{"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\/fr\/pbgene-dmd-no-serious-adverse-events-week-8\/","title":{"rendered":"Precision BioSciences annonce un bilan de s\u00e9curit\u00e9 positif pour PBGENE-DMD\u00a0: aucun \u00e9v\u00e9nement ind\u00e9sirable grave n\u2019a \u00e9t\u00e9 observ\u00e9 jusqu\u2019\u00e0 la huiti\u00e8me semaine."},"content":{"rendered":"

Precision BioSciences has announced a positive safety review of PBGENE-DMD, its investigational gene-editing treatment for Duchenne muscular dystrophy (DMD).<\/strong> 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) through the first eight weeks following treatment.<\/p>\n\n\n\n

Following the completion of the patient’s initial eight-week safety observation period, the DSMB identified no safety concerns and endorsed proceeding with the treatment of additional patients in Part 1 of the study. This decision represents an important milestone for the ongoing clinical trial and supports the continuation of patient enrollment. Learn More: Qu'est-ce que PBGENE-DMD ?<\/a><\/p>\n\n\n\n

PBGENE-DMD: A Gene-Editing Approach for Duchenne<\/h3>\n\n\n\n

PBGENE-DMD is designed to address the underlying genetic cause of DMD by using the body’s own dystrophin gene. The treatment employs two complementary ARCUS nucleases, delivered through a single adeno-associated virus (AAV), to remove exons 45\u201355 from the dystrophin gene. The goal is to restore production of a near full-length, functional dystrophin protein rather than relying on synthetic, shortened microdystrophin.<\/p>\n\n\n\n

About the FUNCTION-DMD Clinical Trial<\/h3>\n\n\n\n

The Phase 1\/2 FUNCTION-DMD study is evaluating the safety, tolerability, and efficacy of PBGENE-DMD in ambulatory boys with DMD between 2 and 7 years of age who have mutations involving exons 45\u201355. The study will assess dystrophin protein expression and functional outcomes in participants. Apprendre encore plus:<\/strong> Essai clinique de phase 1\/2a PBGENE-DMD<\/a><\/p>\n\n\n\n

Precision BioSciences stated that the initial eight-week monitoring period was particularly important for identifying potential early safety events associated with AAV-based therapies. The company will continue safety monitoring as the study progresses.<\/p>\n\n\n\n

The company expects to provide further safety and efficacy updates from the FUNCTION-DMD study throughout 2027.<\/p>\n\n\n\n

The latest announcement provides an encouraging initial safety signal: no serious adverse events or adverse events of special interest were reported in the first patient through week eight. However, these early findings do not establish the treatment’s overall safety or efficacy.<\/p>\n\n\n\n

D\u00e9couvrez-en plus<\/strong>: Carte des sites d'essais cliniques sur la dystrophie musculaire de Duchenne<\/a><\/p>","protected":false},"excerpt":{"rendered":"

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) […]<\/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 annonce une \u00e9tape encourageante en mati\u00e8re de s\u00e9curit\u00e9 pour le PBGENE-DMD dans l'essai FUNCTION-DMD. Le premier patient n'a pr\u00e9sent\u00e9 aucun \u00e9v\u00e9nement ind\u00e9sirable grave ni \u00e9v\u00e9nement d'int\u00e9r\u00eat particulier jusqu'\u00e0 la semaine 8, ce qui confirme la progression de l'\u00e9tude.","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/posts\/8916","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/users\/4"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/comments?post=8916"}],"version-history":[{"count":2,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/posts\/8916\/revisions"}],"predecessor-version":[{"id":8920,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/posts\/8916\/revisions\/8920"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/media\/8918"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/media?parent=8916"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/categories?post=8916"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/tags?post=8916"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}