{"id":22177,"date":"2023-12-16T21:12:50","date_gmt":"2023-12-16T21:12:50","guid":{"rendered":"https:\/\/dupmecp2.eu\/huidagene-une-technologie-crispr-cas13-prometteuse-venue-de-chine-shanghai\/"},"modified":"2023-12-20T18:46:25","modified_gmt":"2023-12-20T18:46:25","slug":"huidagene-une-technologie-crispr-cas13-prometteuse-venue-de-chine-shanghai","status":"publish","type":"post","link":"https:\/\/dupmecp2.eu\/de\/huidagene-une-technologie-crispr-cas13-prometteuse-venue-de-chine-shanghai\/","title":{"rendered":"Huidagene : Une technologie CRISPR-CAS13 prometteuse venue de Chine (Shanghai)"},"content":{"rendered":"

Huidagene Therapeutics est une soci\u00e9t\u00e9 de biotechnologie bas\u00e9e \u00e0 Shanghai et dans le New Jersey, qui se concentre sur la d\u00e9couverte, l’ing\u00e9nierie et le d\u00e9veloppement de nouveaux m\u00e9dicaments bas\u00e9s sur la technologie CRISPR. L’entreprise se concentre sur les troubles musculaires, ophtalmiques et neurologiques.<\/p>\n\n\n\n

Huidagene a d\u00e9velopp\u00e9 HG204, un vecteur viral ad\u00e9no-associ\u00e9 (AAV) d\u00e9livrant un innovant complexe CRISPR-Cas13 dans les cellules. Sp\u00e9cialement con\u00e7u pour le syndrome de duplication du g\u00e8ne MECP2, HG204 vise \u00e0 r\u00e9guler la surproduction de la prot\u00e9ine MeCP2 en induisant la d\u00e9gradation de l’ARN MECP2 en exc\u00e8s.<\/strong> Ce candidat-m\u00e9dicament innovant a le potentiel de gu\u00e9rir le syndrome de la duplication du g\u00e8ne MECP2 en une seule administration.<\/p>\n\n\n\n

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Notre organisation a eu des discussions productives avec les dirigeants de Huidagene pour en savoir plus sur cette technologie innovante et ce qu’elle repr\u00e9sente pour l’avenir. Des \u00e9tudes pr\u00e9cliniques utilisant HG204 dans un mod\u00e8le de souris humanis\u00e9 ont d\u00e9montr\u00e9 une r\u00e9duction significative de la prot\u00e9ine MeCP2 et une inversion des sympt\u00f4mes.<\/strong> En plus du fait que ces r\u00e9sultats soient encourageants, il s’agit \u00e9galement de la deuxi\u00e8me preuve de la r\u00e9versibilit\u00e9 des sympt\u00f4mes du syndrome apr\u00e8s la r\u00e9gulation des niveaux de prot\u00e9ine MECP2 (comme pr\u00e9c\u00e9demment d\u00e9montr\u00e9 par l’administration de l’ASO par le Pr. H. Zoghbi).<\/p>\n\n\n\n

<\/div>\n\n\n\n

Huidagene finalise actuellement les \u00e9tudes pr\u00e9cliniques sur le HG204 et se pr\u00e9pare \u00e0 un essai clinique pour les patients en Chine, qui devrait d\u00e9buter en \u00e9t\u00e9 2024. <\/strong>Si cette \u00e9tude s’av\u00e8re concluante, Huidagene pr\u00e9voit de mener des essais cliniques dans d’autres pays, comme les \u00c9tats-Unis et l’Europe, en 2025-2026.<\/p>\n\n\n\n


Le 31 octobre, la FDA (Food and Drug Administration, l’autorit\u00e9 de sant\u00e9 des \u00c9tats-Unis) a accord\u00e9 \u00e0 HG204 les d\u00e9signations de maladie p\u00e9diatrique rare et de m\u00e9dicament orphelin. Cette d\u00e9signation r\u00e9glementaire est une premi\u00e8re \u00e9tape vers un essai clinique en dehors de la chine et permettra un examen acc\u00e9l\u00e9r\u00e9 du dossier lors de l’enregistrement du m\u00e9dicament. Bien que la nouvelle soit totalement inattendue, elle apporte un espoir suppl\u00e9mentaire \u00e0 la communaut\u00e9.<\/p>\n\n\n\n

Notre \u00e9quipe restera en contact avec Huidagene et tiendra notre communaut\u00e9 inform\u00e9e de tout nouveau d\u00e9veloppement.<\/p>\n\n\n\n

\"\"<\/figure>\n\n\n\n
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Vers la revue de presse<\/a><\/div>\n<\/div>","protected":false},"excerpt":{"rendered":"

Huidagene Therapeutics est une soci\u00e9t\u00e9 de biotechnologie bas\u00e9e \u00e0 Shanghai et dans le New Jersey, qui se concentre sur la d\u00e9couverte, l’ing\u00e9nierie et le d\u00e9veloppement de nouveaux m\u00e9dicaments bas\u00e9s sur […]<\/p>","protected":false},"author":1,"featured_media":22151,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[7],"tags":[],"class_list":["post-22177","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-actualite"],"yoast_head":"\nHuidagene : Une technologie CRISPR-CAS13 prometteuse venue de Chine (Shanghai) - DupMECP2<\/title>\n<meta name=\"description\" content=\"Une nouvelle perspective potentielle pour le traitement du syndrome de duplication MECP2 avec le CRIPSR-Cas13 de Huidagene. 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