{"id":6203,"date":"2026-02-11T17:08:53","date_gmt":"2026-02-11T14:08:53","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=6203"},"modified":"2026-02-11T17:08:55","modified_gmt":"2026-02-11T14:08:55","slug":"precision-biosciences-pbgene-dmd-function-dmd-phase-1-2-clinical-study","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/precision-biosciences-pbgene-dmd-function-dmd-phase-1-2-clinical-study\/","title":{"rendered":"Precision BioSciences, Duchenne Musk\u00fcler Distrofisi Tedavisi i\u00e7in \u0130lk S\u0131n\u0131f \u0130la\u00e7 Olan PBGENE-DMD&#039;nin Ara\u015ft\u0131rma Ama\u00e7l\u0131 Yeni \u0130la\u00e7 Ba\u015fvurusuna (IND) ABD FDA Onay\u0131n\u0131 Ald\u0131"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Precision BioSciences, \u015eirketin ABD G\u0131da ve \u0130la\u00e7 \u0130daresi&#039;nden (FDA) \u00c7al\u0131\u015fmaya Devam Edilmesine \u0130zin Verilmesine \u0130li\u015fkin Bildirim ald\u0131\u011f\u0131n\u0131 duyurdu (FDA). <strong>Bu, Precision BioSciences&#039;nin, ayakta tedavi g\u00f6ren Duchenne kas distrofisi (DMD) hastalar\u0131n\u0131n tedavisi i\u00e7in PBGENE-DMD&#039;nin Faz 1\/2 klinik denemesi olan FUNCTION-DMD i\u00e7in, son derece uzmanla\u015fm\u0131\u015f klinik deneme merkezlerinde Etik Kurul faaliyetlerini ve klinik deneme merkezi aktivasyonunu ba\u015flatmas\u0131na olanak tan\u0131r.<\/strong> Bu \u00e7al\u0131\u015fman\u0131n amac\u0131, DMD hastal\u0131\u011f\u0131na yakalanm\u0131\u015f hastalarda distrofin protein ekspresyonu ve fonksiyonel sonu\u00e7lar da dahil olmak \u00fczere g\u00fcvenli\u011fi, tolerans\u0131 ve etkinli\u011fi de\u011ferlendirmektir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u2013 ABD&#039;de FDA \u00c7al\u0131\u015fmas\u0131n\u0131n \u0130lerleyebilece\u011fine Dair Bildirim, Duchenne kas distrofisi (DMD) hastalar\u0131nda FUNCTION-DMD Faz 1\/2 klinik \u00e7al\u0131\u015fmas\u0131 i\u00e7in klinik ara\u015ft\u0131rma sahas\u0131 aktivasyonunun ba\u015flat\u0131lmas\u0131n\u0131 m\u00fcmk\u00fcn k\u0131l\u0131yor.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u2013 D\u00fcnya standartlar\u0131nda bir\u00e7ok DMD klinik ara\u015ft\u0131rma merkezinde Kurumsal Etik Kurul (IRB) s\u00fcreci devam etmektedir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u2013 PBGENE-DMD, DMD&#039;nin alt\u0131nda yatan genetik nedeni yeni bir tek seferlik gen d\u00fczenleme terapisiyle d\u00fczeltmek i\u00e7in tasarlanm\u0131\u015f, canl\u0131 organizmada gen d\u00fczenleme \u00fczerine ara\u015ft\u0131rma a\u015famas\u0131nda olan bir \u00fcr\u00fcnd\u00fcr.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u2013 \u015eirket, Kas Distrofisi Derne\u011fi konferans\u0131ndan sonra Mart ay\u0131nda, PBGENE-DMD ve FUNCTION-DMD klinik \u00e7al\u0131\u015fmalar\u0131n\u0131 ele almak \u00fczere, hasta savunucular\u0131 da dahil olmak \u00fczere \u00f6nde gelen kanaat \u00f6nderlerinin kat\u0131l\u0131m\u0131yla sanal bir etkinlik d\u00fczenlemeyi planl\u0131yor.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">PBGENE-DMD nedir?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">PBGENE-DMD, Precision&#039;\u0131n tamamen sahip oldu\u011fu, s\u0131n\u0131f\u0131nda ilk olan ve gen eksizyon yakla\u015f\u0131m\u0131n\u0131 kullanan bir in vivo gen d\u00fczenleme program\u0131d\u0131r. Bu program, DMD&#039;li hastalar\u0131n en b\u00fcy\u00fck molek\u00fcler alt k\u00fcmesini olu\u015fturan ve yakla\u015f\u0131k 60% olan &quot;s\u0131cak nokta&quot; b\u00f6lgesi olarak bilinen 45 ile 55. eksonlar aras\u0131ndaki mutasyonlara sahip hastalarda distrofin genini kal\u0131c\u0131 olarak d\u00fczeltmeyi ama\u00e7lamaktad\u0131r. <strong>Tedavinin amac\u0131, distrofin geninin neredeyse tam uzunlukta i\u015flevsel bir distrofin proteini \u00fcretme yetene\u011fini geri kazand\u0131rmakt\u0131r. Bu protein, tam uzunluktaki distrofinin 80%&#039;sini korur ve g\u00fcn\u00fcm\u00fczde onaylanm\u0131\u015f veya geli\u015ftirilmekte olan sentetik mikro-distrofin yap\u0131lar\u0131ndan (tam uzunluktaki distrofinin yakla\u015f\u0131k 34%&#039;si kadard\u0131r) \u00f6nemli \u00f6l\u00e7\u00fcde daha b\u00fcy\u00fckt\u00fcr.<\/strong><\/p>\n\n\n\n<h2 class=\"wp-block-heading\">\u0130lk klinik ara\u015ft\u0131rma sahas\u0131n\u0131n 2026 y\u0131l\u0131n\u0131n ilk yar\u0131s\u0131nda faaliyete ge\u00e7irilmesi hedefleniyor.<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u201cFDA taraf\u0131ndan PBGENE-DMD i\u00e7in al\u0131nan \u201d\u00c7al\u0131\u015fmaya Devam Edilebilir\u2019 bildirimi, tamamen bize ait ikinci program\u0131m\u0131z\u0131 klinik a\u015famaya do\u011fru ilerletirken Precision BioSciences i\u00e7in bir ba\u015fka d\u00fczenleyici ba\u015far\u0131y\u0131 temsil ediyor. G\u00fcn\u00fcm\u00fczde onaylanm\u0131\u015f tedavilere ra\u011fmen, DMD&#039;li erkek \u00e7ocuklarda zaman i\u00e7inde fonksiyonel iyile\u015fmelere yol a\u00e7an tedaviler eksik. DMD i\u00e7in bu yeni gen eksizyon yakla\u015f\u0131m\u0131n\u0131 klinik a\u015famaya getirmekten ve 2026 y\u0131l\u0131n\u0131n ilk yar\u0131s\u0131nda ABD&#039;deki ilk klinik merkezi aktif hale getirmekten heyecan duyuyoruz. Y\u0131l sonuna kadar IND ba\u015fvurusunu yapma ve ilk \u00e7eyrekte FDA \u2018\u00c7al\u0131\u015fmaya Devam Edilebilir\u2019 bildirimini alma hedefimize ula\u015fan ekibimizle gurur duyuyorum. \u015eirket, klinik merkez aktivasyonlar\u0131n\u0131 ba\u015flatmak i\u00e7in \u00e7e\u015fitli Etik Kurullar ve FDA ile \u00e7al\u0131\u015fmaya devam edecektir,\u201d dedi Precision BioSciences CEO&#039;su Michael Amoroso.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Bu Sayfay\u0131 Takip Edin<\/strong>&nbsp;&gt;&gt;&gt;&nbsp;<a href=\"https:\/\/dmdwarrior.com\/tr\/dmd-therapies\/\" target=\"_blank\" rel=\"noreferrer noopener\">Duchenne i\u00e7in T\u00fcm Klinik Ara\u015ft\u0131rmalar<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>Precision BioSciences announced that the Company received a Study May Proceed notification from the U.S. Food and Drug Administration (FDA). This allows Precision BioSciences to initiate IRB activities and clinical trial site activation for the FUNCTION-DMD Phase 1\/2 clinical trial for PBGENE-DMD for the treatment of ambulatory Duchenne muscular dystrophy (DMD) patients at highly specialized [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":6205,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[359,479,72,314,313],"class_list":["post-6203","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-full-length-dystrophin","tag-function-dmd","tag-gene-editing","tag-pbgene-dmd","tag-precision-biosciences"],"subtitle":"ABD G\u0131da ve \u0130la\u00e7 \u0130daresi (FDA), PBGENE-DMD \u00fcr\u00fcn\u00fcn\u00fcn Faz 1\/2 klinik denemesi (FUNCTION-DMD) i\u00e7in Precision BioSciences \"yer aktivasyonu\" onay\u0131n\u0131 vermi\u015ftir.","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/6203","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=6203"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/6203\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/6205"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=6203"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=6203"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=6203"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}