{"id":6289,"date":"2026-04-29T17:14:16","date_gmt":"2026-04-29T14:14:16","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=6289"},"modified":"2026-04-29T17:16:44","modified_gmt":"2026-04-29T14:16:44","slug":"precision-biosciences-pbgene-dmd-phase-1-2a-function-dmd-clinical-trial-nct07429240","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/fr\/precision-biosciences-pbgene-dmd-phase-1-2a-function-dmd-clinical-trial-nct07429240\/","title":{"rendered":"Precision BioSciences a annonc\u00e9 le d\u00e9but de l&#039;\u00e9tude de phase 1\/2a (FUNCTION-DMD) du PBGENE-DMD chez des patients ambulatoires atteints de dystrophie musculaire de Duchenne."},"content":{"rendered":"<p class=\"wp-block-paragraph\"><strong>Precision BioSciences a annonc\u00e9 le lancement d&#039;un essai de phase 1\/2a du PBGENE-DMD (NCT07429240) chez des patients ambulatoires atteints de Duchenne \u00e2g\u00e9s de 2 \u00e0 7 ans.<\/strong> Dans cet essai, une dose unique de FUNCTION-DMD sera \u00e9tudi\u00e9e sur une p\u00e9riode de 130 semaines. <strong>En savoir plus:<\/strong> <a href=\"https:\/\/dmdwarrior.com\/fr\/precision-biosciences-pbgene-dmd-function-dmd-phase-1-2-clinical-study\/\" target=\"_blank\" rel=\"noreferrer noopener\">Precision BioSciences obtient l&#039;autorisation am\u00e9ricaine pour sa demande d&#039;autorisation de mise sur le march\u00e9 d&#039;un nouveau m\u00e9dicament exp\u00e9rimental (FDA).<\/a><\/p>\n\n\n\n<div class=\"wp-block-rank-math-toc-block\" id=\"rank-math-toc\"><h2>Table des mati\u00e8res<\/h2><nav><ul><li><a href=\"#what-is-pbgene-dmd-phase-1-2-a-clinical-trial\">Qu\u2019est-ce que l\u2019essai clinique de phase 1\/2a PBGENE-DMD\u00a0?<\/a><\/li><li><a href=\"#pbgene-dmd-phase-1-2-a-nct-07429240\">PBGENE-DMD Phase 1\/\u200b2a \u2013 NCT07429240<\/a><\/li><li><a href=\"#how-to-participate-in-the-pbgene-dmd-phase-1-2-a-clinical-trial\">Comment participer \u00e0 l\u2019essai clinique de phase 1\/2a PBGENE-DMD<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Cette \u00e9tude de phase 1\/2a est con\u00e7ue pour \u00e9valuer la s\u00e9curit\u00e9, la tol\u00e9rance et les premiers signes d&#039;efficacit\u00e9 du PBGENE-DMD chez les personnes atteintes de DMD qui pr\u00e9sentent des mutations adapt\u00e9es \u00e0 l&#039;excision des exons 45 \u00e0 55.<\/strong> Compte tenu des limites des options de traitement actuelles, PBGENE-DMD offre une nouvelle strat\u00e9gie prometteuse visant \u00e0 apporter une correction potentiellement unique et durable du d\u00e9faut g\u00e9n\u00e9tique sous-jacent dans le plus grand sous-groupe mol\u00e9culaire de personnes atteintes de DMD.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Jusqu&#039;\u00e0 18 participants pourront \u00eatre inclus dans cet essai. Dur\u00e9e totale de participation \u00e0 l&#039;essai pour chaque participant\u00a0: environ 130 semaines.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"what-is-pbgene-dmd-phase-1-2-a-clinical-trial\">Qu\u2019est-ce que l\u2019essai clinique de phase 1\/2a PBGENE-DMD\u00a0?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Il s&#039;agit d&#039;un essai clinique de phase pr\u00e9coce \u00e9valuant un nouveau traitement, le PBGENE-DMD, chez des gar\u00e7ons et des hommes atteints de dystrophie musculaire de Duchenne (DMD) pr\u00e9sentant des anomalies g\u00e9n\u00e9tiques sp\u00e9cifiques susceptibles de r\u00e9pondre \u00e0 cette th\u00e9rapie. Les participants recevront une dose unique du traitement par voie intraveineuse (dans une veine du bras). Les m\u00e9decins surveilleront \u00e9troitement la s\u00e9curit\u00e9, les effets secondaires et les premiers signes d&#039;efficacit\u00e9 du traitement.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Afin de r\u00e9duire les \u00e9ventuelles r\u00e9actions immunitaires, les participants recevront \u00e9galement des m\u00e9dicaments \u00e0 court terme au moment du traitement.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">L&#039;\u00e9tude comporte deux parties. Dans la premi\u00e8re, les chercheurs d\u00e9termineront une dose s\u00fbre et bien tol\u00e9r\u00e9e. Si cette dose est confirm\u00e9e, la deuxi\u00e8me partie inclura des participants suppl\u00e9mentaires afin d&#039;\u00e9valuer plus en d\u00e9tail le traitement.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Jusqu&#039;\u00e0 18 participants prendront part \u00e0 l&#039;\u00e9tude. Chaque personne sera suivie pendant environ 2 ans et demi (environ 130 semaines).<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Apprendre encore plus: <a href=\"https:\/\/dmdwarrior.com\/fr\/faq-pbgene-dmd-gene-editing-therapy\/\" target=\"_blank\" rel=\"noreferrer noopener\">Th\u00e9rapie par \u00e9dition g\u00e9nique PBGENE-DMD\u00a0: avantages, admissibilit\u00e9 et questions fr\u00e9quentes<\/a><\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"pbgene-dmd-phase-1-2-a-nct-07429240\">PBGENE-DMD Phase 1\/\u200b2a \u2013 NCT07429240<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u00c9tude de phase 1\/2a, multicentrique, ouverte, visant \u00e0 \u00e9valuer la s\u00e9curit\u00e9, la tol\u00e9rance et l&#039;efficacit\u00e9 pr\u00e9liminaire du PBGENE-DMD chez les participants atteints de dystrophie musculaire de Duchenne (FUNCTION-DMD)<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>D\u00e9but des \u00e9tudes (estim\u00e9)\u00a0:<\/strong> 2026-03<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Ach\u00e8vement du primaire (estim\u00e9) :<\/strong> 2029-11<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Ach\u00e8vement des \u00e9tudes (estimation)\u00a0:<\/strong> 2029-12<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Nombre de participants (estim\u00e9) :<\/strong> 18<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Type d&#039;\u00e9tude :<\/strong> interventionnel<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Emplacement:<\/strong> H\u00f4pital pour enfants de l&#039;Arkansas<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u00c2ges admissibles \u00e0 l&#039;\u00e9tude\u00a0:<\/strong> De 2 \u00e0 7 ans (enfant)<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Principaux crit\u00e8res d\u2019inclusion et d\u2019exclusion. \u2013 <a href=\"https:\/\/clinicaltrials.gov\/study\/NCT07429240\" target=\"_blank\" rel=\"noreferrer noopener\">NCT07429240<\/a><\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"how-to-participate-in-the-pbgene-dmd-phase-1-2-a-clinical-trial\">Comment participer \u00e0 l\u2019essai clinique de phase 1\/2a PBGENE-DMD<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Pour participer \u00e0 l&#039;essai clinique de phase 1\/2a PBGENE-DMD, vous pouvez utiliser les informations de contact ci-dessous.&nbsp;<strong>Apprendre encore plus<\/strong>:&nbsp;<a href=\"https:\/\/dmdwarrior.com\/fr\/how-to-participate-in-clinical-trials-for-duchenne\/\" target=\"_blank\" rel=\"noreferrer noopener\">Comment participer aux essais cliniques pour la maladie de Duchenne (DMD) ?<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Nom:<\/strong> Eileen Bristow<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Num\u00e9ro de t\u00e9l\u00e9phone:<\/strong> +1-610-462-1004<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>E-mail:<\/strong> function-DMD@precisionbiosciences.com<\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>En savoir plus<\/strong>:&nbsp;<a href=\"https:\/\/dmdwarrior.com\/fr\/dmd-therapies\/\" target=\"_blank\" rel=\"noreferrer noopener\">Essais cliniques sur la dystrophie musculaire de Duchenne (Liste de toutes les recherches)<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>Precision BioSciences announced the commencement of a Phase 1\/\u200b2a trial of PBGENE-DMD (NCT07429240) in ambulatory patients with Duchenne aged 2 to 7 years. In this trial, single dose of FUNCTION-DMD will be studied over 130 weeks. Read More: Precision BioSciences Receives U.S. FDA Clearance of Investigational New Drug Application This Phase 1\/2a study is designed [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":6291,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[27],"tags":[137,242,359,479,72,493,314,313],"class_list":["post-6289","post","type-post","status-publish","format-standard","has-post-thumbnail","category-research","tag-clinical-trial","tag-clinical-trials","tag-full-length-dystrophin","tag-function-dmd","tag-gene-editing","tag-nct07429240","tag-pbgene-dmd","tag-precision-biosciences"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/posts\/6289","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\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/comments?post=6289"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/posts\/6289\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/media\/6291"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/media?parent=6289"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/categories?post=6289"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/tags?post=6289"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}