{"id":8916,"date":"2026-11-05T16:37:41","date_gmt":"2026-11-05T13:37:41","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=8916"},"modified":"2026-11-05T16:40:46","modified_gmt":"2026-11-05T13:40:46","slug":"pbgene-dmd-no-serious-adverse-events-week-8","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/pbgene-dmd-no-serious-adverse-events-week-8\/","title":{"rendered":"Precision BioSciences, PBGENE-DMD i\u00e7in Olumlu G\u00fcvenlik De\u011ferlendirmesi Bildirdi: Sekizinci Haftaya Kadar Ciddi Yan Etki G\u00f6zlemlenmedi"},"content":{"rendered":"<p class=\"wp-block-paragraph\"><strong>Precision BioSciences, Duchenne kas distrofisi (DMD) i\u00e7in geli\u015ftirdi\u011fi deneysel gen d\u00fczenleme tedavisi PBGENE-DMD'nin olumlu bir g\u00fcvenlik de\u011ferlendirmesi ald\u0131\u011f\u0131n\u0131 duyurdu.<\/strong> Ba\u011f\u0131ms\u0131z bir Veri G\u00fcvenli\u011fi \u0130zleme Kurulu (DSMB), Faz 1\/2 FUNCTION-DMD klinik \u00e7al\u0131\u015fmas\u0131nda tedavi edilen ilk hastadan elde edilen g\u00fcvenlik verilerini inceledi ve tedaviyi takip eden ilk sekiz hafta boyunca ciddi yan etki (SAE) veya \u00f6zel ilgi alan\u0131 olu\u015fturan yan etki (AESI) bildirilmedi\u011fini a\u00e7\u0131klad\u0131.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Hastan\u0131n sekiz haftal\u0131k ilk g\u00fcvenlik g\u00f6zlem d\u00f6neminin tamamlanmas\u0131n\u0131n ard\u0131ndan, DSMB (Veri G\u00fcvenli\u011fi \u0130zleme Kurulu) herhangi bir g\u00fcvenlik endi\u015fesi tespit etmedi ve \u00e7al\u0131\u015fman\u0131n 1. B\u00f6l\u00fcm\u00fcndeki ek hastalar\u0131n tedavisine devam edilmesini onaylad\u0131. Bu karar, devam eden klinik \u00e7al\u0131\u015fma i\u00e7in \u00f6nemli bir d\u00f6n\u00fcm noktas\u0131d\u0131r ve hasta kayd\u0131n\u0131n devam\u0131n\u0131 desteklemektedir. Daha Fazla Bilgi Edinin: <a href=\"https:\/\/dmdwarrior.com\/tr\/faq-pbgene-dmd-gene-editing-therapy\/\" target=\"_blank\" rel=\"noreferrer noopener\">PBGENE-DMD nedir?<\/a><\/p>\n\n\n\n<h3 class=\"wp-block-heading\">PBGENE-DMD : Duchenne Kas Distrofisi \u0130\u00e7in Gen D\u00fczenleme Yakla\u015f\u0131m\u0131<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">PBGENE-DMD, v\u00fccudun kendi distrofin genini kullanarak DMD'nin alt\u0131nda yatan genetik nedeni ele almak \u00fczere tasarlanm\u0131\u015ft\u0131r. Tedavi, distrofin geninden 45-55. eksonlar\u0131 \u00e7\u0131karmak i\u00e7in tek bir adeno-ili\u015fkili vir\u00fcs (AAV) arac\u0131l\u0131\u011f\u0131yla iletilen iki tamamlay\u0131c\u0131 ARCUS n\u00fckleaz\u0131 kullan\u0131r. Ama\u00e7, sentetik, k\u0131salt\u0131lm\u0131\u015f mikrodistrofin yerine, neredeyse tam uzunlukta, i\u015flevsel bir distrofin proteininin \u00fcretimini geri kazand\u0131rmakt\u0131r.<\/p>\n\n\n\n<h3 class=\"wp-block-heading\">FUNCTION-DMD Klinik Deneyi Hakk\u0131nda<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">Faz 1\/2 FUNCTION-DMD \u00e7al\u0131\u015fmas\u0131, 45-55. eksonlar\u0131 i\u00e7eren mutasyonlara sahip, 2 ila 7 ya\u015f aras\u0131 ayakta tedavi g\u00f6ren DMD'li erkek \u00e7ocuklarda PBGENE-DMD'nin g\u00fcvenli\u011fini, tolerans\u0131n\u0131 ve etkinli\u011fini de\u011ferlendirmektedir. \u00c7al\u0131\u015fma, kat\u0131l\u0131mc\u0131larda distrofin protein ekspresyonunu ve fonksiyonel sonu\u00e7lar\u0131 de\u011ferlendirecektir. <strong>Daha fazla bilgi edin:<\/strong> <a href=\"https:\/\/dmdwarrior.com\/clinical-trials\/pbgene-dmd-phase-1-2a-clinical-trial-for-duchenne-function-dmd\/\" target=\"_blank\" rel=\"noreferrer noopener\">PBGENE-DMD Faz 1\/2a Klinik \u00c7al\u0131\u015fmas\u0131<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Precision BioSciences, ilk sekiz haftal\u0131k izleme d\u00f6neminin, AAV tabanl\u0131 tedavilerle ili\u015fkili potansiyel erken g\u00fcvenlik olaylar\u0131n\u0131 belirlemek i\u00e7in \u00f6zellikle \u00f6nemli oldu\u011funu belirtti. \u015eirket, \u00e7al\u0131\u015fma ilerledik\u00e7e g\u00fcvenlik izlemesine devam edecektir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u015eirket , FUNCTION-DMD \u00e7al\u0131\u015fmas\u0131ndan elde edilen g\u00fcvenlik ve etkinlik g\u00fcncellemelerini 2027 y\u0131l\u0131 boyunca payla\u015fmay\u0131 bekliyor.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Son duyuru, cesaret verici bir ilk g\u00fcvenlik sinyali sunuyor: \u0130lk hastada sekizinci haftaya kadar ciddi yan etki veya \u00f6zel ilgi gerektiren yan etki bildirilmedi. Bununla birlikte, bu erken bulgular tedavinin genel g\u00fcvenli\u011fini veya etkinli\u011fini kan\u0131tlamaz.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Daha Fazlas\u0131n\u0131 Ke\u015ffedin<\/strong>:&nbsp;<a href=\"https:\/\/dmdwarrior.com\/clinical-trials\/map\/\" target=\"_blank\" rel=\"noopener\">Duchenne Klinik Ara\u015ft\u0131rma Yerleri Haritas\u0131<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>Precision BioSciences has announced a positive safety review of PBGENE-DMD, its investigational gene-editing treatment for Duchenne muscular dystrophy (DMD). An independent Data Safety Monitoring Board (DSMB) reviewed safety data from the first patient treated in the Phase 1\/2 FUNCTION-DMD clinical trial and reported no serious adverse events (SAEs) or adverse events of special interest (AESIs) [&hellip;]<\/p>\n","protected":false},"author":4,"featured_media":8918,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[479,314,724,313],"class_list":["post-8916","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-function-dmd","tag-pbgene-dmd","tag-pbgene-dmd-therapy","tag-precision-biosciences"],"subtitle":"Precision BioSciences, FUNCTION-DMD \u00e7al\u0131\u015fmas\u0131nda PBGENE-DMD i\u00e7in cesaret verici bir g\u00fcvenlik d\u00f6n\u00fcm noktas\u0131na ula\u015f\u0131ld\u0131\u011f\u0131n\u0131 bildiriyor. \u0130lk hastada 8. haftaya kadar ciddi yan etki veya \u00f6zel ilgi gerektiren herhangi bir olay g\u00f6r\u00fclmedi; bu da \u00e7al\u0131\u015fman\u0131n ilerleyi\u015fini destekliyor.","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/8916","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\/4"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/comments?post=8916"}],"version-history":[{"count":2,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/8916\/revisions"}],"predecessor-version":[{"id":8920,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/8916\/revisions\/8920"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/8918"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=8916"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=8916"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=8916"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}