{"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\/de\/hg204-huidagene\/","title":{"rendered":"Huidagene: Eine vielversprechende CRISPR-CAS13-Technologie aus Shanghai (China)"},"content":{"rendered":"<p>Huidagene Therapeutics ist ein in Shanghai und New Jersey ans\u00e4ssiges Biotechnologieunternehmen, das sich auf die Entdeckung, Konstruktion und Entwicklung neuartiger CRISPR-basierter Medikamente konzentriert. Das Unternehmen konzentriert sich auf muskul\u00e4re, ophthalmische und neurologische Erkrankungen.<\/p>\n\n\n\n<p>Huidagene hat HG204 entwickelt, einen adeno-assoziierten viralen (AAV) Vektor, der einen neuartigen CRISPR-Cas13-Komplex in Zellen liefert. Entwickelt f\u00fcr das MECP2-Duplikationssyndrom, <strong>HG204 zielt darauf ab, die \u00dcberproduktion des MeCP2-Proteinspiegels zu regulieren, indem es den Abbau der \u00fcberexprimierten MECP2-RNA induziert.<\/strong> Dieser innovative Wirkstoffkandidat besitzt das Potenzial, MDS durch eine einmalige Verabreichung zu heilen.<\/p>\n\n\n\n<div style=\"height:25px\" aria-hidden=\"true\" class=\"wp-block-spacer\"><\/div>\n\n\n\n<p>Unsere Organisation hatte 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. <strong>Vorklinische Studien mit HG204 in einem humanisierten MDS-Mausmodell demonstrierten eine signifikante Reduktion des MeCP2-Proteins und eine Umkehrung der Symptome.<\/strong> While these are encouraging results, this is also the second proof of the reversibility of MDS symptoms after the regulation of MECP2 protein levels (as previously demonstrated by the administration of the ASO by 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 ist dabei, vorklinische Studien mit HG204 abzuschlie\u00dfen und f\u00fcr eine klinische Studie f\u00fcr Patienten in China bereit zu werden, die voraussichtlich Mitte 2024 beginnen wird. <\/strong>Wenn dieser Versuch erfolgreich ist, wird Huidagene in den Jahren 2025-2026 klinische Versuche in anderen L\u00e4ndern wie den USA und Europa planen.<\/p>\n\n\n\n<p><br>Am 31. Oktober erteilte die FDA (Food and Drug Administration, die US-Gesundheitsbeh\u00f6rde) HG204 sowohl die Bezeichnung Rare Pediatric Disease als auch Orphan Drug Designation. Diese Zulassung ist ein erster Schritt auf dem Weg zu einer klinischen Studie au\u00dferhalb Chinas und erm\u00f6glicht eine beschleunigte \u00dcberpr\u00fcfung der Unterlagen nach der Registrierung des Medikaments. Auch wenn die Nachricht v\u00f6llig unerwartet kam, bringt sie der Gemeinschaft zus\u00e4tzliche Hoffnung.<\/p>\n\n\n\n<p>Unser Team wird mit Huidagene in Kontakt bleiben und unsere Community \u00fcber alle neuen Entwicklungen auf dem Laufenden halten.<\/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\">To press release<\/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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