{"id":4428,"date":"2025-09-07T13:07:26","date_gmt":"2025-09-07T10:07:26","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=4428"},"modified":"2025-11-03T20:20:56","modified_gmt":"2025-11-03T17:20:56","slug":"gnr-097-duchenne-gene-therapy-phase-1-2-study-started-by-generium","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/fr\/gnr-097-duchenne-gene-therapy-phase-1-2-study-started-by-generium\/","title":{"rendered":"GNR-097\u00a0: \u00c9tude de phase\u00a01\/2 sur la th\u00e9rapie g\u00e9nique de la maladie de Duchenne lanc\u00e9e par Generium"},"content":{"rendered":"<p class=\"wp-block-paragraph\"><strong>Generium est une soci\u00e9t\u00e9 de biotechnologie innovante russe qui a re\u00e7u l&#039;autorisation de mener un essai clinique du m\u00e9dicament de th\u00e9rapie g\u00e9nique GNR-097 pour le traitement de la dystrophie musculaire progressive de Duchenne (DMD).<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Cet essai multicentrique, randomis\u00e9, en simple aveugle et contr\u00f4l\u00e9 par placebo consiste en une administration intraveineuse unique du m\u00e9dicament de th\u00e9rapie g\u00e9nique \u00e0 des patients p\u00e9diatriques atteints de dystrophie musculaire de Duchenne progressive. Cette \u00e9tude de phase 1\/2 vise \u00e0 \u00e9valuer la tol\u00e9rance, l&#039;innocuit\u00e9 et l&#039;efficacit\u00e9 du m\u00e9dicament GNR-097.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Th\u00e9rapie g\u00e9nique de la maladie de Duchenne GNR-097<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>GNR-097 est un vecteur viral ad\u00e9no-associ\u00e9 recombinant du s\u00e9rotype 9, codant pour un g\u00e8ne de dystrophine tronqu\u00e9.<\/strong><\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Daniil Talyansky, PDG de GENERIUM\u00a0:<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">La th\u00e9rapie g\u00e9nique est le domaine le plus r\u00e9cent et le plus innovant de la pharmacie et de la m\u00e9decine mondiales. L&#039;expertise unique des scientifiques russes a permis le d\u00e9veloppement d&#039;un vecteur viral ad\u00e9no-associ\u00e9 recombinant codant pour le g\u00e8ne de la microdystrophine. Nous lan\u00e7ons actuellement les essais cliniques de ce m\u00e9dicament original, tr\u00e8s prometteur.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Essai clinique de la th\u00e9rapie g\u00e9nique GNR-097<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">L&#039;essai clinique de ce m\u00e9dicament innovant est international et sera men\u00e9 en F\u00e9d\u00e9ration de Russie et en R\u00e9publique de Bi\u00e9lorussie. En Russie, l&#039;\u00e9tude se d\u00e9roulera dans cinq centres m\u00e9dicaux\u00a0: l&#039;Institut clinique de recherche en p\u00e9diatrie et chirurgie p\u00e9diatrique du nom de l&#039;acad\u00e9micien Yu.E. Veltishchev, l&#039;Universit\u00e9 nationale russe de recherche m\u00e9dicale Pirogov, l&#039;Universit\u00e9 d&#039;\u00c9tat de p\u00e9diatrie de Saint-P\u00e9tersbourg, l&#039;H\u00f4pital clinique r\u00e9gional pour enfants d&#039;Ekaterinbourg et le Centre national de recherche m\u00e9dicale pour la sant\u00e9 infantile. En Bi\u00e9lorussie, l&#039;essai sera men\u00e9 au Centre scientifique et pratique r\u00e9publicain \u00ab\u00a0M\u00e8re et enfant\u00a0\u00bb.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>En savoir plus<\/strong>:\u00a0<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>Generium is a Russian innovative biotechnology company has received approval to conduct a clinical trial of GNR-097 gene therapy drug for the treatment of progressive Duchenne muscular dystrophy (DMD). The multicenter, single-blind, randomized placebo-controlled trial involves a single intravenous administration of the gene therapy drug to pediatric patients with progressive Duchenne muscular dystrophy. This Phase [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":4430,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[27],"tags":[137,415,61,241,414,135],"class_list":["post-4428","post","type-post","status-publish","format-standard","has-post-thumbnail","category-research","tag-clinical-trial","tag-daniil-talyansky","tag-gene-therapy","tag-generium","tag-gnr-097","tag-russia"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/posts\/4428","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=4428"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/posts\/4428\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/media\/4430"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/media?parent=4428"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/categories?post=4428"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/tags?post=4428"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}