y\u0131l\u0131 sonunda Biyolojik Lisans Ba\u015fvurusu&#039;nu (BLA) sunma yolunda ilerliyor<\/li>\n<\/ul>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"biologics-license-application-bla-to-fda\">FDA&#039;ye Biyolojik Lisans Ba\u015fvurusu (BLA)<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Avidity Ba\u015fkan\u0131 ve \u0130cra Kurulu Ba\u015fkan\u0131 Sarah Boyce, &quot;\u0130lk kez, s\u00fcrd\u00fcr\u00fclebilir kas korumas\u0131n\u0131n DMD&#039;de birden fazla temel i\u015flevsel \u00f6l\u00e7\u00fcmde anlaml\u0131 geli\u015fmelere yol a\u00e7t\u0131\u011f\u0131n\u0131 g\u00f6steren verilere sahibiz&quot; dedi.<strong>Bu e\u015fi benzeri g\u00f6r\u00fclmemi\u015f veriler, RNA&#039;y\u0131 do\u011frudan kaslara ula\u015ft\u0131rmaya y\u00f6nelik devrim niteli\u011findeki hedefli yakla\u015f\u0131m\u0131m\u0131z\u0131n etkisini vurguluyor.<\/strong> Del-Zota geli\u015ftirme program\u0131n\u0131 h\u0131zla ilerletmek ve h\u0131zland\u0131r\u0131lm\u0131\u015f onay i\u00e7in 2025 y\u0131l\u0131 sonunda FDA i\u00e7in Biyolojik Lisans Ba\u015fvurusu (BLA) sunma yolunda ilerlemek i\u00e7in acilen harekete ge\u00e7iyoruz. Bu amans\u0131z ve y\u0131k\u0131c\u0131 hastal\u0131k i\u00e7in yeni bir tedavi se\u00e7ene\u011fi aray\u0131\u015f\u0131m\u0131zda, ara\u015ft\u0131rmac\u0131lar\u0131n ve ekiplerinin, en \u00f6nemlisi de klinik \u00e7al\u0131\u015fmalar\u0131m\u0131za kat\u0131lanlar\u0131n ve ailelerinin s\u00fcrekli \u00f6zverisi i\u00e7in en derin \u015f\u00fckranlar\u0131m\u0131z\u0131 sunuyoruz.\u201d<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"explore-44-exon-44-skipping-program\">EXPLORE44: Ekzon 44 Atlama Program\u0131<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Del-Zota ile tedavi edilen deney kat\u0131l\u0131mc\u0131lar\u0131nda distrofin \u00fcretiminde normalin yakla\u015f\u0131k y\u00fczde 25'i oran\u0131nda istatistiksel olarak anlaml\u0131 art\u0131\u015flar g\u00f6r\u00fcld\u00fc ve toplam distrofin normalin 'ine kadar geri kazand\u0131r\u0131ld\u0131.<\/strong> Kreatin kinaz (CK) seviyeleri, ba\u015flang\u0131\u00e7 seviyesine k\u0131yasla &#039;den fazla h\u0131zla d\u00fc\u015ft\u00fc ve de\u011ferlendirme s\u00fcresince neredeyse normal seviyelerde kald\u0131; kat\u0131l\u0131mc\u0131lar 16 aya kadar takip edildi. Ayr\u0131ca, kat\u0131l\u0131mc\u0131lar\u0131n &#039;sinin CK seviyeleri, tedavinin birinci y\u0131l\u0131nda normal aral\u0131ktayd\u0131. <a href=\"https:\/\/dmdwarrior.com\/tr\/what-is-creatine-kinase-ck\/\" target=\"_blank\" data-type=\"post\" data-id=\"2167\" rel=\"noreferrer noopener\">Kreatin Kinaz (CK) Nedir?<\/a> \u2013<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">EXPLORE44\u00ae'nin del-zota ile tedavi edilen koluna ba\u015flay\u0131p EXPLORE44-OLE\u2122'ye devam eden toplam 17 kat\u0131l\u0131mc\u0131 (12'si ayaktan ve 5'i ayaktan) yakla\u015f\u0131k bir y\u0131l boyunca takip edildi. \u00c7al\u0131\u015fma tasar\u0131m\u0131 g\u00f6z \u00f6n\u00fcne al\u0131nd\u0131\u011f\u0131nda, baz\u0131 kat\u0131l\u0131mc\u0131lar EXPLORE44 s\u0131ras\u0131nda alt\u0131 haftada bir (Q6W) 5 mg\/kg, baz\u0131lar\u0131 ise sekiz haftada bir 10 mg\/kg ald\u0131. T\u00fcm kat\u0131l\u0131mc\u0131lar EXPLORE44-OLE s\u0131ras\u0131nda 5 mg\/kg (Q6W) doz program\u0131na ge\u00e7irildi. T\u00fcm kat\u0131l\u0131mc\u0131lar t\u00fcm de\u011ferlendirmeleri tamamlayamad\u0131. Del-Zota ile tedavi edilen kat\u0131l\u0131mc\u0131lar i\u00e7in bu birle\u015ftirilmi\u015f doz kohortlar\u0131ndan elde edilen fonksiyonel veriler, DMD44 do\u011fal ge\u00e7mi\u015fi (PRO-DMD-01) ile kar\u015f\u0131la\u015ft\u0131r\u0131ld\u0131\u011f\u0131nda iyile\u015fme g\u00f6sterdi:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>4 Merdiven T\u0131rman\u0131\u015f\u0131 (4SC)<\/strong>: Ba\u015flang\u0131\u00e7 seviyesinden 2,1 saniye iyile\u015fme sa\u011fland\u0131. Buna kar\u015f\u0131l\u0131k, do\u011fal tarih grubu ba\u015flang\u0131\u00e7 seviyesinden 2,7 saniye d\u00fc\u015f\u00fc\u015f g\u00f6sterdi (DMD44 Nat Hx N=22; del-zota N=10).<\/li>\n\n\n\n<li><strong>10 Metre Y\u00fcr\u00fcme\/Ko\u015fu Testi (10mWRT)<\/strong>: Ba\u015flang\u0131\u00e7 seviyesinden 0,7 saniye iyile\u015fme sa\u011fland\u0131. Buna kar\u015f\u0131l\u0131k, do\u011fal tarih grubu ba\u015flang\u0131\u00e7 seviyesinden 1,5 saniye d\u00fc\u015f\u00fc\u015f g\u00f6sterdi (DMD44 Nat Hx N=22; del-zota N=10).<\/li>\n\n\n\n<li><strong>Yerden Kalkma Zaman\u0131 (TTR)<\/strong>: Ba\u015flang\u0131\u00e7 seviyesinden 3,2 saniye iyile\u015fme sa\u011fland\u0131. Buna kar\u015f\u0131l\u0131k, do\u011fal tarih grubu ba\u015flang\u0131\u00e7 seviyesinden 1,6 saniye d\u00fc\u015f\u00fc\u015f g\u00f6sterdi (DMD Nat Hx N=19; del-zota N=6).<\/li>\n\n\n\n<li><strong>Kuzey Y\u0131ld\u0131z\u0131 Ayaktan De\u011ferlendirme (NSAA)<\/strong>: Sabit kald\u0131. Buna kar\u015f\u0131l\u0131k, do\u011fal tarih grubu ba\u015flang\u0131\u00e7 seviyesine g\u00f6re 2,4 puan d\u00fc\u015f\u00fc\u015f g\u00f6sterdi (DMD44 Nat Hx N=20; del-zota N=10).<\/li>\n\n\n\n<li><strong>\u00dcst Ekstremite Performans\u0131 (PUL2)<\/strong>: Ba\u015flang\u0131\u00e7 seviyesine g\u00f6re 1,5 puan iyile\u015fme. Buna kar\u015f\u0131l\u0131k, do\u011fal tarih grubu ba\u015flang\u0131\u00e7 seviyesine g\u00f6re 0,7 puan d\u00fc\u015f\u00fc\u015f g\u00f6sterdi.ii Hem y\u00fcr\u00fcyen hem de y\u00fcr\u00fcyemeyen kat\u0131l\u0131mc\u0131larda benzer PUL iyile\u015fmeleri g\u00f6r\u00fcld\u00fc (DMD44 Nat Hx N=27; del-zota N=17).<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>EXPLORE44-OLE denemesindeki t\u00fcm kat\u0131l\u0131mc\u0131larda g\u00fcvenlik de\u011ferlendirildi ve del-zota uzun vadede olumlu bir g\u00fcvenlik ve tolere edilebilirlik profili g\u00f6stermeye devam etti.<\/strong> Tedaviyle ortaya \u00e7\u0131kan advers olaylar\u0131n (TEAE&#039;ler) \u00e7o\u011fu hafif veya orta \u015fiddetteydi; en s\u0131k g\u00f6r\u00fclen TEAE&#039;ler (3&#039;ten fazla kat\u0131l\u0131mc\u0131da g\u00f6r\u00fclen) \u00fcst solunum yolu semptomlar\u0131, ishal, d\u00fc\u015fme, s\u0131rt a\u011fr\u0131s\u0131 ve ba\u015f a\u011fr\u0131s\u0131yd\u0131. Bir kat\u0131l\u0131mc\u0131, a\u015f\u0131r\u0131 duyarl\u0131l\u0131k olay\u0131n\u0131n ard\u0131ndan EXPLORE44-OLE tedavisini b\u0131rakt\u0131.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Avidity, 2025 y\u0131l\u0131 sonunda ABD G\u0131da ve \u0130la\u00e7 Dairesi&#039;ne (FDA) bir BLA sunma yolunda ilerlemektedir.<\/strong> Bu, \u015eirketin 12 ayl\u0131k bir s\u00fcre i\u00e7inde planlad\u0131\u011f\u0131 \u00fc\u00e7 BLA ba\u015fvurusunun ilki olacak. Avidity, tam k\u00fcresel onay\u0131 desteklemek i\u00e7in do\u011frulay\u0131c\u0131 bir \u00e7al\u0131\u015fma i\u00e7in haz\u0131rl\u0131klar\u0131n\u0131 s\u00fcrd\u00fcr\u00fcyor.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Devam\u0131n\u0131 oku<\/strong>: <a href=\"https:\/\/dmdwarrior.com\/tr\/upcoming-exon-44-skipping-therapies-for-dmd\/\" target=\"_blank\" rel=\"noreferrer noopener\">Duchenne Musk\u00fcler Distrofi Tedavisinde Yakla\u015fan Ekzon 44 Atlama Terapileri<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Daha fazla bilgi edin<\/strong>: <a href=\"https:\/\/dmdwarrior.com\/tr\/mutations-and-deletions-amenable-to-exon-44-skipping-therapies-dmd\/\" target=\"_blank\" rel=\"noreferrer noopener\">Duchenne Musk\u00fcler Distrofisi \u0130\u00e7in Ekzon 44 Atlama Terapilerine Uygun Mutasyonlar ve Delesyonlar<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>Avidity Biosciences announced positive new data from participants treated continuously with del-zota for one year in the EXPLORE44 and EXPLORE44-OLE trials. These data demonstrated reversal of disease progression and unprecedented improvement compared to baseline and natural history across multiple functional measures. Additional data from the EXPLORE44 program will be presented at upcoming scientific congresses. &#8211; [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":4468,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[126,298,129,73,141,127,246,299,359],"class_list":["post-4466","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-avidity-biosciences","tag-del-zota","tag-dmd44","tag-dystrophin","tag-exon-44","tag-exon-44-skipping","tag-exon-44-skipping-therapies","tag-explore44","tag-full-length-dystrophin"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/4466","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=4466"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/4466\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/4468"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=4466"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=4466"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=4466"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}