{"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\/pt\/gnr-097-duchenne-gene-therapy-phase-1-2-study-started-by-generium\/","title":{"rendered":"GNR-097: Estudo de Fase 1\/2 da Terapia G\u00eanica de Duchenne iniciado por Generium"},"content":{"rendered":"<p class=\"wp-block-paragraph\"><strong>A Generium, uma empresa russa de biotecnologia inovadora, recebeu aprova\u00e7\u00e3o para conduzir um ensaio cl\u00ednico do medicamento de terapia gen\u00e9tica GNR-097 para o tratamento da distrofia muscular de Duchenne progressiva (DMD).<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">O ensaio cl\u00ednico multic\u00eantrico, simples-cego, randomizado e controlado por placebo envolve uma \u00fanica administra\u00e7\u00e3o intravenosa do medicamento de terapia g\u00eanica em pacientes pedi\u00e1tricos com distrofia muscular de Duchenne progressiva. Este estudo de Fase 1\/2 visa avaliar a tolerabilidade, a seguran\u00e7a e a efic\u00e1cia do medicamento GNR-097.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Terapia G\u00eanica GNR-097 Duchenne<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>GNR-097 \u00e9 um vetor viral adeno-associado recombinante do sorotipo 9, que codifica um gene de distrofina truncado.<\/strong><\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Daniil Talyansky, CEO da GENERIUM:<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">A terapia g\u00eanica \u00e9 o campo mais jovem e inovador da ind\u00fastria farmac\u00eautica e da ci\u00eancia m\u00e9dica global. A expertise \u00fanica de cientistas russos permitiu o desenvolvimento de um vetor viral adenoassociado recombinante que codifica o gene da microdistrofina. Estamos iniciando agora os ensaios cl\u00ednicos deste medicamento original, que \u00e9 muito promissor.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Ensaio cl\u00ednico da terapia gen\u00e9tica GNR-097<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">O ensaio cl\u00ednico deste medicamento inovador \u00e9 internacional e ser\u00e1 conduzido na Federa\u00e7\u00e3o Russa e na Rep\u00fablica da Bielorr\u00fassia. Na R\u00fassia, o estudo ser\u00e1 realizado em cinco centros m\u00e9dicos: o Instituto de Pesquisa Cl\u00ednica de Pediatria e Cirurgia Pedi\u00e1trica, em homenagem ao acad\u00eamico Yu.E. Veltishchev, a Universidade Nacional Russa de Pesquisa M\u00e9dica Pirogov, a Universidade Estadual de Pediatria de S\u00e3o Petersburgo, o Hospital Cl\u00ednico Infantil Regional de Ecaterimburgo e o Centro Nacional de Pesquisa M\u00e9dica para a Sa\u00fade Infantil. Na Bielorr\u00fassia, o ensaio cl\u00ednico ser\u00e1 realizado no Centro Cient\u00edfico e Pr\u00e1tico Republicano &quot;M\u00e3e e Filho&quot;.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Ler mais<\/strong>:\u00a0<a href=\"https:\/\/dmdwarrior.com\/pt\/dmd-therapies\/\" target=\"_blank\" rel=\"noreferrer noopener\">Ensaios cl\u00ednicos para distrofia muscular de Duchenne (lista de todas as pesquisas)<\/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\/pt\/wp-json\/wp\/v2\/posts\/4428","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=4428"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/posts\/4428\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/media\/4430"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/media?parent=4428"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/categories?post=4428"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/tags?post=4428"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}