{"id":25235,"date":"2024-04-03T14:59:40","date_gmt":"2024-04-03T14:59:40","guid":{"rendered":"https:\/\/dupmecp2.eu\/?p=25235"},"modified":"2025-07-23T10:39:41","modified_gmt":"2025-07-23T08:39:41","slug":"studi-clinici-su-huidagene","status":"publish","type":"post","link":"https:\/\/dupmecp2.eu\/it\/studi-clinici-su-huidagene\/","title":{"rendered":"HuidaGene: verso la sperimentazione clinica"},"content":{"rendered":"

Nel febbraio 2024, David Covini, presidente di DupMECP2, ha visitato la societ\u00e0 biotecnologica HuidaGene Therapeutics a Shanghai. La visita \u00e8 stata un'occasione unica per incontrare un team dinamico il cui candidato clinico potrebbe rivoluzionare il futuro del trattamento della sindrome da duplicazione del gene MECP2 (MDS). <\/p>\n\n\n\n

Guidata dal Dr. Alvin Luk, CEO di HuidagGene, l'azienda \u00e8 all'avanguardia nello sviluppo di una tecnologia rivoluzionaria che potrebbe cambiare la vita dei bambini affetti da SMD. La visita \u00e8 stata un'incredibile opportunit\u00e0 per scoprire le strutture all'avanguardia e l'ambiente creativo che supporta questa ricerca. In particolare, lo spazio dell'ufficio comprende anche una parete da arrampicata, che favorisce un'atmosfera di lavoro creativa. <\/p>\n\n\n\n

\"\"<\/figure>\n\n\n\n

Alvin e il suo direttore medico per le SMD, la dottoressa Gloria Tang, hanno presentato i dettagli della tecnologia di editing dell'RNA CRISPR CAS13 per la sindrome da duplicazione genica. MECP2<\/em>denominato HG204. La qualit\u00e0 promettente dei dati e i risultati preliminari degli studi di tossicit\u00e0 sui primati non umani hanno portato HuidaGene a uno sviluppo entusiasmante. La sperimentazione clinica dell'azienda, il cui inizio \u00e8 inizialmente previsto per la seconda met\u00e0 del 2024, ha gi\u00e0 ricevuto l'approvazione del comitato etico di due ospedali, con l'obiettivo di includere pazienti affetti da SMD entro la fine dell'anno.<\/p>\n\n\n\n

Si tratta di un'ottima notizia per la comunit\u00e0 delle SMD. L'imminente sperimentazione clinica di HG204 per le SMD, denominata \"HERO\", prevede inizialmente il trattamento di almeno 4 pazienti che saranno monitorati attentamente per un anno. La prima fase di questo studio innovativo sar\u00e0 condotta in Cina. L'inclusione di altri pazienti in Cina dipender\u00e0 dai primi risultati preliminari dello studio interventistico.<\/p>\n\n\n\n

HuidaGene ha gi\u00e0 compiuto progressi significativi verso l'approvazione normativa, ottenendo due designazioni della FDA (Rare Paediatric Disease Designation (RPDD) e Orphan Drug Designation (ODD)) per HG204. Ha inoltre avviato contatti con la FDA e l'EMA per ulteriori studi negli Stati Uniti e in Europa. <\/p>\n\n\n\n

\u00c8 un privilegio aver avuto l'opportunit\u00e0 di incontrare Alvin e Gloria di persona. Siamo entusiasti di far parte di questa avventura e non vediamo l'ora di tenere informata la nostra comunit\u00e0 sui progressi di questa tecnologia che potrebbe cambiare la vita.<\/p>\n\n\n\n

<\/p>","protected":false},"excerpt":{"rendered":"

En f\u00e9vrier 2024, David Covini, pr\u00e9sident de DupMECP2, a visit\u00e9 l’entreprise de biotechnologie HuidaGene Therapeutics \u00e0 Shanghai. Cette visite a \u00e9t\u00e9 une occasion unique de rencontrer une \u00e9quipe dynamique dont […]<\/p>","protected":false},"author":1,"featured_media":25367,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[7],"tags":[],"class_list":["post-25235","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-actualite"],"yoast_head":"\nHuidaGene: vers les essais cliniques - DupMECP2<\/title>\n<meta name=\"description\" content=\"En f\u00e9vrier 2024, David Covini, pr\u00e9sident de DupMECP2, a visit\u00e9 l'entreprise de biotechnologie HuidaGene Therapeutics \u00e0 Shanghai. 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