{"id":3860,"date":"2025-05-15T13:01:14","date_gmt":"2025-05-15T10:01:14","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=3860"},"modified":"2025-05-15T13:01:16","modified_gmt":"2025-05-15T10:01:16","slug":"precision-biosciences-pbgene-dmd-restores-production-of-nearly-full-length-dystrophin-protein","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/pt\/precision-biosciences-pbgene-dmd-restores-production-of-nearly-full-length-dystrophin-protein\/","title":{"rendered":"Precision BioSciences PBGENE-DMD restaura a produ\u00e7\u00e3o de prote\u00edna distrofina quase completa"},"content":{"rendered":"<p class=\"wp-block-paragraph\">A Precision BioSciences anunciou hoje a prioriza\u00e7\u00e3o estrat\u00e9gica e a acelera\u00e7\u00e3o do PBGENE-DMD, a primeira abordagem de edi\u00e7\u00e3o gen\u00e9tica in vivo da empresa para Distrofia Muscular de Duchenne (DMD).<\/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-pbgene-dmd\">O que \u00e9 PBGENE-DMD?<\/a><\/li><li><a href=\"#how-the-pbgene-dmd-approach-works\">Como funciona a abordagem PBGENE-DMD?<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"what-is-pbgene-dmd\">O que \u00e9 PBGENE-DMD?<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>PBGENE-DMD \u00e9 uma abordagem de edi\u00e7\u00e3o gen\u00e9tica in vivo de primeira classe para a maioria dos pacientes com Distrofia Muscular de Duchenne afetados por muta\u00e7\u00f5es da distrofina na regi\u00e3o de &quot;ponto cr\u00edtico&quot; mais comum entre os \u00e9xons 45-55.<\/li>\n<\/ul>\n\n\n\n<ul class=\"wp-block-list\">\n<li>O candidato cl\u00ednico final PBGENE-DMD demonstra dados pr\u00e9-cl\u00ednicos convincentes para melhorar de forma duradoura o benef\u00edcio funcional ao longo do tempo.<\/li>\n<\/ul>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Segmenta\u00e7\u00e3o de precis\u00e3o para enviar um Novo Medicamento Investigacional (IND) e\/ou Pedido de Ensaio Cl\u00ednico (CTA) para PBGENE-DMD em 2025, com dados cl\u00ednicos esperados para 2026.<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">Leia mais: <a href=\"https:\/\/dmdwarrior.com\/pt\/precision-biosciences-presents-preclinical-efficacy-and-durability-data-on-pbgene-dmd-for-the-treatment-of-duchenne-muscular-dystrophy-dmd-at-the-2025-muscular-dystrophy-association-mda-clinical\/\" target=\"_blank\" rel=\"noreferrer noopener\">PBGENE-DMD para o tratamento da distrofia muscular de Duchenne (DMD)<\/a><\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"how-the-pbgene-dmd-approach-works\">Como funciona a abordagem PBGENE-DMD?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Atualmente, n\u00e3o h\u00e1 terapias aprovadas que possam promover melhorias funcionais musculares significativas e duradouras. O PBGENE-DMD emprega duas nucleases ARCUS complementares administradas em um \u00fanico AAV para excisar os \u00e9xons 45-55 do gene da distrofina com o objetivo de restaurar a produ\u00e7\u00e3o natural do corpo de uma prote\u00edna distrofina funcional.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Em dados pr\u00e9-cl\u00ednicos apresentados no ASGCT, o PBGENE-DMD demonstrou melhora funcional significativa e duradoura em um modelo murino humanizado com DMD. Ap\u00f3s a administra\u00e7\u00e3o do AAV, o PBGENE-DMD restaurou a capacidade do organismo de produzir uma prote\u00edna distrofina funcional amplamente em m\u00faltiplos m\u00fasculos, incluindo os m\u00fasculos card\u00edaco e esquel\u00e9tico. Ao longo de 9 meses, camundongos tratados com PBGENE-DMD apresentaram aumento na express\u00e3o da prote\u00edna distrofina, resultando em melhora muscular funcional substancial e sustentada. Al\u00e9m disso, a transcri\u00e7\u00e3o do mRNA da distrofina editada pelo PBGENE-DMD em c\u00e9lulas-tronco sat\u00e9lite musculares, que s\u00e3o c\u00e9lulas progenitoras de novas c\u00e9lulas musculares, refor\u00e7a o potencial de durabilidade a longo prazo.<\/p>","protected":false},"excerpt":{"rendered":"<p>Precision BioSciences, today announced the strategic prioritization and acceleration of PBGENE-DMD, the Company\u2019s first-in-class in vivo gene editing approach for Duchenne Muscular Dystrophy (DMD). What is PBGENE-DMD? Read more: PBGENE-DMD for the Treatment of Duchenne Muscular Dystrophy (DMD) How the PBGENE-DMD Approach Works? There are currently no approved therapies that can drive significant and durable [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":3863,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[359,72,314,313],"class_list":["post-3860","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-full-length-dystrophin","tag-gene-editing","tag-pbgene-dmd","tag-precision-biosciences"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/posts\/3860","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=3860"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/posts\/3860\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/media\/3863"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/media?parent=3860"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/categories?post=3860"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/tags?post=3860"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}