{"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-eine-vielversprechende-crispr-cas13-technologie-aus-china-shanghai","status":"publish","type":"post","link":"https:\/\/dupmecp2.eu\/de\/huidagene-eine-vielversprechende-crispr-cas13-technologie-aus-china-shanghai\/","title":{"rendered":"Huidagene: Eine vielversprechende CRISPR-CAS13-Technologie aus China (Shanghai)"},"content":{"rendered":"

Huidagene Therapeutics ist ein Biotechnologieunternehmen mit Sitz in Shanghai und New Jersey, das sich auf die Entdeckung, Konstruktion und Entwicklung neuer Medikamente auf der Grundlage der CRISPR-Technologie konzentriert. Das Unternehmen konzentriert sich auf muskul\u00e4re, ophthalmische und neurologische Erkrankungen.<\/p>\n\n\n\n

Huidagene hat HG204 entwickelt, einen Adeno-assoziierten viralen Vektor (AAV), der einen innovativen CRISPR-Cas13-Komplex in die Zellen einbringt. Er wurde speziell f\u00fcr das MECP2-Genduplikationssyndrom entwickelt, HG204 zielt darauf ab, die \u00dcberproduktion des MeCP2-Proteins zu regulieren, indem es den Abbau \u00fcbersch\u00fcssiger MECP2-RNA induziert.<\/strong> Dieser innovative Arzneimittelkandidat hat das Potenzial, das MECP2-Genduplikationssyndrom mit einer einzigen Verabreichung zu heilen.<\/p>\n\n\n\n

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

Unsere Organisation f\u00fchrte produktive Gespr\u00e4che mit den F\u00fchrungskr\u00e4ften von Huidagene, um mehr \u00fcber diese innovative Technologie und ihre Bedeutung f\u00fcr die Zukunft zu erfahren. Vorklinische Studien mit HG204 in einem humanisierten Mausmodell zeigten eine signifikante Reduktion des MeCP2-Proteins und eine Umkehrung der Symptome.<\/strong> Abgesehen davon, dass diese Ergebnisse ermutigend sind, handelt es sich auch um den zweiten Beweis f\u00fcr die Umkehrbarkeit der Symptome des Syndroms nach Regulierung der MECP2-Proteinspiegel (wie zuvor durch die Verabreichung von ASO durch Prof. H. Zoghbi nachgewiesen).<\/p>\n\n\n\n

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Huidagene schlie\u00dft derzeit die pr\u00e4klinischen Studien zu HG204 ab und bereitet sich auf eine klinische Studie f\u00fcr Patienten in China vor, die im Sommer 2024 beginnen soll. <\/strong>Wenn die Studie erfolgreich ist, plant Huidagene, in den Jahren 2025-2026 klinische Studien in anderen L\u00e4ndern, wie den USA und Europa, durchzuf\u00fchren.<\/p>\n\n\n\n


Am 31. Oktober erteilte die US-amerikanische Gesundheitsbeh\u00f6rde FDA (Food and Drug Administration) HG204 die Bezeichnungen seltene p\u00e4diatrische Krankheit und Arzneimittel f\u00fcr seltene Leiden (Orphan Drug). Diese beh\u00f6rdliche Bezeichnung ist ein erster Schritt auf dem Weg zu einer klinischen Studie au\u00dferhalb Chinas und wird eine beschleunigte Pr\u00fcfung des Dossiers bei der Zulassung des Medikaments erm\u00f6glichen. Obwohl die Nachricht v\u00f6llig unerwartet kommt, bringt sie der Gemeinschaft zus\u00e4tzliche Hoffnung.<\/p>\n\n\n\n

Unser Team wird mit Huidagene in Kontakt bleiben und unsere Gemeinschaft \u00fcber alle neuen Entwicklungen auf dem Laufenden halten.<\/p>\n\n\n\n

\"\"<\/figure>\n\n\n\n
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Zum Pressespiegel<\/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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