{"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\/pl\/hg204-huidagene\/","title":{"rendered":"Huidagene: obiecuj\u0105ca technologia CRISPR-CAS13 z Szanghaju (Chiny)"},"content":{"rendered":"<p>Huidagene Therapeutics to firma biotechnologiczna z siedzib\u0105 w Szanghaju i New Jersey, koncentruj\u0105ca si\u0119 na odkrywaniu, in\u017cynierii i opracowywaniu nowych lek\u00f3w opartych na CRISPR. Firma koncentruje si\u0119 na zaburzeniach mi\u0119\u015bniowych, okulistycznych i neurologicznych.<\/p>\n\n\n\n<p>Firma Huidagene opracowa\u0142a HG204, wektor adenowirusowy (AAV) dostarczaj\u0105cy nowy kompleks CRISPR-Cas13 do kom\u00f3rek. Zaprojektowany dla zespo\u0142u duplikacji MECP2, <strong>HG204 ma na celu regulacj\u0119 nadprodukcji poziomu bia\u0142ka MeCP2 poprzez indukcj\u0119 degradacji nadeksprymowanego RNA MECP2.<\/strong> Ten innowacyjny kandydat na lek ma potencja\u0142 do wyleczenia MDS poprzez pojedyncze podanie.<\/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 owocne rozmowy z liderami Huidagene, aby dowiedzie\u0107 si\u0119 wi\u0119cej o tej innowacyjnej technologii i jej znaczeniu dla przysz\u0142o\u015bci. <strong>Badania przedkliniczne z u\u017cyciem HG204 w humanizowanym mysim modelu MDS wykaza\u0142y znaczn\u0105 redukcj\u0119 bia\u0142ka MeCP2 i odwr\u00f3cenie objaw\u00f3w.<\/strong> Chocia\u017c s\u0105 to zach\u0119caj\u0105ce wyniki, jest to r\u00f3wnie\u017c drugi dow\u00f3d na odwracalno\u015b\u0107 objaw\u00f3w MDS po regulacji poziomu bia\u0142ka MECP2 (jak wcze\u015bniej wykazano po podaniu ASO przez prof. H. Zoghbi i zesp\u00f3\u0142).<\/p>\n\n\n\n<div style=\"height:25px\" aria-hidden=\"true\" class=\"wp-block-spacer\"><\/div>\n\n\n\n<p><strong>Huidagene jest w trakcie finalizowania bada\u0144 przedklinicznych z HG204 i przygotowuje si\u0119 do bada\u0144 klinicznych dla pacjent\u00f3w w Chinach, kt\u00f3rych rozpocz\u0119cie zaplanowano na po\u0142ow\u0119 2024 roku. <\/strong>Je\u015bli ta pr\u00f3ba zako\u0144czy si\u0119 sukcesem, Huidagene zaplanuje badania kliniczne w innych krajach, takich jak USA i Europa w latach 2025-2026.<\/p>\n\n\n\n<p><br>31 pa\u017adziernika FDA (Agencja ds. \u017bywno\u015bci i Lek\u00f3w, organ ds. zdrowia w USA) przyzna\u0142a HG204 oznaczenie rzadkiej choroby pediatrycznej i sierocego produktu leczniczego. To oznaczenie regulacyjne jest pierwszym krokiem w kierunku bada\u0144 klinicznych poza Chinami i umo\u017cliwi przyspieszony przegl\u0105d dokumentacji po rejestracji leku. Cho\u0107 wiadomo\u015b\u0107 ta by\u0142a ca\u0142kowicie nieoczekiwana, daje ona spo\u0142eczno\u015bci dodatkow\u0105 nadziej\u0119.<\/p>\n\n\n\n<p>Nasz zesp\u00f3\u0142 pozostanie w kontakcie z Huidagene i b\u0119dzie na bie\u017c\u0105co informowa\u0142 nasz\u0105 spo\u0142eczno\u015b\u0107 o wszelkich nowych wydarzeniach.<\/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\">Do komunikatu prasowego<\/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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