{"id":3150,"date":"2017-10-16T15:35:01","date_gmt":"2017-10-16T19:35:01","guid":{"rendered":"https:\/\/www.gensight-biologics.com\/gensight-biologics-presente-des-resultats-de-gs010-et-gs030-au-congres-2017-de-la-european-society-of-gene-cell-therapy\/"},"modified":"2026-09-24T00:09:53","modified_gmt":"2026-09-24T04:09:53","slug":"gensight-biologics-presente-des-resultats-de-gs010-et-gs030-au-congres-2017-de-la-european-society-of-gene-cell-therapy","status":"publish","type":"post","link":"https:\/\/www.gensight-biologics.com\/fr\/2017\/10\/16\/gensight-biologics-presente-des-resultats-de-gs010-et-gs030-au-congres-2017-de-la-european-society-of-gene-cell-therapy\/","title":{"rendered":"GenSight Biologics pr\u00e9sente des r\u00e9sultats de GS010 et GS030 au Congr\u00e8s 2017 de la European Society of Gene &#038; Cell Therapy"},"content":{"rendered":"\n<strong>Paris, le 16 octobre \u00a02017, 7h30 CET \u2013<\/strong> GenSight Biologics (Euronext : SIGHT, ISIN : 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 plusieurs pr\u00e9sentations de r\u00e9sultats au Congr\u00e8s 2017 de la European Society of Gene and Cell Therapy (ESGCT) \u00e0 Berlin, Allemagne, du 17 au 20 octobre 2017.\n<h2>GS010 \u2013 Neuropathie \u00a0Optique H\u00e9r\u00e9ditaire \u00a0de Leber (NOHL)<\/h2>\n<h3>\u201c78-week follow-up study results \u00a0after intravitreal rAAV2\/2-ND4 (GS010) injection in patients \u00a0with vision \u00a0loss due to G11778A ND4 Leber Hereditary \u00a0Optic Neuropathy\u201d<\/h3>\n<ul>\n \t<li>Anne Galy, GS010 Project Director, GenSight Biologics<\/li>\n \t<li>Poster P215<\/li>\n \t<li>Mercredi 18 octobre 2017, 18h30-21h00<\/li>\n<\/ul>\n<h3>\u201cHumoral and cellular \u00a0immune responses to AAV2 and ocular inflammation in patients \u00a0after intravitreal injection of rAAV2\/2-ND4 (GS010), an investigational gene therapy for the treatment \u00a0of ND4 LHON\u201d<\/h3>\n<ul>\n \t<li>C\u00e9line Bouquet, Senior Preclinical Manager, GenSight Biologics<\/li>\n \t<li>Poster P210<\/li>\n \t<li>Jeudi 19 octobre 2017, 18h45-20h15<\/li>\n<\/ul>\n<h2>GS030 \u2013 Optog\u00e9n\u00e9tique dans la R\u00e9tinopathie Pigmentaire (RP)<\/h2>\n<h3>\u201cEfficacy and Safety of ocular \u00a0AAV mediated \u00a0optogenetic therapy \u00a0for retinitis pigmentosa in rd1 mice and non-human primates \u00a0support the First-in-Human clinical trial of GS030\u201d<\/h3>\n<ul>\n \t<li>Anne Douar, GS030 Project Director, GenSight Biologics<\/li>\n \t<li>Pr\u00e9sentation orale OR07 \u2013 Session S1b: Ocular and CNS Gene and Cell Therapy I<\/li>\n \t<li>Mercredi 18 octobre 2017, 8h30-10h40<\/li>\n<\/ul>\n<h3>\u201cOcular tolerability of AAV2.7m8-ChrimsonR-tdTomato (GS030-DP) gene therapy product on blind rd1 mice injected \u00a0intravitreously and exposed to 595 nm LED light\u201d<\/h3>\n<ul>\n \t<li>C\u00e9line Bouquet, Senior Preclinical Manager, GenSight Biologics<\/li>\n \t<li>Poster P231<\/li>\n \t<li>Mercredi 18 octobre 2017, 18h30-21h00<\/li>\n<\/ul>\n<h3>\u201cToxicity, \u00a0immunogenicity and \u00a0biodistribution of \u00a0AAV2.7m8-ChrimsonR-tdTomato (GS030-DP) gene therapy product after bilateral \u00a0intravitreal administration in non-human primates\u201d<\/h3>\n<ul>\n \t<li>C\u00e9line Bouquet, Senior Preclinical Manager, GenSight Biologics<\/li>\n \t<li>Poster P214<\/li>\n \t<li>Jeudi 19 octobre 2017, 18h45-20h15<\/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<h2>\u00c0 propos \u00a0de GS030<\/h2>\nGS030 s\u2019appuie sur la technologie optog\u00e9n\u00e9tique de GenSight, une approche innovante visant \u00e0 restaurer la vision chez les patients en utilisant la th\u00e9rapie g\u00e9nique afin d\u2019introduire un g\u00e8ne codant pour une prot\u00e9ine photosensible \u00e0 l\u2019int\u00e9rieur de cellules sp\u00e9cifiques de la r\u00e9tine pour les rendre sensibles \u00e0 la lumi\u00e8re. Un dispositif de stimulation prenant la forme de lunettes, et visant \u00e0 stimuler sp\u00e9cifiquement les cellules transduites, est d\u00e9velopp\u00e9 afin d\u2019amplifier le signal lumineux. Sur la base de sa technologie optog\u00e9n\u00e9tique, et avec le soutien de l\u2019Institut de la Vision \u00e0 Paris, GenSight d\u00e9veloppe son second candidat-m\u00e9dicament, GS030, dans le but de restaurer la vision chez des patients souffrant de r\u00e9tinopathie pigmentaire, ou RP. La RP est une maladie orpheline caus\u00e9e par de multiples mutations de plusieurs g\u00e8nes impliqu\u00e9s dans le cycle visuel. La technologie optog\u00e9n\u00e9tique de GenSight est ind\u00e9pendante des mutations g\u00e9n\u00e9tiques responsables de la maladie. En moyenne, les patients souffrant de RP commencent \u00e0 perdre la vue \u00e0 l\u2019\u00e2ge de jeune adulte, pour atteindre la c\u00e9cit\u00e9 entre 40 et 45 ans. Il n\u2019y a actuellement aucun traitement pour la RP. La pr\u00e9valence est estim\u00e9e \u00e0 1,5 million de personnes affect\u00e9es dans le monde. GS030 pourrait b\u00e9n\u00e9ficier \u00e0 des stades pr\u00e9coces de la maladie.\n<h2>\u00c0 propos \u00a0de l\u2019Optog\u00e9n\u00e9tique<\/h2>\nL\u2019optog\u00e9n\u00e9tique est une technique biologique qui vise \u00e0 transf\u00e9rer un g\u00e8ne codant pour une prot\u00e9ine photosensible dans des cellules neuronales dans le but de provoquer une r\u00e9ponse de ces cellules \u00e0 la stimulation lumineuse. Il s\u2019agit ainsi d\u2019une m\u00e9thode de neuromodulation pouvant \u00eatre utilis\u00e9e pour modifier ou contr\u00f4ler individuellement l\u2019activit\u00e9 de certains neurones, dans des tissus vivants ou m\u00eame in-vivo, avec une extr\u00eame pr\u00e9cision spatiale et temporelle. \u00a0L\u2019optog\u00e9n\u00e9tique combine l\u2019utilisation de la th\u00e9rapie g\u00e9nique, pour transf\u00e9rer le g\u00e8ne dans les neurones cibles, et l\u2019utilisation de l\u2019optique et de l\u2019optronique pour d\u00e9livrer la lumi\u00e8re aux cellules transduites. L\u2019optog\u00e9n\u00e9tique est largement utilis\u00e9e dans les laboratoires de recherche du monde entier et est une approche prometteuse dans les domaines de la malvoyance et des maladies neurologiques.\n<p style=\"font-size: 16px\"><\/p>\n","protected":false},"excerpt":{"rendered":"<p>Paris, le 16 octobre \u00a02017, 7h30 CET \u2013 GenSight Biologics (Euronext : SIGHT, ISIN : 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 plusieurs pr\u00e9sentations de r\u00e9sultats au Congr\u00e8s 2017 de la [&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-3150","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 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>GenSight Biologics pr\u00e9sente des r\u00e9sultats de GS010 et GS030 au Congr\u00e8s 2017 de la European Society of Gene &amp; 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