{"id":4576,"date":"2025-09-25T16:23:32","date_gmt":"2025-09-25T13:23:32","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=4576"},"modified":"2025-09-25T16:23:33","modified_gmt":"2025-09-25T13:23:33","slug":"spot-mrna03-gene-therapy-early-phase-1-clinical-trial-nct07188012","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/fr\/spot-mrna03-gene-therapy-early-phase-1-clinical-trial-nct07188012\/","title":{"rendered":"La th\u00e9rapie g\u00e9nique SPOT-mRNA03 pour la dystrophie musculaire de Duchenne d\u00e9bute un essai clinique de phase 1 pr\u00e9coce"},"content":{"rendered":"<p class=\"wp-block-paragraph\">L&#039;\u00e9tude de phase 1 pr\u00e9coce (NCT07188012) de la th\u00e9rapie g\u00e9nique SPOT-mRNA03 pour la dystrophie musculaire de Duchenne (DMD) d\u00e9velopp\u00e9e par Shanghai Siponuoyin Biotechnology est une \u00e9tude pilote sur la s\u00e9curit\u00e9 et l&#039;expression de la dystrophine dans les muscles squelettiques apr\u00e8s administration.<\/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-spot-m-rna-03-gene-therapy\">Qu&#039;est-ce que la th\u00e9rapie g\u00e9nique SPOT-mRNA03<\/a><\/li><li><a href=\"#spot-m-rna-03-gene-therapy-early-phase-1-nct-07188012\">Th\u00e9rapie g\u00e9nique SPOT-mRNA03 Phase 1 pr\u00e9coce \u2013 NCT07188012<\/a><\/li><li><a href=\"#how-to-participate-in-the-sgt-003-clinical-trial\">Comment participer \u00e0 l&#039;essai clinique SPOT-mRNA03<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"what-is-spot-m-rna-03-gene-therapy\"><strong>Qu&#039;est-ce que la th\u00e9rapie g\u00e9nique SPOT-mRNA03<\/strong><\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>SPOT-mRNA03 est le produit de th\u00e9rapie g\u00e9nique pour la dystrophie musculaire de Duchenne (DMD).<\/strong> Apr\u00e8s administration aux patients atteints de DMD, le peptide de ciblage mol\u00e9culaire sur la membrane des EV peut diriger SPOT-mRNA03 dans les cellules musculaires du patient, permettant la traduction et la s\u00e9cr\u00e9tion de dystrophine intacte pleine longueur endog\u00e8ne par des cellules r\u00e9ceptrices d\u00e9ficientes en dystrophine utilisant des ribosomes intracellulaires et des acides amin\u00e9s.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"spot-m-rna-03-gene-therapy-early-phase-1-nct-07188012\"><strong>Th\u00e9rapie g\u00e9nique SPOT-mRNA03 Phase 1 pr\u00e9coce \u2013 NCT07188012<\/strong><\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Il s&#039;agit d&#039;une \u00e9tude clinique exploratoire monocentrique, ouverte et \u00e0 bras unique, utilisant le g\u00e8ne SPOT-mRNA03 administr\u00e9 par voie intraveineuse \u00e0 des patients atteints de DMD. L&#039;ARNm complet de la dystrophine est contenu dans des v\u00e9sicules extracellulaires (VE) musculaires cibl\u00e9es appel\u00e9es SPOT-mRNA03. Des peptides de ciblage mol\u00e9culaire pr\u00e9sents sur la membrane des VE conf\u00e8rent \u00e0 SPOT-mRNA03 sa capacit\u00e9 de ciblage, permettant ainsi la distribution de l&#039;ARNm de la dystrophine comme produit de th\u00e9rapie g\u00e9nique pour la DMD.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>D\u00e9but de l&#039;\u00e9tude (estim\u00e9)<\/strong>: 2025-08-06<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Ach\u00e8vement du primaire (estim\u00e9)<\/strong>: 2026-12<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Ach\u00e8vement de l&#039;\u00e9tude (estim\u00e9)<\/strong>: 2026-12<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Nombre de participants (estim\u00e9)<\/strong>: 6<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Type d&#039;\u00e9tude<\/strong>:Phase 1 pr\u00e9coce<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Emplacement<\/strong>: <a href=\"https:\/\/maps.app.goo.gl\/Z2CyEqLydJ4Kxh9aA\" target=\"_blank\" rel=\"noreferrer noopener\">Centre m\u00e9dical pour enfants de Shanghai<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u00c2ges admissibles aux \u00e9tudes<\/strong>: 2 ans \u00e0 6 ans (hommes ambulants)<\/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\/NCT07188012\" target=\"_blank\" rel=\"noreferrer noopener\">NCT07188012<\/a> \u2013<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"how-to-participate-in-the-sgt-003-clinical-trial\"><strong>Comment participer \u00e0 l&#039;essai clinique SPOT-mRNA03<\/strong><\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Pour participer \u00e0 l&#039;essai clinique de phase 1 pr\u00e9coce de th\u00e9rapie g\u00e9nique SPOT-mRNA03, vous pouvez utiliser les coordonn\u00e9es ci-dessous.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Nom<\/strong>: Winston Town<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Num\u00e9ro de t\u00e9l\u00e9phone<\/strong>: 212-920-5501<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>E-mail<\/strong>: Winston.town@spotbiosystems.com<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><\/p>","protected":false},"excerpt":{"rendered":"<p>The early phase 1 study (NCT07188012) of SPOT-mRNA03 gene therapy for Duchenne Muscular Dystrophy (DMD) developed by Shanghai Siponuoyin Biotechnology is a pilot study of the safety and expression of dystrophin in skeletal muscles after administration. What is SPOT-mRNA03 Gene Therapy SPOT-mRNA03 is the gene therapy product for Duchenne Muscular Dystrophy (DMD). Following administration to [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":4580,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[27],"tags":[123,137,242,359,61,424,423,425],"class_list":["post-4576","post","type-post","status-publish","format-standard","has-post-thumbnail","category-research","tag-china","tag-clinical-trial","tag-clinical-trials","tag-full-length-dystrophin","tag-gene-therapy","tag-shanghai-siponuoyin","tag-spot-mrna03","tag-spot-mrna03-gene-therapy"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/posts\/4576","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=4576"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/posts\/4576\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/media\/4580"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/media?parent=4576"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/categories?post=4576"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/tags?post=4576"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}