{"id":22138,"date":"2023-12-16T21:12:50","date_gmt":"2023-12-16T21:12:50","guid":{"rendered":"https:\/\/dupmecp2.eu\/?p=22138"},"modified":"2023-12-20T18:44:05","modified_gmt":"2023-12-20T18:44:05","slug":"hg204-huidagene","status":"publish","type":"post","link":"https:\/\/dupmecp2.eu\/it\/hg204-huidagene\/","title":{"rendered":"Huidagene: una promettente tecnologia CRISPR-CAS13 da Shanghai (Cina)"},"content":{"rendered":"<p>Huidagene Therapeutics \u00e8 un'azienda biotecnologica con sede a Shanghai e nel New Jersey, focalizzata sulla scoperta, l'ingegnerizzazione e lo sviluppo di nuovi farmaci basati su CRISPR. L'azienda si concentra sui disturbi muscolari, oftalmici e neurologici.<\/p>\n\n\n\n<p>Huidagene ha sviluppato HG204, un vettore virale adeno-associato (AAV) che trasporta nelle cellule un nuovo complesso CRISPR-Cas13. Progettato per la sindrome da duplicazione MECP2, <strong>HG204 mira a regolare la sovrapproduzione dei livelli di proteina MeCP2 inducendo la degradazione dell'RNA di MECP2 sovraespresso.<\/strong> Questo candidato farmaco innovativo ha il potenziale di curare le SMD con un'unica somministrazione.<\/p>\n\n\n\n<div style=\"height:25px\" aria-hidden=\"true\" class=\"wp-block-spacer\"><\/div>\n\n\n\n<p>La nostra organizzazione ha avuto colloqui produttivi con i leader di Huidagene per saperne di pi\u00f9 su questa tecnologia innovativa e su ci\u00f2 che significa per il futuro. <strong>Gli studi preclinici condotti con HG204 in un modello murino umanizzato di MDS hanno dimostrato una riduzione significativa della proteina MeCP2 e un'inversione dei sintomi.<\/strong> Sebbene si tratti di risultati incoraggianti, questa \u00e8 anche la seconda prova della reversibilit\u00e0 dei sintomi della SMD dopo la regolazione dei livelli della proteina MECP2 (come precedentemente dimostrato dalla somministrazione dell'ASO da parte del Prof. H. Zoghbi &amp; Team).<\/p>\n\n\n\n<div style=\"height:25px\" aria-hidden=\"true\" class=\"wp-block-spacer\"><\/div>\n\n\n\n<p><strong>Huidagene sta ultimando gli studi preclinici con HG204 e si sta preparando per una sperimentazione clinica su pazienti in Cina, il cui inizio \u00e8 previsto per la met\u00e0 del 2024. <\/strong>Se questa sperimentazione avr\u00e0 successo, Huidagene pianificher\u00e0 studi clinici in altri Paesi, come gli Stati Uniti e l'Europa, nel 2025-2026.<\/p>\n\n\n\n<p><br>Il 31 ottobre, la FDA (Food and Drug Administration, l'autorit\u00e0 sanitaria degli Stati Uniti) ha concesso a HG204 la designazione di farmaco orfano e di malattia pediatrica rara. Questa designazione normativa \u00e8 un primo passo verso una sperimentazione clinica al di fuori della Cina e consentir\u00e0 una revisione accelerata del dossier al momento della registrazione del farmaco. La notizia era del tutto inaspettata, ma porta ulteriore speranza alla comunit\u00e0.<\/p>\n\n\n\n<p>Il nostro team rimarr\u00e0 in contatto con Huidagene e terr\u00e0 aggiornata la nostra comunit\u00e0 su eventuali nuovi sviluppi.<\/p>\n\n\n\n<figure class=\"wp-block-image size-large is-resized\"><img decoding=\"async\" data-src=\"https:\/\/dupmecp2.eu\/wp-content\/uploads\/2023\/12\/Huidagene_Pipeline_2023_white-edited.png\" alt=\"\" class=\"wp-image-22144 lazyload\" style=\"--smush-placeholder-width: 1920px; --smush-placeholder-aspect-ratio: 1920\/1080;width:840px;height:auto\" src=\"data:image\/svg+xml;base64,PHN2ZyB3aWR0aD0iMSIgaGVpZ2h0PSIxIiB4bWxucz0iaHR0cDovL3d3dy53My5vcmcvMjAwMC9zdmciPjwvc3ZnPg==\" \/><\/figure>\n\n\n\n<div class=\"wp-block-buttons is-layout-flex wp-block-buttons-is-layout-flex\">\n<div class=\"wp-block-button\"><a class=\"wp-block-button__link has-background wp-element-button\" href=\"https:\/\/www.huidagene.com\/new\/news\/48\" style=\"border-radius:16px;background-color:#f7a13c\" target=\"_blank\" rel=\"noreferrer noopener\">Al comunicato stampa<\/a><\/div>\n<\/div>","protected":false},"excerpt":{"rendered":"<p>Huidagene Therapeutics is a biotechnology company based in Shanghai and New Jersey, focusing on discovering, engineering, and developing novel CRISPR-based medicines. The company focuses on muscular, ophthalmic and neurological disorders. [&hellip;]<\/p>","protected":false},"author":1,"featured_media":22150,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[87],"tags":[],"class_list":["post-22138","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-news"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v25.8 - https:\/\/yoast.com\/wordpress\/plugins\/seo\/ -->\n<title>Huidagene: A promising CRISPR-CAS13 technology from Shanghai (China) - DupMECP2<\/title>\n<meta name=\"description\" content=\"A potential new perspective for the treatment of MECP2 duplication syndrome with Huidagene&#039;s CRIPSR-Cas13. 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