{"id":3117,"date":"2017-04-20T15:09:37","date_gmt":"2017-04-20T19:09:37","guid":{"rendered":"https:\/\/www.gensight-biologics.com\/gensight-biologics-presente-gs010-au-congres-annuel-2017-de-lamerican-academy-of-neurology\/"},"modified":"2026-09-24T00:09:58","modified_gmt":"2026-09-24T04:09:58","slug":"gensight-biologics-presente-gs010-au-congres-annuel-2017-de-lamerican-academy-of-neurology","status":"publish","type":"post","link":"https:\/\/www.gensight-biologics.com\/fr\/2017\/04\/20\/gensight-biologics-presente-gs010-au-congres-annuel-2017-de-lamerican-academy-of-neurology\/","title":{"rendered":"GenSight Biologics pr\u00e9sente GS010 au Congr\u00e8s annuel 2017 de l\u2019American Academy of Neurology"},"content":{"rendered":"\n<strong>Paris, 20 avril 2017, 7h30 CET \u2013<\/strong> GenSight Biologics (Euronext : SIGHT, FR0013183985, \u00e9ligible PEA- PME), soci\u00e9t\u00e9 biopharmaceutique d\u00e9di\u00e9e \u00e0 la d\u00e9couverte et au d\u00e9veloppement de th\u00e9rapies g\u00e9niques innovantes pour le traitement des maladies neurod\u00e9g\u00e9n\u00e9ratives de la r\u00e9tine et du syst\u00e8me nerveux central, annonce aujourd\u2019hui qu\u2019une communication a \u00e9t\u00e9 s\u00e9lectionn\u00e9e pour une pr\u00e9sentation orale au Congr\u00e8s Annuel 2017 de l\u2019American Academy of Neurology (AAN) \u00e0 Boston, MA, Etats-Unis, du 22 au 28 avril 2017.\n\n<strong>\u201cIntravitreal rAAV2\/2-ND4 (GS010): A gene therapy for vision loss in Leber&rsquo;s Hereditary Optic Neuropathy (LHON) caused by the G11778A ND4 mitochondrial mutation\u201d<\/strong> sera pr\u00e9sent\u00e9 par le <strong>Pr. Jos\u00e9-Alain Sahel, MD<\/strong>, Directeur de l\u2019Institut de la Vision, Paris, Chef du service d\u2019ophtalmologie au Centre Hospitalier National d&rsquo;Ophtalmologie des XV-XX, Paris, Professeur et Chef du service d\u2019ophtalmologie \u00e0 l\u2019University of Pittsburg School of Medicine and Medical Center, et co-fondateur de GenSight Biologics.\n<ul>\n \t<li>Pr\u00e9sentation orale<\/li>\n \t<li>S26 \u2013 Neuro-ophthalmology \/ Neuro-otology<\/li>\n \t<li>Mardi 25 avril 2017, 16h18-16h30 (heure locale)<\/li>\n<\/ul>\n<h2>\u00c0 propos \u00a0de GenSight Biologics<\/h2>\nGenSight Biologics S.A. (GenSight Biologics) est une soci\u00e9t\u00e9 biopharmaceutique d\u00e9di\u00e9e \u00e0 la d\u00e9couverte et au d\u00e9veloppement de th\u00e9rapies g\u00e9niques innovantes pour le traitement des maladies neurod\u00e9g\u00e9n\u00e9ratives de la r\u00e9tine et du syst\u00e8me nerveux central. Le portefeuille de recherche de GenSight Biologics s\u2019appuie sur deux plates-formes technologiques : le ciblage mitochondrial (Mitochondrial Targeting Sequence, ou MTS) et l\u2019optog\u00e9n\u00e9tique, visant \u00e0 pr\u00e9server ou restaurer la vision chez les patients atteints de maladies neurod\u00e9g\u00e9n\u00e9ratives de la r\u00e9tine. Le candidat m\u00e9dicament le plus avanc\u00e9 de GenSight Biologics, GS010, est en Phase III pour le traitement de la neuropathie optique h\u00e9r\u00e9ditaire de Leber (NOHL), une maladie mitochondriale rare qui conduit \u00e0 une perte irr\u00e9versible de la vue chez les adolescents et les jeunes adultes. En utilisant son approche de th\u00e9rapie g\u00e9nique, les candidats m\u00e9dicaments de GenSight Biologics sont destin\u00e9s \u00e0 offrir aux patients une r\u00e9cup\u00e9ration visuelle fonctionnelle durable apr\u00e8s une seule injection intravitr\u00e9enne dans chaque \u0153il.\n<h2>\u00c0 propos \u00a0de GS010<\/h2>\nGS010 cible la neuropathie optique h\u00e9r\u00e9ditaire de Leber (NOHL), une maladie mitochondriale rare, de transmission maternelle, caract\u00e9ris\u00e9e par une d\u00e9g\u00e9n\u00e9rescence des cellules ganglionnaires de la r\u00e9tine et provoquant en moins d\u2019un an une perte brutale et irr\u00e9versible de la vision conduisant g\u00e9n\u00e9ralement \u00e0 la c\u00e9cit\u00e9 l\u00e9gale. Ces sympt\u00f4mes apparaissent principalement chez les adolescents et les jeunes adultes. GS010 s\u2019appuie sur une technologie propri\u00e9taire de s\u00e9quence de ciblage mitochondrial (MTS), issue des travaux de l\u2019Institut de la Vision, qui, lorsqu\u2019elle est associ\u00e9e au g\u00e8ne d\u2019int\u00e9r\u00eat, permet de l\u2019adresser sp\u00e9cifiquement \u00e0 l\u2019int\u00e9rieur de la mitochondrie gr\u00e2ce \u00e0 un vecteur AAV (Adeno-Associated Virus). Le g\u00e8ne d\u2019int\u00e9r\u00eat est ainsi transf\u00e9r\u00e9 dans la cellule pour y \u00eatre exprim\u00e9 et produire la prot\u00e9ine fonctionnelle, qui sera achemin\u00e9e \u00e0 l\u2019int\u00e9rieur des mitochondries gr\u00e2ce aux s\u00e9quences nucl\u00e9otidiques sp\u00e9cifiques, afin de restaurer la fonction mitochondriale d\u00e9ficiente ou manquante.\n","protected":false},"excerpt":{"rendered":"<p>Paris, 20 avril 2017, 7h30 CET \u2013 GenSight Biologics (Euronext : SIGHT, FR0013183985, \u00e9ligible PEA- PME), soci\u00e9t\u00e9 biopharmaceutique d\u00e9di\u00e9e \u00e0 la d\u00e9couverte et au d\u00e9veloppement de th\u00e9rapies g\u00e9niques innovantes pour le traitement des maladies neurod\u00e9g\u00e9n\u00e9ratives de la r\u00e9tine et du syst\u00e8me nerveux central, annonce aujourd\u2019hui qu\u2019une communication a \u00e9t\u00e9 s\u00e9lectionn\u00e9e pour une pr\u00e9sentation orale au [&hellip;]<\/p>\n","protected":false},"author":3,"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":[54],"tags":[],"featured-area":[],"news-topic":[],"news-type":[],"post-year":[],"class_list":["post-3117","post","type-post","status-publish","format-standard","hentry","category-communiques-de-presse-financiers"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v28.8 - 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