{"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\/hu\/2024\/04\/03\/huidagene-moving-towards-clinical-trials\/","title":{"rendered":"HuidaGene: A klinikai vizsg\u00e1latok fel\u00e9 haladva mieloid diszpl\u00e1zi\u00e1s szindr\u00f3ma (MDS) eset\u00e9n"},"content":{"rendered":"

2024 febru\u00e1rj\u00e1ban David Covini, a DupMECP2 eln\u00f6ke megl\u00e1togatta a HuidaGene Therapeutics innovat\u00edv biotechnol\u00f3giai v\u00e1llalatot Sanghajban. A l\u00e1togat\u00e1s izgalmas betekint\u00e9st ny\u00fajtott a MECP2 duplik\u00e1ci\u00f3s szindr\u00f3ma (MDS) kezel\u00e9s\u00e9nek j\u00f6v\u0151j\u00e9be.<\/p>\n\n\n\n

Dr. Alvin Luk, a HuidaGene vez\u00e9rigazgat\u00f3ja vezet\u00e9s\u00e9vel a v\u00e1llalat \u00e9len j\u00e1r egy \u00fatt\u00f6r\u0151 CRISPR technol\u00f3gia kifejleszt\u00e9s\u00e9ben, amely forradalmas\u00edthatja az MDS-ben szenved\u0151 gyermekek \u00e9let\u00e9t. <\/p>\n\n\n\n

A l\u00e1togat\u00e1s hihetetlen lehet\u0151s\u00e9g volt arra, hogy els\u0151 k\u00e9zb\u0151l l\u00e1thassuk a legmodernebb l\u00e9tes\u00edtm\u00e9nyeket \u00e9s azt a kreat\u00edv k\u00f6rnyezetet, amely ilyen innovat\u00edv kutat\u00e1sokat t\u00e1mogat. Az irodahelyis\u00e9gek k\u00f6z\u00f6tt m\u00e9g egy m\u00e1sz\u00f3fal is tal\u00e1lhat\u00f3, ami el\u0151seg\u00edti a dinamikus \u00e9s kreat\u00edv munkahelyi l\u00e9gk\u00f6rt.<\/p>\n\n\n\n

\"\"<\/figure>\n\n\n\n

Alvin \u00e9s az MDS orvosi igazgat\u00f3ja, Dr. Gloria Tang bemutatta a MECP2 duplik\u00e1ci\u00f3s szindr\u00f3ma, named HG204 CRISPR-Cas13 RNS-szerkeszt\u00e9si technol\u00f3gi\u00e1j\u00e1nak r\u00e9szleteit. Az \u00edg\u00e9retes adatok \u00e9s a f\u0151eml\u0151s \u00e1llatokon v\u00e9gzett toxicit\u00e1si vizsg\u00e1latok el\u0151zetes eredm\u00e9nyei izgalmas fejleszt\u00e9shez vezettek. A T\u00e1rsas\u00e1g MDS klinikai vizsg\u00e1lata, amelyet eredetileg 2024 m\u00e1sodik fel\u00e9ben terveztek elind\u00edtani, m\u00e1r megkapta k\u00e9t k\u00f3rh\u00e1z etikai bizotts\u00e1g\u00e1nak j\u00f3v\u00e1hagy\u00e1s\u00e1t, \u00e9s a tervek szerint m\u00e9g az \u00e9v v\u00e9ge el\u0151tt MDS betegek felv\u00e9tel\u00e9t tervezik.<\/p>\n\n\n\n

Ez fantasztikus h\u00edr az MDS k\u00f6z\u00f6ss\u00e9g sz\u00e1m\u00e1ra. A k\u00f6zelg\u0151 HG204 MDS klinikai vizsg\u00e1lat, az named \"HERO\" kezdetben legal\u00e1bb 4 beteget fog kezelni, akiket egy \u00e9ven kereszt\u00fcl szorosan nyomon fognak k\u00f6vetni. A vizsg\u00e1lat els\u0151 f\u00e1zis\u00e1t K\u00edn\u00e1ban fogj\u00e1k v\u00e9gezni. A tov\u00e1bbi betegfelv\u00e9tel K\u00edn\u00e1ban a beavatkoz\u00e1si vizsg\u00e1lat els\u0151 el\u0151zetes eredm\u00e9nyeit\u0151l f\u00fcgg. <\/p>\n\n\n\n

A HuidaGene m\u00e1r eddig is jelent\u0151s l\u00e9p\u00e9seket tett a hat\u00f3s\u00e1gi enged\u00e9lyez\u00e9s ter\u00e9n, \u00e9s k\u00e9t FDA-jel\u00f6l\u00e9st (Rare Pediatric Disease Designation (RPDD) \u00e9s Orphan Drug Designation (ODD)) kapott a HG204-re a MECP2<\/em> duplik\u00e1ci\u00f3s szindr\u00f3ma kezel\u00e9s\u00e9re. Az FDA-val \u00e9s az EMA-val is kezdem\u00e9nyezt\u00e9k a kapcsolatfelv\u00e9telt tov\u00e1bbi vizsg\u00e1latok c\u00e9lj\u00e1b\u00f3l az Egyes\u00fclt \u00c1llamokban \u00e9s Eur\u00f3p\u00e1ban.<\/p>\n\n\n\n

Kiv\u00e1lts\u00e1gos volt a lehet\u0151s\u00e9g, hogy szem\u00e9lyesen besz\u00e9lhettem Alvinnal \u00e9s Glori\u00e1val. Nagyon \u00f6r\u00fcl\u00fcnk, hogy r\u00e9szesei lehet\u00fcnk ennek az \u00fatnak, \u00e9s alig v\u00e1rjuk, hogy folyamatosan t\u00e1j\u00e9koztathassuk k\u00f6z\u00f6ss\u00e9g\u00fcnket ennek a potenci\u00e1lisan \u00e9letet megv\u00e1ltoztat\u00f3 technol\u00f3gi\u00e1nak az el\u0151rehalad\u00e1s\u00e1r\u00f3l.<\/p>","protected":false},"excerpt":{"rendered":"

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 […]<\/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\/hu\/wp-json\/wp\/v2\/posts\/1402","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/remeds.org\/hu\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/remeds.org\/hu\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/remeds.org\/hu\/wp-json\/wp\/v2\/users\/73"}],"replies":[{"embeddable":true,"href":"https:\/\/remeds.org\/hu\/wp-json\/wp\/v2\/comments?post=1402"}],"version-history":[{"count":4,"href":"https:\/\/remeds.org\/hu\/wp-json\/wp\/v2\/posts\/1402\/revisions"}],"predecessor-version":[{"id":3561,"href":"https:\/\/remeds.org\/hu\/wp-json\/wp\/v2\/posts\/1402\/revisions\/3561"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/remeds.org\/hu\/wp-json\/wp\/v2\/media\/3480"}],"wp:attachment":[{"href":"https:\/\/remeds.org\/hu\/wp-json\/wp\/v2\/media?parent=1402"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/remeds.org\/hu\/wp-json\/wp\/v2\/categories?post=1402"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/remeds.org\/hu\/wp-json\/wp\/v2\/tags?post=1402"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}