{"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\/es\/huidagene-eine-vielversprechende-crispr-cas13-technologie-aus-shanghai-china\/","title":{"rendered":"Huidagene: una tecnolog\u00eda CRISPR-CAS13 muy avanzada de Shangh\u00e1i (China)"},"content":{"rendered":"<p>Huidagene Therapeutics es una empresa de biotecnolog\u00eda con sedes en Shangh\u00e1i y Nueva Jersey que se dedica a la investigaci\u00f3n, la ingenier\u00eda y el desarrollo de nuevos medicamentos basados en CRISPR. La empresa se centra en los trastornos musculares, oculares y neurol\u00f3gicos.<\/p>\n\n\n\n<p>Huidagene ha desarrollado el HG204, un virus adenoasociado (AAV) que contiene un nuevo complejo CRISPR-Cas13. <strong>HG204 fue desarrollado para el s\u00edndrome de duplicidad de MECP2 y tiene como objetivo regular la producci\u00f3n de las prote\u00ednas MeCP2, induciendo la reducci\u00f3n del ARN MECP2 sobreexprimido. Este innovador candidato a f\u00e1rmaco tiene el potencial de curar los SMD mediante un \u00fanico tratamiento.<\/strong><\/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 conversaciones productivas con los responsables de Huidagene para conocer mejor esta innovadora tecnolog\u00eda y su importancia para el futuro. <strong>Los estudios cl\u00ednicos con HG204 en un modelo humano de SMD muestran una reducci\u00f3n significativa de las prote\u00ednas MeCP2 y un aumento de los s\u00edntomas.<\/strong> Estos resultados son concluyentes, ya que se trata de una segunda reducci\u00f3n de la incidencia de los s\u00edntomas del SMD tras la regulaci\u00f3n del esp\u00e9cimen de la prote\u00edna MECP2 (como se hizo hasta ahora mediante la verificaci\u00f3n de la ASO por el 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>Hasta ahora, Huidagene ha abandonado los estudios cl\u00ednicos previos sobre el HG204 y se ha centrado en un estudio cl\u00ednico con pacientes chinos que comenzar\u00e1 en verano de 2024.<\/strong> Si este estudio se lleva a cabo con \u00e9xito, se planea realizar en los a\u00f1os 2025-2026 estudios cl\u00ednicos en otros pa\u00edses, como Estados Unidos y Europa.<\/p>\n\n\n\n<p><br>Am 31. Oktober hat die FDA (Food and Drug Administration, die Gesundheitsbeh\u00f6rde der USA) sowohl die Auszeichnung als seltene p\u00e4diatrische Erkrankung als auch den Orphan-Drug-Status f\u00fcr HG204 verliehen. Esta deficiente observaci\u00f3n es el primer paso en el camino hacia un estudio cl\u00ednico en China y puede facilitar la revisi\u00f3n de los expedientes en el momento del registro. A pesar de que la noticia no ha sido publicada, la Comunidad se siente muy satisfecha.<\/p>\n\n\n\n<p>Nuestro equipo estar\u00e1 en contacto con Huidagene y nuestra comunidad estar\u00e1 al tanto de todos los nuevos avances.<\/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\">Comunicados de prensa<\/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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