{"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\/es\/spot-mrna03-gene-therapy-early-phase-1-clinical-trial-nct07188012\/","title":{"rendered":"La terapia g\u00e9nica SPOT-mRNA03 para la distrofia muscular de Duchenne comienza su ensayo cl\u00ednico de fase 1."},"content":{"rendered":"<p class=\"wp-block-paragraph\">El estudio de fase 1 temprana (NCT07188012) de la terapia g\u00e9nica SPOT-mRNA03 para la distrofia muscular de Duchenne (DMD) desarrollado por Shanghai Siponuoyin Biotechnology es un estudio piloto de la seguridad y expresi\u00f3n de la distrofina en los m\u00fasculos esquel\u00e9ticos despu\u00e9s de su administraci\u00f3n.<\/p>\n\n\n\n<div class=\"wp-block-rank-math-toc-block\" id=\"rank-math-toc\"><h2>Tabla de contenido<\/h2><nav><ul><li><a href=\"#what-is-spot-m-rna-03-gene-therapy\">\u00bfQu\u00e9 es la terapia g\u00e9nica SPOT-mRNA03?<\/a><\/li><li><a href=\"#spot-m-rna-03-gene-therapy-early-phase-1-nct-07188012\">Terapia g\u00e9nica SPOT-mRNA03, fase inicial 1 \u2013 NCT07188012<\/a><\/li><li><a href=\"#how-to-participate-in-the-sgt-003-clinical-trial\">C\u00f3mo participar en el ensayo 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>\u00bfQu\u00e9 es la terapia g\u00e9nica SPOT-mRNA03?<\/strong><\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>SPOT-mRNA03 es el producto de terapia gen\u00e9tica para la distrofia muscular de Duchenne (DMD).<\/strong> Despu\u00e9s de la administraci\u00f3n a pacientes con DMD, el p\u00e9ptido de orientaci\u00f3n molecular en la membrana de las EV puede dirigir SPOT-mRNA03 hacia las c\u00e9lulas musculares del paciente, lo que permite la traducci\u00f3n y secreci\u00f3n de distrofina intacta de longitud completa end\u00f3gena por c\u00e9lulas receptoras deficientes en distrofina utilizando ribosomas intracelulares y 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 g\u00e9nica SPOT-mRNA03, fase inicial 1 \u2013 NCT07188012<\/strong><\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Se trata de una investigaci\u00f3n cl\u00ednica exploratoria de un solo brazo, abierta, FIH y unic\u00e9ntrica que utiliza SPOT-mRNA03 administrado por v\u00eda intravenosa a pacientes con DMD. El ARNm completo de la distrofina se encuentra en ves\u00edculas extracelulares (VE) dirigidas al m\u00fasculo, denominadas SPOT-mRNA03. Los p\u00e9ptidos moleculares diana en la membrana de las VE confieren a SPOT-mRNA03 su capacidad de diana, lo que permite distribuir el ARNm de la distrofina como producto de terapia g\u00e9nica para la DMD.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Inicio del estudio (estimado)<\/strong>: 2025-08-06<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Finalizaci\u00f3n primaria (estimada)<\/strong>: 2026-12<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Finalizaci\u00f3n del estudio (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 estudio<\/strong>:Fase temprana 1<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Ubicaci\u00f3n<\/strong>: <a href=\"https:\/\/maps.app.goo.gl\/Z2CyEqLydJ4Kxh9aA\" target=\"_blank\" rel=\"noreferrer noopener\">Centro M\u00e9dico Infantil de Shangh\u00e1i<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Edades elegibles para estudiar<\/strong>:De 2 a 6 a\u00f1os (varones ambulantes)<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Criterios principales de inclusi\u00f3n y exclusi\u00f3n. \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>C\u00f3mo participar en el ensayo cl\u00ednico SPOT-mRNA03<\/strong><\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Para participar en el ensayo cl\u00ednico de fase 1 temprana de la terapia g\u00e9nica SPOT-mRNA03, puede utilizar la informaci\u00f3n de contacto que aparece a continuaci\u00f3n.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Nombre<\/strong>: Ciudad de Winston<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>N\u00famero de tel\u00e9fono<\/strong>: 212-920-5501<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Correo electr\u00f3nico<\/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\/es\/wp-json\/wp\/v2\/posts\/4576","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/dmdwarrior.com\/es\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/dmdwarrior.com\/es\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/es\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/es\/wp-json\/wp\/v2\/comments?post=4576"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/es\/wp-json\/wp\/v2\/posts\/4576\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/es\/wp-json\/wp\/v2\/media\/4580"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/es\/wp-json\/wp\/v2\/media?parent=4576"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/es\/wp-json\/wp\/v2\/categories?post=4576"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/es\/wp-json\/wp\/v2\/tags?post=4576"}],"curies":[{"name":"gracias","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}