Audentes Therapeutics, a biotechnology company, launched the first gene therapy clinical trial in children with myotubular myopathy disease using a gene therapy product designed at Genethon. At the 12-week timepoint, the first 3 children treated show early signs of efficacy.<\/p>\n
With the support of the AFM-T\u00e9l\u00e9thon and the Myotubular Trust, Dr Ana Buj-Bello led the Genethon team in designing an adeno-associated virus (AAV) capable of delivering the MTM1 gene to muscle cells. Preclinical studies demonstrated spectacular efficacy in a mouse model and in dogs naturally affected by the disease. On the basis of this early work, Audentes Therapeutics initiated an international phase I \/ II clinical trial, aimed at evaluating the safety and efficacy of the product in approximately 12 patients less than 5 years of age.
\nTwelve weeks after administration in the first child, Audentes Therapeutics reported positive interim data from the first cohort of patients, including:
\n– Significant improvements in neuromuscular function
\n– Significant improvements in respiratory function
\n– Improvement of the general condition of treated children<\/p>\n
Discover in detail the results for each of the 3 children treated<\/a><\/p>\n","protected":false},"excerpt":{"rendered":" Audentes Therapeutics, a biotechnology company, launched the first gene therapy clinical trial in children with myotubular myopathy disease using a gene therapy product designed at Genethon. At the 12-week timepoint, the first 3 children treated show early signs of efficacy. With the support of the AFM-T\u00e9l\u00e9thon and the Myotubular Trust, Dr Ana Buj-Bello led the … [Read more]<\/a><\/p>\n","protected":false},"author":3,"featured_media":0,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[9],"tags":[],"class_list":["post-5984","post","type-post","status-publish","format-standard","hentry","category-press-releases"],"_links":{"self":[{"href":"https:\/\/www.institut-biotherapies.fr\/en\/wp-json\/wp\/v2\/posts\/5984","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.institut-biotherapies.fr\/en\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.institut-biotherapies.fr\/en\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.institut-biotherapies.fr\/en\/wp-json\/wp\/v2\/users\/3"}],"replies":[{"embeddable":true,"href":"https:\/\/www.institut-biotherapies.fr\/en\/wp-json\/wp\/v2\/comments?post=5984"}],"version-history":[{"count":2,"href":"https:\/\/www.institut-biotherapies.fr\/en\/wp-json\/wp\/v2\/posts\/5984\/revisions"}],"predecessor-version":[{"id":5986,"href":"https:\/\/www.institut-biotherapies.fr\/en\/wp-json\/wp\/v2\/posts\/5984\/revisions\/5986"}],"wp:attachment":[{"href":"https:\/\/www.institut-biotherapies.fr\/en\/wp-json\/wp\/v2\/media?parent=5984"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.institut-biotherapies.fr\/en\/wp-json\/wp\/v2\/categories?post=5984"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.institut-biotherapies.fr\/en\/wp-json\/wp\/v2\/tags?post=5984"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}