{"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-obiecujaca-technologia-crispr-cas13-z-chinskiego-szanghaju","status":"publish","type":"post","link":"https:\/\/dupmecp2.eu\/pl\/huidagene-obiecujaca-technologia-crispr-cas13-z-chinskiego-szanghaju\/","title":{"rendered":"Huidagene: obiecuj\u0105ca technologia CRISPR-CAS13 z Chin (Szanghaj)"},"content":{"rendered":"<p class=\"wp-block-paragraph\">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 technologii CRISPR. Firma koncentruje si\u0119 na zaburzeniach mi\u0119\u015bniowych, okulistycznych i neurologicznych.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Firma Huidagene opracowa\u0142a HG204, wektor wirusa adenowirusowego (AAV) dostarczaj\u0105cy innowacyjny kompleks CRISPR-Cas13 do kom\u00f3rek. Zaprojektowany specjalnie dla zespo\u0142u duplikacji genu MECP2, <strong>HG204 ma na celu regulacj\u0119 nadprodukcji bia\u0142ka MeCP2 poprzez indukowanie degradacji nadmiaru RNA MECP2.<\/strong> Ten innowacyjny kandydat na lek mo\u017ce potencjalnie leczy\u0107 zesp\u00f3\u0142 duplikacji genu MECP2 za pomoc\u0105 jednego podania.<\/p>\n\n\n\n<div style=\"height:25px\" aria-hidden=\"true\" class=\"wp-block-spacer\"><\/div>\n\n\n\n<p class=\"wp-block-paragraph\">Nasza organizacja przeprowadzi\u0142a owocne rozmowy z kierownictwem Huidagene, aby dowiedzie\u0107 si\u0119 wi\u0119cej o tej innowacyjnej technologii i jej przysz\u0142o\u015bci. <strong>Badania przedkliniczne z u\u017cyciem HG204 w humanizowanym modelu mysim wykaza\u0142y znacz\u0105ce zmniejszenie bia\u0142ka MeCP2 i odwr\u00f3cenie objaw\u00f3w.<\/strong> Opr\u00f3cz tego, \u017ce wyniki te s\u0105 zach\u0119caj\u0105ce, jest to r\u00f3wnie\u017c drugi dow\u00f3d na odwracalno\u015b\u0107 objaw\u00f3w zespo\u0142u po regulacji poziomu bia\u0142ka MECP2 (jak wcze\u015bniej wykazano po podaniu ASO przez prof. H. Zoghbi).<\/p>\n\n\n\n<div style=\"height:25px\" aria-hidden=\"true\" class=\"wp-block-spacer\"><\/div>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Huidagene finalizuje obecnie badania przedkliniczne nad HG204 i przygotowuje si\u0119 do badania klinicznego dla pacjent\u00f3w w Chinach, kt\u00f3re ma rozpocz\u0105\u0107 si\u0119 latem 2024 roku. <\/strong>Je\u015bli badanie to oka\u017ce si\u0119 rozstrzygaj\u0105ce, Huidagene planuje przeprowadzi\u0107 badania kliniczne w innych krajach, takich jak Stany Zjednoczone i Europa, w latach 2025-2026.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><br>W dniu 31 pa\u017adziernika Ameryka\u0144ska Agencja ds. \u017bywno\u015bci i Lek\u00f3w (FDA) 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 zarejestrowaniu leku. Chocia\u017c wiadomo\u015b\u0107 ta jest ca\u0142kowicie nieoczekiwana, przynosi spo\u0142eczno\u015bci dodatkow\u0105 nadziej\u0119.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Nasz zesp\u00f3\u0142 pozostanie w kontakcie z Huidagene i b\u0119dzie 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 przegl\u0105du prasy<\/a><\/div>\n<\/div>","protected":false},"excerpt":{"rendered":"<p>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&rsquo;ing\u00e9nierie et le d\u00e9veloppement de nouveaux m\u00e9dicaments bas\u00e9s sur [&hellip;]<\/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":"<!-- This site is optimized with the Yoast SEO plugin v25.8 - https:\/\/yoast.com\/wordpress\/plugins\/seo\/ -->\n<title>Huidagene : 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. Le candidat-m\u00e9dicament HG204 a d\u00e9montr\u00e9 une r\u00e9duction significative de la prot\u00e9ine MeCP2 et la r\u00e9versibilit\u00e9 des sympt\u00f4mes.\" \/>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/dupmecp2.eu\/pl\/huidagene-obiecujaca-technologia-crispr-cas13-z-chinskiego-szanghaju\/\" \/>\n<meta property=\"og:locale\" content=\"pl_PL\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Huidagene : Une technologie CRISPR-CAS13 prometteuse venue de Chine (Shanghai) - DupMECP2\" \/>\n<meta property=\"og:description\" content=\"Une nouvelle perspective potentielle pour le traitement du syndrome de duplication MECP2 avec le CRIPSR-Cas13 de Huidagene. Le candidat-m\u00e9dicament HG204 a d\u00e9montr\u00e9 une r\u00e9duction significative de la prot\u00e9ine MeCP2 et la r\u00e9versibilit\u00e9 des sympt\u00f4mes.\" \/>\n<meta property=\"og:url\" content=\"https:\/\/dupmecp2.eu\/pl\/huidagene-obiecujaca-technologia-crispr-cas13-z-chinskiego-szanghaju\/\" \/>\n<meta property=\"og:site_name\" content=\"DupMECP2\" \/>\n<meta property=\"article:published_time\" content=\"2023-12-16T21:12:50+00:00\" \/>\n<meta property=\"article:modified_time\" content=\"2023-12-20T18:46:25+00:00\" \/>\n<meta property=\"og:image\" content=\"https:\/\/dupmecp2.eu\/wp-content\/uploads\/2023\/12\/Huidagene-Logo.png\" \/>\n\t<meta property=\"og:image:width\" content=\"318\" \/>\n\t<meta property=\"og:image:height\" content=\"112\" \/>\n\t<meta property=\"og:image:type\" content=\"image\/png\" \/>\n<meta name=\"author\" content=\"Caroline\" \/>\n<meta name=\"twitter:card\" content=\"summary_large_image\" \/>\n<meta name=\"twitter:label1\" content=\"Napisane przez\" \/>\n\t<meta name=\"twitter:data1\" content=\"Caroline\" \/>\n\t<meta name=\"twitter:label2\" content=\"Szacowany czas czytania\" \/>\n\t<meta name=\"twitter:data2\" content=\"2 minuty\" \/>\n<script type=\"application\/ld+json\" class=\"yoast-schema-graph\">{\"@context\":\"https:\/\/schema.org\",\"@graph\":[{\"@type\":\"Article\",\"@id\":\"https:\/\/dupmecp2.eu\/huidagene-une-technologie-crispr-cas13-prometteuse-venue-de-chine-shanghai\/#article\",\"isPartOf\":{\"@id\":\"https:\/\/dupmecp2.eu\/huidagene-une-technologie-crispr-cas13-prometteuse-venue-de-chine-shanghai\/\"},\"author\":{\"name\":\"Caroline\",\"@id\":\"https:\/\/dupmecp2.eu\/#\/schema\/person\/b41e8cbba153cd974ee340386af279ee\"},\"headline\":\"Huidagene : Une technologie CRISPR-CAS13 prometteuse venue de Chine (Shanghai)\",\"datePublished\":\"2023-12-16T21:12:50+00:00\",\"dateModified\":\"2023-12-20T18:46:25+00:00\",\"mainEntityOfPage\":{\"@id\":\"https:\/\/dupmecp2.eu\/huidagene-une-technologie-crispr-cas13-prometteuse-venue-de-chine-shanghai\/\"},\"wordCount\":400,\"commentCount\":0,\"publisher\":{\"@id\":\"https:\/\/dupmecp2.eu\/#organization\"},\"image\":{\"@id\":\"https:\/\/dupmecp2.eu\/huidagene-une-technologie-crispr-cas13-prometteuse-venue-de-chine-shanghai\/#primaryimage\"},\"thumbnailUrl\":\"https:\/\/dupmecp2.eu\/wp-content\/uploads\/2023\/12\/Huidagene-Logo.png\",\"articleSection\":[\"Actualit\u00e9\"],\"inLanguage\":\"pl-PL\",\"potentialAction\":[{\"@type\":\"CommentAction\",\"name\":\"Comment\",\"target\":[\"https:\/\/dupmecp2.eu\/huidagene-une-technologie-crispr-cas13-prometteuse-venue-de-chine-shanghai\/#respond\"]}]},{\"@type\":\"WebPage\",\"@id\":\"https:\/\/dupmecp2.eu\/huidagene-une-technologie-crispr-cas13-prometteuse-venue-de-chine-shanghai\/\",\"url\":\"https:\/\/dupmecp2.eu\/huidagene-une-technologie-crispr-cas13-prometteuse-venue-de-chine-shanghai\/\",\"name\":\"Huidagene : Une technologie CRISPR-CAS13 prometteuse venue de Chine (Shanghai) - DupMECP2\",\"isPartOf\":{\"@id\":\"https:\/\/dupmecp2.eu\/#website\"},\"primaryImageOfPage\":{\"@id\":\"https:\/\/dupmecp2.eu\/huidagene-une-technologie-crispr-cas13-prometteuse-venue-de-chine-shanghai\/#primaryimage\"},\"image\":{\"@id\":\"https:\/\/dupmecp2.eu\/huidagene-une-technologie-crispr-cas13-prometteuse-venue-de-chine-shanghai\/#primaryimage\"},\"thumbnailUrl\":\"https:\/\/dupmecp2.eu\/wp-content\/uploads\/2023\/12\/Huidagene-Logo.png\",\"datePublished\":\"2023-12-16T21:12:50+00:00\",\"dateModified\":\"2023-12-20T18:46:25+00:00\",\"description\":\"Une nouvelle perspective potentielle pour le traitement du syndrome de duplication MECP2 avec le CRIPSR-Cas13 de Huidagene. 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