{"id":20429,"date":"2025-09-03T10:06:17","date_gmt":"2025-09-03T10:06:17","guid":{"rendered":"https:\/\/itbusinesstoday.com\/?p=20429"},"modified":"2025-09-03T10:06:17","modified_gmt":"2025-09-03T10:06:17","slug":"medipal-and-jcr-ink-global-deal-for-gm2-drug-jr-479","status":"publish","type":"post","link":"https:\/\/itbusinesstoday.com\/ja\/health-tech\/pharma\/medipal-and-jcr-ink-global-deal-for-gm2-drug-jr-479\/","title":{"rendered":"\u30e1\u30c7\u30a3\u30d1\u30eb\u3068JCR\u3001GM2\u6cbb\u7642\u85acJR-479\u306e\u30b0\u30ed\u30fc\u30d0\u30eb\u5951\u7d04\u3092\u7de0\u7d50"},"content":{"rendered":"<?xml encoding=\"utf-8\" ?><p>\u682a\u5f0f\u4f1a\u793e\u30e1\u30c7\u30a3\u30d1\u30eb\u30db\u30fc\u30eb\u30c7\u30a3\u30f3\u30b0\u30b9\u3068JCR\u30d5\u30a1\u30fc\u30de\u30b7\u30e5\u30fc\u30c6\u30a3\u30ab\u30eb\u30ba\u682a\u5f0f\u4f1a\u793e\u306f\u3001\u8d85\u5e0c\u5c11\u306a\u30e9\u30a4\u30bd\u30be\u30fc\u30e0\u8caf\u8535\u969c\u5bb3\u3067\u3042\u308bGM2\u30ac\u30f3\u30b0\u30ea\u30aa\u30b7\u30c9\u30fc\u30b7\u30b9\u6cbb\u7642\u85ac\u300cJR-479\u300d\u306b\u3064\u3044\u3066\u3001\u30b0\u30ed\u30fc\u30d0\u30eb\u3067\u306e\u72ec\u5360\u7684\u30e9\u30a4\u30bb\u30f3\u30b9\u5951\u7d04\u304a\u3088\u3073\u65e5\u672c\u306b\u304a\u3051\u308b\u5171\u540c\u958b\u767a\u30fb\u5546\u696d\u5316\u63d0\u643a\u3092\u7de0\u7d50\u3057\u305f\u3068\u767a\u8868\u3057\u307e\u3057\u305f\u3002.<\/p><p>GM2\u30ac\u30f3\u30b0\u30ea\u30aa\u30b7\u30c9\u30fc\u30b7\u30b9\u306f\u3001\u30e9\u30a4\u30bd\u30be\u30fc\u30e0\u3067\u306eGM2\u30ac\u30f3\u30b0\u30ea\u30aa\u30b7\u30c9\u306e\u6b63\u5e38\u306a\u5206\u89e3\u3092\u59a8\u3052\u308b\u907a\u4f1d\u5b50\u5909\u7570\u306b\u3088\u3063\u3066\u5f15\u304d\u8d77\u3053\u3055\u308c\u308b\u3001\u307e\u308c\u306a\u907a\u4f1d\u6027\u75be\u60a3\u7fa4\u3067\u3042\u308a\u3001\u7279\u306b\u795e\u7d4c\u7d30\u80de\u306b\u304a\u3044\u3066\u84c4\u7a4d\u3092\u5f15\u304d\u8d77\u3053\u3057\u307e\u3059\u3002GM2\u30ac\u30f3\u30b0\u30ea\u30aa\u30b7\u30c9\u30fc\u30b7\u30b9\u306e\u6700\u3082\u4e00\u822c\u7684\u306a2\u3064\u306e\u30bf\u30a4\u30d7\u306f\u30c6\u30a4-\u30b5\u30c3\u30af\u30b9\u75c5\u3068\u30b5\u30f3\u30c9\u30db\u30d5\u75c5\u3067\u3001\u5909\u7570\u306e\u7a2e\u985e\u306b\u3088\u3063\u3066\u7570\u306a\u308a\u307e\u3059\u3002GM2\u30ac\u30f3\u30b0\u30ea\u30aa\u30b7\u30c9\u30fc\u30b7\u30b9\u306f\u3001\u4e16\u754c\u4e2d\u306730\u4e07\u4eba\u306b1\u4eba\u306e\u5272\u5408\u3067\u767a\u75c7\u3059\u308b\u3068\u63a8\u5b9a\u3055\u308c\u3001\u73fe\u5728\u627f\u8a8d\u3055\u308c\u305f\u6cbb\u7642\u6cd5\u304c\u5b58\u5728\u3057\u306a\u3044\u3001\u751f\u547d\u3092\u8105\u304b\u3059\u8d85\u5e0c\u5c11\u75be\u60a3\u3068\u8003\u3048\u3089\u308c\u3066\u3044\u307e\u3059\u3002.<\/p><p>JR-479 is a novel drug candidate developed with JCR&rsquo;s proprietary J-Brain Cargo&reg; platform, clinically validated through IZCARGO&reg; for I.V. infusion 10mg, which gained approval in Japan in 2021 for the treatment of a lysosomal storage disorder. In preclinical studies,1 JR-479 was delivered throughout the body, including to the central nervous system, reducing disease-related substrates and extending survival in animal models.<\/p><h4>\u3053\u3061\u3089\u3082\u304a\u8aad\u307f\u304f\u3060\u3055\u3044\uff1a <a class=\"p-url\" href=\"https:\/\/itbusinesstoday.com\/ja\/health-tech\/pharma\/eisais-tazemetostat-approved-as-orphan-drug-in-japan\/\" rel=\"bookmark\">Eisai&rsquo;s Tazemetostat Approved as Orphan Drug in Japan<\/a><\/h4><p>\u4eca\u56de\u306e\u30b0\u30ed\u30fc\u30d0\u30eb\u30e9\u30a4\u30bb\u30f3\u30b9\u5951\u7d04\u306b\u3088\u308a\u3001\u30e1\u30c7\u30a3\u30d1\u30eb\u306fJR-479\u306e\u958b\u767a\u3001\u88fd\u9020\u3001\u5546\u696d\u5316\u306b\u95a2\u3059\u308b\u5168\u4e16\u754c\uff08\u65e5\u672c\u3092\u9664\u304f\uff09\u3067\u306e\u72ec\u5360\u7684\u6a29\u5229\u3092\u6709\u3057\u3001\u30b5\u30d6\u30e9\u30a4\u30bb\u30f3\u30b9\u6a29\u3082\u6709\u3057\u307e\u3059\u3002\u65e5\u672c\u3067\u306f\u3001\u30e1\u30c7\u30a3\u30d1\u30eb\u304c\u5171\u540c\u958b\u767a\u3092\u901a\u3058\u3066\u81e8\u5e8a\u8a66\u9a13\u306e\u30ed\u30b8\u30b9\u30c6\u30a3\u30af\u30b9\u3001\u75be\u60a3\u5553\u767a\u306e\u652f\u63f4\u3001\u696d\u52d9\u306e\u652f\u63f4\u3092\u884c\u3044\u3001JCR\u304c\u5546\u696d\u5316\u3092\u4e3b\u5c0e\u3057\u3001\u56fd\u5185\u3067\u306e\u53ce\u76ca\u3092\u8a08\u4e0a\u3057\u307e\u3059\u3002\u307e\u305f\u3001JCR\u306f\u5951\u7d04\u4e00\u6642\u91d1\u304a\u3088\u3073\u6d77\u5916\u58f2\u4e0a\u9ad8\u306b\u5fdc\u3058\u305f\u30ed\u30a4\u30e4\u30eb\u30c6\u30a3\u3092\u53d7\u3051\u53d6\u308a\u307e\u3059\u3002.<\/p><p>\u672c\u5951\u7d04\u306b\u3088\u308a\u3001JR-479\u306f\u3001\u4ee5\u4e0b\u306e\u6226\u7565\u7684\u63d0\u643a\u306b\u52a0\u308f\u308a\u307e\u3059\u3002 <a href=\"https:\/\/www.medipal.co.jp\/\" data-no-translation=\"\">MEDIPAL<\/a> \u305d\u3057\u3066 <a href=\"https:\/\/www.jcrpharm.co.jp\/\" data-no-translation=\"\">JCR<\/a>, JR-479\u306f\u30012022\u5e7410\u6708\u306b\u767a\u58f2\u3055\u308c\u305f\u3001\u8d85\u5e0c\u5c11\u75be\u60a3\u306e\u6cbb\u7642\u85ac\u958b\u767a\u306b\u95a2\u3059\u308b\u30d1\u30a4\u30d7\u30e9\u30a4\u30f3\u3067\u3059\u3002JR-479\u306f\u3001\u30d5\u30b3\u30b7\u30c9\u30fc\u30b7\u30b92\u6cbb\u7642\u85acJR-471\u3001\u30e0\u30b3\u591a\u7cd6\u75c7IIIB\u578b3\u6cbb\u7642\u85acJR-446\u306b\u7d9a\u304f3\u756a\u76ee\u306e\u30d1\u30a4\u30d7\u30e9\u30a4\u30f3\u3068\u306a\u308a\u307e\u3059\u3002.<\/p><p>\u4e21\u793e\u306f\u3001\u672c\u5408\u610f\u306b\u3088\u308b\u696d\u7e3e\u3078\u306e\u5f71\u97ff\u3092\u65e2\u306b2026\u5e743\u6708\u671f\u306e\u9023\u7d50\u696d\u7e3e\u4e88\u60f3\u306b\u7e54\u308a\u8fbc\u3093\u3067\u304a\u308a\u307e\u3059\u3002.<\/p><p><strong>\u30bd\u30fc\u30b9 <a href=\"https:\/\/www.businesswire.com\/news\/home\/20250902432425\/en\/MEDIPAL-HOLDINGS-and-JCR-Pharmaceuticals-Sign-Global-Licensing-and-Japan-Co-Development-Agreement-for-JR-479-a-Novel-Therapy-for-GM2-Gangliosidosis\" data-no-translation=\"\">BusinessWire<\/a><\/strong><\/p>","protected":false},"excerpt":{"rendered":"<p>MEDIPAL HOLDINGS CORPORATION and JCR Pharmaceuticals Co., Ltd. announced that the two companies have signed an exclusive global licensing deal and a co-development and commercialization partnership in Japan for JR-479, an investigational therapy for GM2 gangliosidosis, an ultra-rare lysosomal storage disorder.GM2 gangliosidoses are a group of rare, inherited disorders caused by genetic mutations that prevent the normal breakdown of GM2 gangliosides in lysosomes, leading to their buildup, especially in neurons. Tay-Sachs disease and Sandhoff disease are the two most common types of GM2 gangliosidosis depending on the specific mutation. GM2 gangliosidosis affects an estimated one in 300,000 people worldwide and is considered an ultra-rare, life-threatening disorder for which no approved [&hellip;]<\/p>\n","protected":false},"author":4,"featured_media":20430,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_bbp_topic_count":0,"_bbp_reply_count":0,"_bbp_total_topic_count":0,"_bbp_total_reply_count":0,"_bbp_voice_count":0,"_bbp_anonymous_reply_count":0,"_bbp_topic_count_hidden":0,"_bbp_reply_count_hidden":0,"_bbp_forum_subforum_count":0,"wprm-recipe-roundup-name":"","wprm-recipe-roundup-description":"","postBodyCss":"","postBodyMargin":[],"postBodyPadding":[],"postBodyBackground":{"backgroundType":"classic","gradient":""},"footnotes":""},"categories":[208,89,211,237,182,206],"tags":[7560,7562,7561,7563,424],"ppma_author":[205],"class_list":["post-20429","post","type-post","status-publish","format-standard","has-post-thumbnail","category-apac","category-health-tech","category-japan","category-news","category-pharma","category-regional","tag-gm2-gangliosidosis","tag-jcr-pharmaceuticals","tag-jr-479","tag-medipal-holdings","tag-pharma"],"yoast_head":"<!-- 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