{"id":4127,"date":"2012-12-12T15:13:29","date_gmt":"2012-12-12T13:13:29","guid":{"rendered":"https:\/\/www.institut-biotherapies.fr\/?page_id=4127"},"modified":"2026-08-07T15:43:12","modified_gmt":"2026-08-07T13:43:12","slug":"genethon","status":"publish","type":"page","link":"https:\/\/www.institut-biotherapies.fr\/en\/about-us\/genethon\/","title":{"rendered":"Genethon"},"content":{"rendered":"<p>Created in 1990 and funded almost exclusively by the AFM-Telethon, Genethon is a major player in gene therapy for rare diseases.<\/p>\n<p class=\"isSelectedEnd\">Genethon&#8217;s mission is to make innovative gene therapy treatments available to patients.<\/p>\n<p>After playing a pioneering role in deciphering the human genome (publishing the first maps of the human genome between 1992 and 1996), Genethon is now, with nearly 260 employees (researchers, physicians, engineers and regulatory affairs specialists), one of the leading international centres for preclinical and clinical research and development in gene therapy for rare diseases. In 2025, the laboratory published 29 scientific articles and filed 2 patents.<\/p>\n<p><img decoding=\"async\" class=\"alignleft wp-image-3246\" title=\"G\u00e9n\u00e9thon\" src=\"https:\/\/www.institut-biotherapies.fr\/wp-content\/uploads\/2012\/08\/image-genethon-325x216.jpg\" alt=\"G\u00e9n\u00e9thon\" width=\"210\" srcset=\"https:\/\/www.institut-biotherapies.fr\/wp-content\/uploads\/2012\/08\/image-genethon-325x216.jpg 325w, https:\/\/www.institut-biotherapies.fr\/wp-content\/uploads\/2012\/08\/image-genethon-420x280.jpg 420w, https:\/\/www.institut-biotherapies.fr\/wp-content\/uploads\/2012\/08\/image-genethon.jpg 670w\" sizes=\"(max-width: 325px) 100vw, 325px\" \/>Genethon has 15 products in clinical trials (in 2025), originating from its R&amp;D or developed in collaboration with partners, and 7 products are expected to enter clinical trials over the next five years. The programmes mainly focus on neuromuscular and liver diseases; Genethon has also conducted programmes for diseases affecting the immune system, blood and vision.<\/p>\n<p class=\"isSelectedEnd\">Highlights of 2025 include: the start of the pivotal phase of the clinical trial for Duchenne muscular dystrophy (GNT-0004), authorised by the European Medicines Agency and the United Kingdom, conducted as a double-blind trial in 72 boys aged 6 to 10 \u2014 with the first enrolments starting in France; the continuation of trials for limb-girdle muscular dystrophies (FKRP and gamma-sarcoglycanopathy); and the first results from the trial in patients with Crigler-Najjar syndrome who have anti-vector antibodies, demonstrating the feasibility and safety of the approach.<\/p>\n<p class=\"isSelectedEnd\">In addition, nearly 5,000 children with spinal muscular atrophy have been treated with gene therapy thanks to technologies resulting from Genethon&#8217;s research.<\/p>\n<p>Genethon is also the lead organisation of the GenoTher biocluster, selected as part of the France 2030 plan and awarded \u20ac70 million over five years, with the aim of accelerating the development of gene therapy medicines nationwide.<\/p>\n<h2>Expertise<\/h2>\n<ul>\n<li><strong>Research and development:<\/strong> design of therapeutic approaches and preclinical evaluation of gene therapy products for rare diseases; development of administration methods and optimisation of vectors; development of innovative technologies for bioproduction and control of gene therapy products; evaluation and control of the immune response; search for biomarkers; imaging and histology;<\/li>\n<li><strong>Clinical trials:<\/strong> large-scale production of batches of drug candidates to GMP standards (Genethon Bioprod) design, development and conduct of clinical trials (neuromuscular diseases, immunodeficiencies, vision disorders&#8230;); development of regulatory dossiers.<\/li>\n<\/ul>\n","protected":false},"excerpt":{"rendered":"<p>Created in 1990 and funded almost exclusively by the AFM-Telethon, Genethon is a major player in gene therapy for rare diseases. Genethon&#8217;s mission is to make innovative gene therapy treatments available to patients. After playing a pioneering role in deciphering the human genome (publishing the first maps of the human genome between 1992 and 1996), &hellip; <a href=\"https:\/\/www.institut-biotherapies.fr\/en\/about-us\/genethon\/\">[Read more]<\/a><\/p>\n","protected":false},"author":3,"featured_media":0,"parent":2997,"menu_order":30,"comment_status":"closed","ping_status":"closed","template":"page-sidecolumn.php","meta":{"footnotes":""},"class_list":["post-4127","page","type-page","status-publish","hentry"],"_links":{"self":[{"href":"https:\/\/www.institut-biotherapies.fr\/en\/wp-json\/wp\/v2\/pages\/4127","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.institut-biotherapies.fr\/en\/wp-json\/wp\/v2\/pages"}],"about":[{"href":"https:\/\/www.institut-biotherapies.fr\/en\/wp-json\/wp\/v2\/types\/page"}],"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=4127"}],"version-history":[{"count":6,"href":"https:\/\/www.institut-biotherapies.fr\/en\/wp-json\/wp\/v2\/pages\/4127\/revisions"}],"predecessor-version":[{"id":6884,"href":"https:\/\/www.institut-biotherapies.fr\/en\/wp-json\/wp\/v2\/pages\/4127\/revisions\/6884"}],"up":[{"embeddable":true,"href":"https:\/\/www.institut-biotherapies.fr\/en\/wp-json\/wp\/v2\/pages\/2997"}],"wp:attachment":[{"href":"https:\/\/www.institut-biotherapies.fr\/en\/wp-json\/wp\/v2\/media?parent=4127"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}