{"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\/es\/hg204-huidagene\/","title":{"rendered":"Huidagene: una prometedora tecnolog\u00eda CRISPR-CAS13 de Shanghai (China)"},"content":{"rendered":"<p>Huidagene Therapeutics es una empresa de biotecnolog\u00eda con sede en Shanghai y Nueva Jersey, centrada en el descubrimiento, la ingenier\u00eda y el desarrollo de nuevos medicamentos basados en CRISPR. La empresa se centra en trastornos musculares, oft\u00e1lmicos y neurol\u00f3gicos.<\/p>\n\n\n\n<p>Huidagene ha desarrollado HG204, un vector viral adenoasociado (AAV) que introduce en las c\u00e9lulas un novedoso complejo CRISPR-Cas13. Dise\u00f1ado para el s\u00edndrome de duplicaci\u00f3n MECP2, <strong>El HG204 pretende regular la sobreproducci\u00f3n de los niveles de prote\u00edna MeCP2 induciendo la degradaci\u00f3n del ARN MECP2 sobreexpresado.<\/strong> Este innovador candidato a f\u00e1rmaco tiene el potencial de curar los SMD mediante una \u00fanica administraci\u00f3n.<\/p>\n\n\n\n<div style=\"height:25px\" aria-hidden=\"true\" class=\"wp-block-spacer\"><\/div>\n\n\n\n<p>Nuestra organizaci\u00f3n ha mantenido fruct\u00edferas conversaciones con los responsables de Huidagene para conocer mejor esta innovadora tecnolog\u00eda y lo que significa para el futuro. <strong>Los estudios precl\u00ednicos realizados con HG204 en un modelo de rat\u00f3n humanizado con SMD demostraron una reducci\u00f3n significativa de la prote\u00edna MeCP2 y la reversi\u00f3n de los s\u00edntomas.<\/strong> Si bien se trata de resultados alentadores, \u00e9sta es tambi\u00e9n la segunda prueba de la reversibilidad de los s\u00edntomas del SMD tras la regulaci\u00f3n de los niveles de prote\u00edna MECP2 (como se demostr\u00f3 anteriormente con la administraci\u00f3n del ASO por el Prof. H. Zoghbi y su equipo).<\/p>\n\n\n\n<div style=\"height:25px\" aria-hidden=\"true\" class=\"wp-block-spacer\"><\/div>\n\n\n\n<p><strong>Huidagene est\u00e1 finalizando los estudios precl\u00ednicos con el HG204 y prepar\u00e1ndose para un ensayo cl\u00ednico con pacientes en China, cuyo inicio est\u00e1 previsto para mediados de 2024. <\/strong>Si este ensayo tiene \u00e9xito, Huidagene planear\u00e1 ensayos cl\u00ednicos en otros pa\u00edses como EE.UU. y Europa en 2025-2026.<\/p>\n\n\n\n<p><br>El 31 de octubre, la FDA (Food and Drug Administration, autoridad sanitaria de EE.UU.) concedi\u00f3 al HG204 las designaciones de enfermedad pedi\u00e1trica rara y medicamento hu\u00e9rfano. Esta designaci\u00f3n reglamentaria es un primer paso hacia un ensayo cl\u00ednico fuera de China y permitir\u00e1 una revisi\u00f3n acelerada del expediente tras el registro del f\u00e1rmaco. Aunque la noticia fue totalmente inesperada, aporta una esperanza adicional a la comunidad.<\/p>\n\n\n\n<p>Nuestro equipo se mantendr\u00e1 en contacto con Huidagene y mantendr\u00e1 informada a nuestra comunidad de cualquier novedad.<\/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\">Ir al comunicado de prensa<\/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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