{"id":3674,"date":"2021-02-12T16:07:51","date_gmt":"2021-02-12T21:07:51","guid":{"rendered":"https:\/\/www.gensight-biologics.com\/4eme-congres-annuel-gene-therapy-for-rare-disorders-format-virtuel\/"},"modified":"2026-07-15T15:57:52","modified_gmt":"2026-07-15T19:57:52","slug":"4eme-congres-annuel-gene-therapy-for-rare-disorders-format-virtuel","status":"publish","type":"post","link":"https:\/\/www.gensight-biologics.com\/fr\/2021\/02\/12\/4eme-congres-annuel-gene-therapy-for-rare-disorders-format-virtuel\/","title":{"rendered":"4\u00e8me Congr\u00e8s Annuel Gene Therapy for Rare Disorders – format virtuel"},"content":{"rendered":"\nGensight Biologics participera \u00e0 la 4\u00e8me \u00e9dition du congr\u00e8s annuel Gene Therapy for Rare Disorders<\/a> qui se tiendra dans un format virtuel du lundi 22 f\u00e9vrier au jeudi 25 f\u00e9vrier 2021. L’\u00e9v\u00e9nement se concentrera exclusivement sur la r\u00e9solution des d\u00e9fis commerciaux \u00e0 venir auxquels sont confront\u00e9s les d\u00e9veloppeurs de m\u00e9dicaments lorsqu’ils commercialisent des th\u00e9rapies g\u00e9niques sur le march\u00e9.\n\nMagali Taiel<\/strong>, Directeur M\u00e9dical, donnera une pr\u00e9sentation orale sur LUMEVOQ\u00ae (GS010 ; lenadogene nolparvovec), une th\u00e9rapie g\u00e9nique ciblant la Neuropathie Optique H\u00e9r\u00e9ditaire de Leber (NOHL), une maladie mitochondriale rare qui conduit \u00e0 la c\u00e9cit\u00e9 chez les jeunes adultes.\n\nTitre : Lumevoq<\/span><\/strong>\u00ae<\/strong> Gene Therapy in Leber Hereditary Optic Neuropathy (LHON)<\/strong><\/span>\n
    \n \t
  • LHON est une maladie mitochondriale h\u00e9r\u00e9ditaire maternelle. C’est une maladie rare, avec une incidence de 1500 nouveaux patients par an en Europe et aux \u00c9tats-Unis. La mutation 11778 sur le g\u00e8ne mitochondrial ND4 est la mutation \u00e0 l’origine de la forme clinique la plus s\u00e9v\u00e8re de LHON. Lumevoq\u00ae cible sp\u00e9cifiquement cette mutation.<\/li>\n \t
  • Lumevoq\u00ae est un vecteur ad\u00e9novirus recombinant de type 2, contenant l’ADN ND4 de type sauvage. Lumevoq\u00ae est inject\u00e9 par injection intravitr\u00e9enne.<\/li>\n \t
  • GenSight Biologics a d\u00e9pos\u00e9 une demande d’enregistrement aupr\u00e8s de l\u2019EMA en septembre 2020, puis pr\u00e9pare le d\u00e9p\u00f4t d\u2019un BLA pour l’enregistrement am\u00e9ricain en 2021.<\/li>\n<\/ul>\n \n\nDate & Heure : Mercredi 24 f\u00e9vrier, 8.30 9.00am EST (14h30 \u2013 15h00 heure de Paris)<\/strong>\n\nSession:\u00a0 The View from The Cutting Edge: Gaining the Perspective of the Industry Leaders<\/strong>\n","protected":false},"excerpt":{"rendered":"

    Gensight Biologics participera \u00e0 la 4\u00e8me \u00e9dition du congr\u00e8s annuel Gene Therapy for Rare Disorders qui se tiendra dans un format virtuel du lundi 22 f\u00e9vrier au jeudi 25 f\u00e9vrier 2021. L’\u00e9v\u00e9nement se concentrera exclusivement sur la r\u00e9solution des d\u00e9fis commerciaux \u00e0 venir auxquels sont confront\u00e9s les d\u00e9veloppeurs de m\u00e9dicaments lorsqu’ils commercialisent des th\u00e9rapies g\u00e9niques […]<\/p>\n","protected":false},"author":4,"featured_media":0,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"footnotes":"","_links_to":"","_links_to_target":""},"categories":[55],"tags":[],"featured-area":[],"news-topic":[73,79],"news-type":[74,75,77],"post-year":[],"class_list":["post-3674","post","type-post","status-publish","format-standard","hentry","category-evenements","news-topic-gs010-lumevoq","news-topic-company-and-personnel","news-type-product-candidates","news-type-clinical","news-type-conferences"],"acf":[],"yoast_head":"\n4\u00e8me Congr\u00e8s Annuel Gene Therapy for Rare Disorders - format virtuel - GenSight Biologics<\/title>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/www.gensight-biologics.com\/fr\/2021\/02\/12\/4eme-congres-annuel-gene-therapy-for-rare-disorders-format-virtuel\/\" \/>\n<meta property=\"og:locale\" content=\"fr_FR\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"4\u00e8me Congr\u00e8s Annuel Gene Therapy for Rare Disorders - format virtuel - GenSight Biologics\" \/>\n<meta property=\"og:description\" content=\"Gensight Biologics participera \u00e0 la 4\u00e8me \u00e9dition du congr\u00e8s annuel Gene Therapy for Rare Disorders qui se tiendra dans un format virtuel du lundi 22 f\u00e9vrier au jeudi 25 f\u00e9vrier 2021. 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