{"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\/pt\/spot-mrna03-gene-therapy-early-phase-1-clinical-trial-nct07188012\/","title":{"rendered":"Terapia gen\u00e9tica SPOT-mRNA03 para distrofia muscular de Duchenne inicia ensaio cl\u00ednico de fase 1"},"content":{"rendered":"<p class=\"wp-block-paragraph\">O estudo de fase 1 inicial (NCT07188012) da terapia gen\u00e9tica SPOT-mRNA03 para Distrofia Muscular de Duchenne (DMD), desenvolvido pela Shanghai Siponuoyin Biotechnology, \u00e9 um estudo piloto sobre a seguran\u00e7a e a express\u00e3o da distrofina nos m\u00fasculos esquel\u00e9ticos ap\u00f3s a administra\u00e7\u00e3o.<\/p>\n\n\n\n<div class=\"wp-block-rank-math-toc-block\" id=\"rank-math-toc\"><h2>\u00cdndice<\/h2><nav><ul><li><a href=\"#what-is-spot-m-rna-03-gene-therapy\">O que \u00e9 a terapia gen\u00e9tica SPOT-mRNA03<\/a><\/li><li><a href=\"#spot-m-rna-03-gene-therapy-early-phase-1-nct-07188012\">Terapia gen\u00e9tica SPOT-mRNA03 Fase inicial 1 \u2013 NCT07188012<\/a><\/li><li><a href=\"#how-to-participate-in-the-sgt-003-clinical-trial\">Como participar do ensaio cl\u00ednico 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>O que \u00e9 a terapia gen\u00e9tica SPOT-mRNA03<\/strong><\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>SPOT-mRNA03 \u00e9 o produto de terapia gen\u00e9tica para Distrofia Muscular de Duchenne (DMD).<\/strong> Ap\u00f3s a administra\u00e7\u00e3o em pacientes com DMD, o pept\u00eddeo de direcionamento molecular na membrana dos EVs pode direcionar o SPOT-mRNA03 para as c\u00e9lulas musculares do paciente, permitindo a tradu\u00e7\u00e3o e a secre\u00e7\u00e3o de distrofina end\u00f3gena intacta de comprimento total por c\u00e9lulas receptoras deficientes em distrofina usando ribossomos intracelulares e amino\u00e1cidos.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"spot-m-rna-03-gene-therapy-early-phase-1-nct-07188012\"><strong>Terapia gen\u00e9tica SPOT-mRNA03 Fase inicial 1 \u2013 NCT07188012<\/strong><\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Trata-se de uma pesquisa cl\u00ednica explorat\u00f3ria, aberta, de bra\u00e7o \u00fanico, FIH e unic\u00eantrico, utilizando SPOT-mRNA03 administrado por via intravenosa em pacientes com DMD. O mRNA completo da distrofina est\u00e1 contido em ves\u00edculas extracelulares (VEs) direcionadas aos m\u00fasculos, chamadas SPOT-mRNA03. Pept\u00eddeos de direcionamento molecular na membrana das VEs conferem ao SPOT-mRNA03 sua capacidade de direcionamento, o que possibilita a distribui\u00e7\u00e3o do mRNA da distrofina como um produto de terapia g\u00eanica para DMD.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>In\u00edcio do estudo (estimado)<\/strong>: 2025-08-06<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Conclus\u00e3o prim\u00e1ria (estimada)<\/strong>: 2026-12<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Conclus\u00e3o do estudo (estimada)<\/strong>: 2026-12<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>N\u00famero de participantes (estimado)<\/strong>: 6<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Tipo de estudo<\/strong>: Fase Inicial 1<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Localiza\u00e7\u00e3o<\/strong>: <a href=\"https:\/\/maps.app.goo.gl\/Z2CyEqLydJ4Kxh9aA\" target=\"_blank\" rel=\"noreferrer noopener\">Centro M\u00e9dico Infantil de Xangai<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Idades eleg\u00edveis para estudo<\/strong>: 2 a 6 anos (machos ambulantes)<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Principais crit\u00e9rios de inclus\u00e3o e exclus\u00e3o. \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>Como participar do ensaio cl\u00ednico SPOT-mRNA03<\/strong><\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Para participar do ensaio cl\u00ednico de fase 1 inicial da terapia gen\u00e9tica SPOT-mRNA03, voc\u00ea pode usar as informa\u00e7\u00f5es de contato abaixo.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Nome<\/strong>: Cidade de Winston<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>N\u00famero de telefone<\/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\/pt\/wp-json\/wp\/v2\/posts\/4576","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/comments?post=4576"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/posts\/4576\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/media\/4580"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/media?parent=4576"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/categories?post=4576"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/tags?post=4576"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}