{"id":22179,"date":"2023-12-16T21:12:50","date_gmt":"2023-12-16T21:12:50","guid":{"rendered":"https:\/\/dupmecp2.eu\/huidagene-eine-vielversprechende-crispr-cas13-technologie-aus-shanghai-china\/"},"modified":"2023-12-20T18:46:19","modified_gmt":"2023-12-20T18:46:19","slug":"huidagene-eine-vielversprechende-crispr-cas13-technologie-aus-shanghai-china","status":"publish","type":"post","link":"https:\/\/dupmecp2.eu\/pl\/huidagene-eine-vielversprechende-crispr-cas13-technologie-aus-shanghai-china\/","title":{"rendered":"Huidagene: Zaawansowana technologia CRISPR-CAS13 z Szanghaju (Chiny)"},"content":{"rendered":"<p>Huidagene Therapeutics to firma biotechnologiczna z siedzib\u0105 w Szanghaju i New Jersey, kt\u00f3ra koncentruje si\u0119 na opracowywaniu, in\u017cynierii i rozwoju nowych lek\u00f3w opartych na CRISPR. Firma koncentruje si\u0119 na chorobach mi\u0119\u015bni, oczu i zaburzeniach neurologicznych.<\/p>\n\n\n\n<p>Huidagene opracowa\u0142 HG204, adeno-assoziierten viralen (AAV) Vektor, kt\u00f3ry wprowadza nowy kompleks CRISPR-Cas13 do kom\u00f3rek. <strong>HG204 zosta\u0142 opracowany w celu leczenia zespo\u0142u duplikacji MECP2 i umo\u017cliwia regulacj\u0119 produkcji bia\u0142ek MeCP2, a tak\u017ce indukuje abbau \u00fcberexprimierten MECP2-RNA. Ten innowacyjny kandydat na lek mo\u017ce potencjalnie leczy\u0107 MDS za pomoc\u0105 pojedynczej terapii.<\/strong><\/p>\n\n\n\n<div style=\"height:25px\" aria-hidden=\"true\" class=\"wp-block-spacer\"><\/div>\n\n\n\n<p>Nasza organizacja przeprowadzi\u0142a produktywne rozmowy z wiod\u0105cymi firmami Huidagene, aby dowiedzie\u0107 si\u0119 wi\u0119cej o tej innowacyjnej technologii i jej znaczeniu dla przysz\u0142o\u015bci. <strong>Badania kliniczne z u\u017cyciem HG204 na ludzkim modelu MDS wykaza\u0142y znaczn\u0105 redukcj\u0119 bia\u0142ek MeCP2 i z\u0142agodzenie objaw\u00f3w.<\/strong> Te wyniki s\u0105 jednoznaczne, poniewa\u017c dotycz\u0105 one r\u00f3wnie\u017c nowego nasilenia objaw\u00f3w MDS po regulacji bia\u0142ka MECP2 (jak to mia\u0142o miejsce wcze\u015bniej podczas weryfikacji ASO przez 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 ko\u0144czy obecnie badania kliniczne nad HG204 i rozpoczyna badanie kliniczne dla pacjent\u00f3w w Chinach, kt\u00f3re rozpocznie si\u0119 we wrze\u015bniu 2024 roku.<\/strong> Je\u015bli badanie zako\u0144czy si\u0119 sukcesem, nale\u017cy przeprowadzi\u0107 badania kliniczne w latach 2025-2026 w innych krajach, takich jak USA i Europa.<\/p>\n\n\n\n<p><br>W dniu 31. Oktober hat die FDA (Food and Drug Administration, die Gesundheitsbeh\u00f6rde der USA) sowoith die Auszeichnung as seltene p\u00e4diatrische Erkrankung as auch den Orphan-Drug-Status f\u00fcr HG204 verliehen. Ten behawioralny wniosek jest pierwszym krokiem na drodze do bada\u0144 klinicznych w Chinach i mo\u017ce pom\u00f3c w ulepszeniu dokumentacji podczas rejestracji. Mimo, \u017ce wiadomo\u015b\u0107 by\u0142a w pe\u0142ni niezrozumia\u0142a, przynios\u0142a ona wsp\u00f3lnocie dodatkowe zadowolenie.<\/p>\n\n\n\n<p>Nasz zesp\u00f3\u0142 po\u0142\u0105czy si\u0119 z Huidagene, a nasza spo\u0142eczno\u015b\u0107 b\u0119dzie mia\u0142a dost\u0119p do wszystkich nowych przedsi\u0119wzi\u0119\u0107.<\/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\">Zur Pressemitteilung<\/a><\/div>\n<\/div>","protected":false},"excerpt":{"rendered":"<p>Huidagene Therapeutics ist ein Biotechnologieunternehmen mit Sitz in Shanghai und New Jersey, das sich auf die Entdeckung, das Engineering und die Entwicklung neuartiger CRISPR-basierter Medikamente konzentriert. Das Unternehmen konzentriert sich [&hellip;]<\/p>","protected":false},"author":1,"featured_media":22152,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[57],"tags":[],"class_list":["post-22179","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-aktualitat"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v25.8 - https:\/\/yoast.com\/wordpress\/plugins\/seo\/ -->\n<title>Huidagene: Eine vielversprechende CRISPR-CAS13 Technologie aus Shanghai (China) - DupMECP2<\/title>\n<meta name=\"description\" content=\"Eine m\u00f6gliche neue Perspektive f\u00fcr die Behandlung des MECP2-Duplikationssyndroms mit CRIPSR-Cas13 von Huidagene. 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