{"id":1404,"date":"2023-12-16T17:30:00","date_gmt":"2023-12-16T16:30:00","guid":{"rendered":"https:\/\/remeds.org\/?p=1404"},"modified":"2025-02-06T18:04:25","modified_gmt":"2025-02-06T17:04:25","slug":"huidagene-a-promising-crispr-cas13-technology-from-shanghai-china","status":"publish","type":"post","link":"https:\/\/remeds.org\/hr\/2023\/12\/16\/huidagene-a-promising-crispr-cas13-technology-from-shanghai-china\/","title":{"rendered":"Huidagene: Obe\u0107avaju\u0107a CRISPR-CAS13 tehnologija iz \u0160angaja (Kina)"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Huidagene Therapeutics je biotehnolo\u0161ka tvrtka sa sjedi\u0161tem u \u0160angaju i New Jerseyju, usmjerena na otkrivanje, in\u017eenjering i razvoj novih lijekova temeljenih na CRISPR-u. Tvrtka se fokusira na mi\u0161i\u0107ne, oftalmolo\u0161ke i neurolo\u0161ke poreme\u0107aje.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Huidagene je razvio HG204, adeno-asocirani virusni (AAV) vektor koji isporu\u010duje novi CRISPR-Cas13 kompleks u stanice. Dizajniran za sindrom duplikacije MECP2,&nbsp;<strong>HG204 ima za cilj regulirati prekomjernu proizvodnju razina proteina MeCP2 induciranjem razgradnje prekomjerno eksprimiranog MECP2 RNA.<\/strong>&nbsp;Ovaj inovativni kandidat za lijek ima potencijal izlije\u010diti MDS kroz jednu primjenu.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Na\u0161a organizacija imala je produktivne razgovore s \u010delnicima Huidagenea kako bi saznala vi\u0161e o ovoj inovativnoj tehnologiji i \u0161to ona zna\u010di za budu\u0107nost.&nbsp;<strong>Pretklini\u010dke studije pomo\u0107u HG204 na humaniziranom mi\u0161jem modelu MDS-a pokazale su zna\u010dajno smanjenje proteina MeCP2 i preokret simptoma.<\/strong>&nbsp;Iako su ovo ohrabruju\u0107i rezultati, ovo je ujedno i drugi dokaz reverzibilnosti simptoma MDS-a nakon regulacije razina proteina MECP2 (kao \u0161to je prethodno dokazano primjenom ASO od strane prof. H. Zoghbi &amp; tima).<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Huidagene je u procesu finaliziranja pretklini\u010dkih studija s HG204 i priprema se za klini\u010dko ispitivanje za pacijente u Kini, zakazano za po\u010detak sredinom 2024. godine.&nbsp;<\/strong>Ako ovo ispitivanje bude uspje\u0161no, Huidagene \u0107e planirati klini\u010dka ispitivanja u drugim zemljama kao \u0161to su SAD i Europa 2025.-2026.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><br>Dana 31. listopada, FDA (Food and Drug Administration, zdravstvena vlast SAD-a) dodijelila je HG204 i Oznaku za rijetke pedijatrijske bolesti i Oznaku za lijekove bez vlasnika.\nOva regulatorna oznaka je prvi korak prema klini\u010dkom ispitivanju izvan Kine i omogu\u0107it \u0107e ubrzani pregled dosjea prilikom registracije lijeka.\nIako je vijest bila potpuno neo\u010dekivana, donosi dodatnu nadu zajednici.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Na\u0161 tim \u0107e ostati u kontaktu s Huidageneom i obavje\u0161tavati na\u0161u zajednicu o svim novim razvojima.<\/p>\n\n\n\n<figure class=\"wp-block-image\"><img decoding=\"async\" src=\"https:\/\/dupmecp2.eu\/wp-content\/uploads\/2023\/12\/Huidagene_Pipeline_2023_white-edited.png\" alt=\"\" class=\"wp-image-22144\"\/><\/figure>\n\n\n\n<p class=\"wp-block-paragraph\"><a href=\"https:\/\/www.huidagene.com\/new\/news\/48\" target=\"_blank\" rel=\"noreferrer noopener\">Za priop\u0107enje za medije<\/a><\/p>","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. Huidagene has developed HG204, an adeno-associated viral (AAV) vector delivering a novel CRISPR-Cas13 complex into cells. Designed for MECP2 duplication syndrome,&nbsp;HG204 aims to regulate the [&hellip;]<\/p>\n","protected":false},"author":73,"featured_media":1429,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"_jf_save_progress":"","footnotes":""},"categories":[12,10,9],"tags":[],"class_list":["post-1404","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-crispr","category-cure","category-research"],"_links":{"self":[{"href":"https:\/\/remeds.org\/hr\/wp-json\/wp\/v2\/posts\/1404","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/remeds.org\/hr\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/remeds.org\/hr\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/remeds.org\/hr\/wp-json\/wp\/v2\/users\/73"}],"replies":[{"embeddable":true,"href":"https:\/\/remeds.org\/hr\/wp-json\/wp\/v2\/comments?post=1404"}],"version-history":[{"count":1,"href":"https:\/\/remeds.org\/hr\/wp-json\/wp\/v2\/posts\/1404\/revisions"}],"predecessor-version":[{"id":1405,"href":"https:\/\/remeds.org\/hr\/wp-json\/wp\/v2\/posts\/1404\/revisions\/1405"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/remeds.org\/hr\/wp-json\/wp\/v2\/media\/1429"}],"wp:attachment":[{"href":"https:\/\/remeds.org\/hr\/wp-json\/wp\/v2\/media?parent=1404"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/remeds.org\/hr\/wp-json\/wp\/v2\/categories?post=1404"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/remeds.org\/hr\/wp-json\/wp\/v2\/tags?post=1404"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}