{"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\/de\/spot-mrna03-gene-therapy-early-phase-1-clinical-trial-nct07188012\/","title":{"rendered":"SPOT-mRNA03-Gentherapie f\u00fcr Duchenne-Muskeldystrophie beginnt mit der fr\u00fchen klinischen Phase-1-Studie"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Bei der fr\u00fchen Phase-1-Studie (NCT07188012) zur SPOT-mRNA03-Gentherapie f\u00fcr die Duchenne-Muskeldystrophie (DMD), die von Shanghai Siponuoyin Biotechnology entwickelt wurde, handelt es sich um eine Pilotstudie zur Sicherheit und Expression von Dystrophin in der Skelettmuskulatur nach der Verabreichung.<\/p>\n\n\n\n<div class=\"wp-block-rank-math-toc-block\" id=\"rank-math-toc\"><h2>Inhaltsverzeichnis<\/h2><nav><ul><li><a href=\"#what-is-spot-m-rna-03-gene-therapy\">Was ist die SPOT-mRNA03-Gentherapie?<\/a><\/li><li><a href=\"#spot-m-rna-03-gene-therapy-early-phase-1-nct-07188012\">SPOT-mRNA03 Gentherapie, fr\u00fche Phase 1 \u2013 NCT07188012<\/a><\/li><li><a href=\"#how-to-participate-in-the-sgt-003-clinical-trial\">So nehmen Sie an der klinischen Studie SPOT-mRNA03 teil<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"what-is-spot-m-rna-03-gene-therapy\"><strong>Was ist die SPOT-mRNA03-Gentherapie?<\/strong><\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>SPOT-mRNA03 ist das Gentherapieprodukt f\u00fcr die Muskeldystrophie Duchenne (DMD).<\/strong> Nach der Verabreichung an DMD-Patienten kann das molekulare Zielpeptid auf der Membran der EVs SPOT-mRNA03 in die Muskelzellen des Patienten leiten und so die Translation und Sekretion von endogenem, intaktem Dystrophin in voller L\u00e4nge durch Dystrophin-defiziente Rezeptorzellen unter Verwendung intrazellul\u00e4rer Ribosomen und Aminos\u00e4uren erm\u00f6glichen.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"spot-m-rna-03-gene-therapy-early-phase-1-nct-07188012\"><strong>SPOT-mRNA03 Gentherapie, fr\u00fche Phase 1 \u2013 NCT07188012<\/strong><\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Es handelt sich um eine einarmige, offene, FIH- und monozentrische explorative klinische Studie mit intraven\u00f6s verabreichtem SPOT-mRNA03 an Patienten mit DMD. Die vollst\u00e4ndige Dystrophin-mRNA ist in muskelgerichteten extrazellul\u00e4ren Vesikeln (EVs) namens SPOT-mRNA03 enthalten. Molekulare Targeting-Peptide auf der EV-Membran verleihen SPOT-mRNA03 seine Targeting-F\u00e4higkeit, die die Verteilung von Dystrophin-mRNA als Gentherapieprodukt f\u00fcr DMD erm\u00f6glicht.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Studienbeginn (voraussichtlich)<\/strong>: 2025-08-06<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Prim\u00e4rer Abschluss (gesch\u00e4tzt)<\/strong>: 2026-12<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Studienabschluss (gesch\u00e4tzt)<\/strong>: 2026-12<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Anzahl der Teilnehmer (gesch\u00e4tzt)<\/strong>: 6<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Studientyp<\/strong>: Fr\u00fche Phase 1<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Standort<\/strong>: <a href=\"https:\/\/maps.app.goo.gl\/Z2CyEqLydJ4Kxh9aA\" target=\"_blank\" rel=\"noreferrer noopener\">Shanghai Kindermedizinisches Zentrum<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Studienberechtigte Altersgruppen<\/strong>: 2 Jahre bis 6 Jahre (gehf\u00e4hige M\u00e4nner)<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Wichtigste Ein- und Ausschlusskriterien. \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>So nehmen Sie an der klinischen Studie SPOT-mRNA03 teil<\/strong><\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Um an der klinischen Studie der fr\u00fchen Phase 1 zur SPOT-mRNA03-Gentherapie teilzunehmen, k\u00f6nnen Sie die unten stehenden Kontaktinformationen verwenden.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Name<\/strong>: Winston Town<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Telefonnummer<\/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\/de\/wp-json\/wp\/v2\/posts\/4576","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/dmdwarrior.com\/de\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/dmdwarrior.com\/de\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/de\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/de\/wp-json\/wp\/v2\/comments?post=4576"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/de\/wp-json\/wp\/v2\/posts\/4576\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/de\/wp-json\/wp\/v2\/media\/4580"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/de\/wp-json\/wp\/v2\/media?parent=4576"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/de\/wp-json\/wp\/v2\/categories?post=4576"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/de\/wp-json\/wp\/v2\/tags?post=4576"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}