{"id":1402,"date":"2024-04-03T17:30:39","date_gmt":"2024-04-03T15:30:39","guid":{"rendered":"https:\/\/remeds.org\/?p=1402"},"modified":"2025-09-10T08:53:27","modified_gmt":"2025-09-10T06:53:27","slug":"huidagene-moving-towards-clinical-trials","status":"publish","type":"post","link":"https:\/\/remeds.org\/da\/2024\/04\/03\/huidagene-moving-towards-clinical-trials\/","title":{"rendered":"HuidaGene: P\u00e5 vej mod kliniske fors\u00f8g"},"content":{"rendered":"<p>I februar 2024 bes\u00f8gte David Covini, formand for DupMECP2, det innovative biotekfirma HuidaGene Therapeutics i Shanghai. Bes\u00f8get gav et sp\u00e6ndende indblik i fremtiden for en behandling af MECP2-duplikationssyndromet (MDS).<\/p>\n\n\n\n<p>Under ledelse af Dr. Alvin Luk, CEO hos HuidaGene, er virksomheden p\u00e5 forkant med udviklingen af en banebrydende CRISPR-teknologi, der kan revolutionere livet for b\u00f8rn, der er ramt af MDS.&nbsp;<\/p>\n\n\n\n<p>Bes\u00f8get var en fantastisk mulighed for p\u00e5 f\u00f8rste h\u00e5nd at se de avancerede faciliteter og det kreative milj\u00f8, der fremmer s\u00e5 innovativ forskning. Kontorlokalerne omfatter endda en klatrev\u00e6g, som fremmer en dynamisk og kreativ arbejdsatmosf\u00e6re.<\/p>\n\n\n\n<figure class=\"wp-block-image size-large\"><img fetchpriority=\"high\" decoding=\"async\" width=\"1920\" height=\"1080\" src=\"https:\/\/remeds.org\/wp-content\/uploads\/2025\/07\/Newsletter-1-edited.png\" alt=\"\" class=\"wp-image-3480\" srcset=\"https:\/\/remeds.org\/wp-content\/uploads\/2025\/07\/Newsletter-1-edited.png 1920w, https:\/\/remeds.org\/wp-content\/uploads\/2025\/07\/Newsletter-1-edited-300x169.png 300w, https:\/\/remeds.org\/wp-content\/uploads\/2025\/07\/Newsletter-1-edited-1024x576.png 1024w, https:\/\/remeds.org\/wp-content\/uploads\/2025\/07\/Newsletter-1-edited-768x432.png 768w, https:\/\/remeds.org\/wp-content\/uploads\/2025\/07\/Newsletter-1-edited-1536x864.png 1536w, https:\/\/remeds.org\/wp-content\/uploads\/2025\/07\/Newsletter-1-edited-18x10.png 18w\" sizes=\"(max-width: 1920px) 100vw, 1920px\" \/><\/figure>\n\n\n\n<p>Alvin og hans medicinske direkt\u00f8r for MDS, Dr. Gloria Tang, pr\u00e6senterede detaljerne i CRISPR-Cas13 RNA-redigeringsteknologien til MECP2-duplikationssyndrom, kaldet HG204. De lovende data og forel\u00f8bige resultater fra toksicitetsunders\u00f8gelser p\u00e5 ikke-menneskelige primater har f\u00f8rt til en sp\u00e6ndende udvikling. Selskabets kliniske fors\u00f8g med MDS, som oprindeligt var planlagt til at starte i anden halvdel af 2024, har allerede modtaget godkendelse fra to hospitalers etiske komit\u00e9er, og det er planen at indskrive MDS-patienter inden \u00e5rets udgang.<\/p>\n\n\n\n<p>Dette er fantastiske nyheder for MDS-samfundet. Det kommende HG204 MDS-kliniske fors\u00f8g, kaldet \"HERO\", vil i f\u00f8rste omgang behandle mindst 4 patienter, som vil blive n\u00f8je overv\u00e5get i et \u00e5r. Den f\u00f8rste fase af dette fors\u00f8g vil blive gennemf\u00f8rt i Kina. Yderligere patientrekruttering i Kina vil afh\u00e6nge af de f\u00f8rste forel\u00f8bige resultater af det interventionelle studie.&nbsp;<\/p>\n\n\n\n<p class=\"translation-block\">HuidaGene har allerede gjort betydelige fremskridt med hensyn til myndighedsgodkendelse og sikret sig to FDA-betegnelser (Rare Pediatric Disease Designation (RPDD) og Orphan Drug Designation (ODD)) for HG204 til behandling af <em>MECP2<\/em>-duplikationssyndrom. De har ogs\u00e5 indledt kontakt med b\u00e5de FDA og EMA med henblik p\u00e5 yderligere unders\u00f8gelser i USA og Europa.<\/p>\n\n\n\n<p>Det var et privilegium at f\u00e5 mulighed for at tale personligt med Alvin og Gloria. Vi er begejstrede for at v\u00e6re en del af denne rejse og ser frem til at holde vores samfund opdateret om udviklingen af denne potentielt livsforandrende teknologi.<\/p>","protected":false},"excerpt":{"rendered":"<p>In February 2024, David Covini, President of DupMECP2, visited the innovative biotech company HuidaGene Therapeutics in Shanghai. The visit provided an exciting glimpse into the future for a treatment for MECP2 duplication syndrome (MDS). Led by Dr. Alvin Luk, CEO at HuidaGene, the Company is at the forefront of developing a groundbreaking CRISPR technology that [&hellip;]<\/p>\n","protected":false},"author":73,"featured_media":3480,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"_jf_save_progress":"","footnotes":""},"categories":[12,10,9],"tags":[],"class_list":["post-1402","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-crispr","category-cure","category-research"],"_links":{"self":[{"href":"https:\/\/remeds.org\/da\/wp-json\/wp\/v2\/posts\/1402","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/remeds.org\/da\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/remeds.org\/da\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/remeds.org\/da\/wp-json\/wp\/v2\/users\/73"}],"replies":[{"embeddable":true,"href":"https:\/\/remeds.org\/da\/wp-json\/wp\/v2\/comments?post=1402"}],"version-history":[{"count":4,"href":"https:\/\/remeds.org\/da\/wp-json\/wp\/v2\/posts\/1402\/revisions"}],"predecessor-version":[{"id":3561,"href":"https:\/\/remeds.org\/da\/wp-json\/wp\/v2\/posts\/1402\/revisions\/3561"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/remeds.org\/da\/wp-json\/wp\/v2\/media\/3480"}],"wp:attachment":[{"href":"https:\/\/remeds.org\/da\/wp-json\/wp\/v2\/media?parent=1402"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/remeds.org\/da\/wp-json\/wp\/v2\/categories?post=1402"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/remeds.org\/da\/wp-json\/wp\/v2\/tags?post=1402"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}