{"id":4166,"date":"2025-07-23T16:48:01","date_gmt":"2025-07-23T13:48:01","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=4166"},"modified":"2025-07-23T16:50:07","modified_gmt":"2025-07-23T13:50:07","slug":"the-fda-designates-pbgene-dmd-as-an-orphan-drug-by-precision-biosciences-to-treat-duchenne-muscular-dystrophy","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/the-fda-designates-pbgene-dmd-as-an-orphan-drug-by-precision-biosciences-to-treat-duchenne-muscular-dystrophy\/","title":{"rendered":"FDA, Precision BioSciences Taraf\u0131ndan Duchenne Kas Distrofisini Tedavi Etmek \u0130\u00e7in Yetim \u0130la\u00e7 Olarak PBGENE-DMD'yi Belirledi"},"content":{"rendered":"

Y\u00fcksek kar\u015f\u0131lanmam\u0131\u015f ihtiya\u00e7lara sahip hastal\u0131klar i\u00e7in in vivo gen d\u00fczenleme terapileri geli\u015ftirmek amac\u0131yla yenilik\u00e7i tescilli ARCUS\u00ae platformunu kullanan klinik a\u015famada gen d\u00fczenleme \u015firketi Precision BioSciences, bug\u00fcn ABD G\u0131da ve \u0130la\u00e7 Dairesi'nin (FDA) PBGENE-DMD'ye Duchenne kas distrofisi (DMD) tedavisi i\u00e7in Yetim \u0130la\u00e7 Tan\u0131m\u0131 verdi\u011fini duyurdu.<\/p>\n\n\n\n

Precision BioSciences Geli\u015ftirme ve \u0130\u015fletme Direkt\u00f6r\u00fc Cindy Atwell, "FDA'den PBGENE-DMD i\u00e7in Yetim \u0130la\u00e7 Tan\u0131m\u0131 al\u0131nmas\u0131, Duchenne kas distrofisi ile ya\u015fayan erkek \u00e7ocuklar i\u00e7in kas fonksiyonunu zamanla \u00f6nemli \u00f6l\u00e7\u00fcde iyile\u015ftiren g\u00fcvenli tedaviler sunma konusundaki kar\u015f\u0131lanmam\u0131\u015f muazzam ihtiyac\u0131 ve aciliyeti vurguluyor," dedi. "Bu d\u00fczenleyici d\u00f6n\u00fcm noktas\u0131, yak\u0131n zamanda ald\u0131\u011f\u0131m\u0131z Nadir Pediatrik Hastal\u0131k tan\u0131m\u0131na dayan\u0131yor ve klinik \u00f6ncesi kan\u0131tlar\u0131m\u0131zla birlikte, bu program\u0131 klini\u011fe do\u011fru ilerletmemizde bize muazzam bir g\u00fcven veriyor. \u0130leriye bakt\u0131\u011f\u0131m\u0131zda, PBGENE-DMD'yi d\u00fczenleyici d\u00f6n\u00fcm noktalar\u0131na do\u011fru ilerletirken FDA ile aktif diyalog halindeyiz ve klinik verilerin 2026'da elde edilmesi bekleniyor."<\/p>\n\n\n\n

Devam\u0131n\u0131 oku:<\/strong> Precision BioSciences, Duchenne Musk\u00fcler Distrofi Tedavisi i\u00e7in PBGENE-DMD'ye \u0130li\u015fkin Yeni Klinik \u00d6ncesi Verileri \u00d6ne \u00c7\u0131kar\u0131yor<\/a><\/p>\n\n\n\n

PBGENE-DMD nedir?<\/h2>\n\n\n\n

PBGENE-DMD, Precision'\u0131n DMD tedavisine y\u00f6nelik geli\u015ftirme program\u0131d\u0131r. Bu yakla\u015f\u0131m, fonksiyonel sonu\u00e7lar\u0131 iyile\u015ftirmek amac\u0131yla v\u00fccuttaki distrofin proteininin neredeyse tam uzunlu\u011funu geri kazand\u0131rmak amac\u0131yla distrofin geninin 45-55. ekzonlar\u0131n\u0131 \u00e7\u0131karmak i\u00e7in tek bir AAV'de tek seferlik uygulama yoluyla verilen iki tamamlay\u0131c\u0131 ARCUS n\u00fckleaz\u0131n\u0131 kullan\u0131r.<\/strong> PBGENE-DMD, DMD hasta pop\u00fclasyonunun 60%'sinden fazlas\u0131n\u0131 hedeflemektedir.<\/p>\n\n\n\n

Klinik \u00f6ncesi \u00e7al\u0131\u015fmalarda, PBGENE-DMD, DMD'nin ilerlemesinde rol oynayan temel kas tiplerini hedefleme yetene\u011fini g\u00f6sterdi ve insanla\u015ft\u0131r\u0131lm\u0131\u015f bir DMD fare modelinde \u00f6nemli, kal\u0131c\u0131 i\u015flevsel iyile\u015ftirmeler \u00fcretti. PBGENE-DMD, v\u00fccudun kardiyak doku ve \u00e7e\u015fitli temel iskelet kas gruplar\u0131 dahil olmak \u00fczere birden fazla kasta neredeyse tam uzunlukta i\u015flevsel bir distrofin proteini \u00fcretme yetene\u011fini geri kazand\u0131rd\u0131. Ek olarak, PBGENE-DMD, uzun vadeli dayan\u0131kl\u0131l\u0131k ve s\u00fcrekli i\u015flevsel iyile\u015ftirme i\u00e7in kritik oldu\u011funa inan\u0131lan uydu kas k\u00f6k h\u00fccrelerini d\u00fczenledi.<\/p>","protected":false},"excerpt":{"rendered":"

Precision BioSciences, a clinical stage gene editing company utilizing its novel proprietary ARCUS\u00ae platform to develop in vivo gene editing therapies for diseases with high unmet need, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for PBGENE-DMD for the treatment of Duchenne muscular dystrophy (DMD). \u201cReceipt of Orphan […]<\/p>\n","protected":false},"author":1,"featured_media":4169,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[88,195,314,313],"class_list":["post-4166","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-fda","tag-orphan-drug","tag-pbgene-dmd","tag-precision-biosciences"],"subtitle":"FDA, Duchenne kas distrofisi i\u00e7in PBGENE-DMD'ye Yetim \u0130la\u00e7 Tan\u0131m\u0131 verildi.","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/4166","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=4166"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/4166\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/4169"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=4166"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=4166"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=4166"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}