{"id":22829,"date":"2023-12-16T21:12:50","date_gmt":"2023-12-16T21:12:50","guid":{"rendered":"https:\/\/dupmecp2.eu\/huidagene-una-promettente-tecnologia-crispr-cas13-da-shanghai-cina\/"},"modified":"2024-01-31T12:43:10","modified_gmt":"2024-01-31T12:43:10","slug":"huidagene-una-prometedora-tecnologia-crispr-cas13-de-shanghai-china","status":"publish","type":"post","link":"https:\/\/dupmecp2.eu\/es\/huidagene-una-prometedora-tecnologia-crispr-cas13-de-shanghai-china\/","title":{"rendered":"Huidagene: una prometedora tecnolog\u00eda CRISPR-CAS13 de Shanghai (China)"},"content":{"rendered":"<p>Huidagene Therapeutics es una empresa biotecnol\u00f3gica con sede en Shangai y Nueva Jersey, dedicada a la b\u00fasqueda y el desarrollo de nuevos medicamentos basados en la tecnolog\u00eda CRISPR. La empresa se centra en enfermedades musculares, oftalmol\u00f3gicas y neurol\u00f3gicas.<\/p>\n\n<p>Huidagene ha desarrollado HG204, un vector viral asociado a un adenovirus (AAV) que rilascia un innovador complesso CRISPR-Cas13 nelle cellule. Ha sido dise\u00f1ado para el s\u00edndrome de duplicaci\u00f3n del gen MECP2, <strong>HG204 regula la producci\u00f3n de la prote\u00edna MeCP2 induciendo la degradaci\u00f3n del ARN MECP2 en exceso.<\/strong> Este innovador f\u00e1rmaco candidato tiene el potencial de curar el S\u00edndrome de Duplicaci\u00f3n del gen MECP2 con una sola administraci\u00f3n.<\/p>\n\n<div style=\"height:25px\" aria-hidden=\"true\" class=\"wp-block-spacer\"><\/div>\n\n<p>Nuestra organizaci\u00f3n ha mantenido fruct\u00edferas conversaciones con los directivos de Huidagene para profundizar en el conocimiento de esta innovadora tecnolog\u00eda y averiguar qu\u00e9 significa para el futuro. <strong>Estudios precl\u00ednicos que utilizaron HG204 en un modelo de topo umanizado han demostrado una reducci\u00f3n significativa de la prote\u00edna MeCP2 y una inversi\u00f3n de los s\u00edntomas.<\/strong> Adem\u00e1s del hecho de que estos resultados son incorregibles, representan tambi\u00e9n la segunda prueba de la reversibilidad de los s\u00edntomas del s\u00edndrome si se interviene en la regulaci\u00f3n de los niveles de prote\u00edna MECP2 (como ya hab\u00edan demostrado anteriormente la Prof. H. Zoghbi y su equipo mediante la administraci\u00f3n de un ASO).<\/p>\n\n<div style=\"height:25px\" aria-hidden=\"true\" class=\"wp-block-spacer\"><\/div>\n\n<p><strong>Huidagene est\u00e1 concluyendo actualmente los estudios precl\u00ednicos en HG204 y se est\u00e1 preparando para un ensayo cl\u00ednico en pacientes en China, que deber\u00eda comenzar en 2024. <\/strong>Si este estudio resulta positivo, Huidagene tiene previsto realizar ensayos cl\u00ednicos en otros pa\u00edses, como Estados Unidos y Europa, en 2025-2026.<\/p>\n\n<p><br\/>El 31 de octubre, la FDA (Food and Drug Administration, autoridad sanitaria de Estados Unidos) concedi\u00f3 a HG204 la designaci\u00f3n sia di farmaco per malattia pediatrica rara che di farmaco orfano. Esta designaci\u00f3n reglamentaria representa un primer paso hacia un estudio cl\u00ednico fuera de China, y permitir\u00e1 una revisi\u00f3n acelerada del expediente durante el proceso de registro del medicamento. Aunque la notificaci\u00f3n no haya sido atendida, dar\u00e1 a la comunidad un nuevo motivo de esperanza.<\/p>\n\n<p>Nuestro equipo se pondr\u00e1 en contacto con Huidagene y mantendr\u00e1 informada a nuestra comunidad sobre cualquier nuevo avance.<\/p>\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<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\">Vai al comunicato stampa<\/a><\/div>\n<\/div>","protected":false},"excerpt":{"rendered":"<p>Huidagene Therapeutics \u00e8 un&rsquo;azienda biotecnologica basata a Shangai e New Jersey, attiva nella scoperta e sviluppo di nuove medicine basate sulla tecnologia CRISPR. L&rsquo;azienda si focalizza su malattie muscolari, oftalmiche [&hellip;]<\/p>","protected":false},"author":1,"featured_media":22153,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[137],"tags":[],"class_list":["post-22829","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-notizie"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v25.8 - https:\/\/yoast.com\/wordpress\/plugins\/seo\/ -->\n<title>Huidagene: una promettente tecnologia CRISPR-CAS13 da Shanghai (Cina) - DupMECP2<\/title>\n<meta name=\"description\" content=\"Una nuova potenziale prospettiva per il trattamento della Sindrome da Duplicazione di MECP2 con il sistema CRISPR-Cas13 di Huidagene. Il candidato farmaco HG204 ha dimostrato una significativa riduzione della proteina MeCP2 e un&#039;inversione dei sintomi.\" \/>\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\/es\/huidagene-una-prometedora-tecnologia-crispr-cas13-de-shanghai-china\/?lang=it\" \/>\n<meta property=\"og:locale\" content=\"es_ES\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Huidagene: una promettente tecnologia CRISPR-CAS13 da Shanghai (Cina) - DupMECP2\" \/>\n<meta property=\"og:description\" content=\"Una nuova potenziale prospettiva per il trattamento della Sindrome da Duplicazione di MECP2 con il sistema CRISPR-Cas13 di Huidagene. 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