{"id":523,"date":"2026-05-05T14:11:06","date_gmt":"2026-05-05T05:11:06","guid":{"rendered":"https:\/\/rmnw.jp\/teams\/?p=523"},"modified":"2026-05-05T14:56:01","modified_gmt":"2026-05-05T05:56:01","slug":"%e7%ac%ac14%e7%ab%a0-%e5%b0%8f%e5%85%90%e7%a7%91%e3%83%bb%e9%81%ba%e4%bc%9d%e6%80%a7%e7%96%be%e6%82%a3","status":"publish","type":"post","link":"https:\/\/rmnw.jp\/teams\/?p=523","title":{"rendered":"RM14.\u7b2c14\u7ae0 \u5c0f\u5150\u79d1\u30fb\u907a\u4f1d\u6027\u75be\u60a3"},"content":{"rendered":"\n<figure class=\"wp-block-embed is-type-video is-provider-youtube wp-block-embed-youtube wp-embed-aspect-16-9 wp-has-aspect-ratio\"><div class=\"wp-block-embed__wrapper\">\n<iframe loading=\"lazy\" title=\"\u7b2c14\u7ae0 \u5c0f\u5150\u79d1\u30fb\u907a\u4f1d\u6027\u75be\u60a3\" width=\"500\" height=\"281\" src=\"https:\/\/www.youtube.com\/embed\/68Hblc8XRsQ?feature=oembed\" frameborder=\"0\" allow=\"accelerometer; autoplay; clipboard-write; encrypted-media; gyroscope; picture-in-picture; web-share\" referrerpolicy=\"strict-origin-when-cross-origin\" allowfullscreen><\/iframe>\n<\/div><\/figure>\n\n\n\n\t\t<div class=\"wppdfemb-frame-container-1\" style=\"-webkit-overflow-scrolling:auto;\">\n\t\t\t<iframe class=\"pdfembed-iframe nonfullscreen wppdf-emb-iframe-1\"\n\t\t\t\tsrc=\"https:\/\/rmnw.jp\/teams\/?pdfemb-data=eyJwZGZJRCI6NTI0LCJ1cmwiOiJodHRwczpcL1wvcm1udy5qcFwvdGVhbXNcL3dwLWNvbnRlbnRcL3VwbG9hZHNcLzIwMjZcLzA1XC9cdTdiMmMxNFx1N2FlMC1cdTVjMGZcdTUxNTBcdTc5ZDFcdTMwZmJcdTkwN2FcdTRmMWRcdTYwMjdcdTc1YmVcdTYwYTMucGRmIiwiaW5kZXgiOjF9\"\n\t\t\t\t\t\t\t\tdata-pdf-id=\"524\"\n\t\t\t\tdata-pdf-index=\"1\"\n\t\t\t\tstyle=\"border:none;width:100%;max-width:100%;height:100vh;\"\n\t\t\t\tscrolling=\"yes\">\n\t\t\t<\/iframe>\n\t\t<\/div>\n\n\t\t\n\n\n<h1 class=\"wp-block-heading\">\u5c0f\u5150\u79d1\u30fb\u907a\u4f1d\u6027\u75be\u60a3\u306b\u304a\u3051\u308b\u907a\u4f1d\u5b50\u6cbb\u7642\u3068\u518d\u751f\u533b\u7642\uff1a\u52d5\u5411\u3068\u8ab2\u984c\u306b\u95a2\u3059\u308b\u30d6\u30ea\u30fc\u30d5\u30a3\u30f3\u30b0\u6587\u66f8<\/h1>\n\n\n\n<h2 class=\"wp-block-heading\">\u30a8\u30b0\u30bc\u30af\u30c6\u30a3\u30d6\u30fb\u30b5\u30de\u30ea\u30fc<\/h2>\n\n\n\n<p>\u672c\u6587\u66f8\u306f\u3001\u5c0f\u5150\u907a\u4f1d\u6027\u75be\u60a3\u306b\u5bfe\u3059\u308b\u907a\u4f1d\u5b50\u6cbb\u7642\u304a\u3088\u3073\u518d\u751f\u533b\u7642\u306e\u73fe\u72b6\u3001\u4e3b\u8981\u306a\u81e8\u5e8a\u7684\u9032\u5c55\u3001\u898f\u5236\u72b6\u6cc1\u3001\u304a\u3088\u3073\u5e02\u5834\u306e\u52d5\u5411\u3092\u307e\u3068\u3081\u305f\u3082\u306e\u3067\u3042\u308b\u3002\u4e3b\u306a\u30dd\u30a4\u30f3\u30c8\u306f\u4ee5\u4e0b\u306e\u901a\u308a\u3067\u3042\u308b\uff1a<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>\u6cbb\u7642\u30d1\u30e9\u30c0\u30a4\u30e0\u306e\u8ee2\u63db\uff1a<\/strong> \u810a\u9ac4\u6027\u7b4b\u840e\u7e2e\u75c7\uff08SMA\uff09\u3084\u82b3\u9999\u65cfL-\u30a2\u30df\u30ce\u9178\u8131\u70ad\u9178\u9175\u7d20\uff08AADC\uff09\u6b20\u640d\u75c7\u306b\u304a\u3044\u3066\u3001in vivo\u907a\u4f1d\u5b50\u6cbb\u7642\u304c\u5287\u7684\u306a\u81e8\u5e8a\u52b9\u679c\u3092\u793a\u3057\u3066\u304a\u308a\u3001\u4e0d\u6cbb\u306e\u75c5\u304b\u3089\u6cbb\u7642\u53ef\u80fd\u306a\u75be\u60a3\u3078\u306e\u8ee2\u63db\u304c\u9032\u3093\u3067\u3044\u308b\u3002<\/li>\n\n\n\n<li><strong>\u65b0\u751f\u5150\u30b9\u30af\u30ea\u30fc\u30cb\u30f3\u30b0\uff08NBS\uff09\u306e\u91cd\u8981\u6027\uff1a<\/strong> SMA\u306e\u300cSPR1NT\u300d\u8a66\u9a13\u306b\u4ee3\u8868\u3055\u308c\u308b\u3088\u3046\u306b\u3001\u767a\u75c7\u524d\u306e\u4ecb\u5165\u304c\u6700\u5927\u306e\u6cbb\u7642\u52b9\u679c\u3092\u3082\u305f\u3089\u3059\u3053\u3068\u304c\u8a3c\u660e\u3055\u308c\u3066\u304a\u308a\u3001NBS\u306e\u62e1\u5145\u304c\u55ab\u7dca\u306e\u8ab2\u984c\u3068\u306a\u3063\u3066\u3044\u308b\u3002<\/li>\n\n\n\n<li><strong>\u627f\u8a8d\u3068\u30d1\u30a4\u30d7\u30e9\u30a4\u30f3\u306e\u62e1\u5927\uff1a<\/strong> Zolgensma\uff08SMA\uff09\u3001Elevidys\uff08DMD\uff09\u3001Roctavian\uff08\u8840\u53cb\u75c5A\uff09\u3001Hemgenix\/Beqvez\uff08\u8840\u53cb\u75c5B\uff09\u3001Kebilidi\uff08AADC\u6b20\u640d\u75c7\uff09\u306a\u3069\u3001\u4e3b\u8981\u306a\u75be\u60a3\u3067\u627f\u8a8d\u304c\u76f8\u6b21\u3044\u3067\u3044\u308b\u3002<\/li>\n\n\n\n<li><strong>\u5b89\u5168\u6027\u3068\u526f\u4f5c\u7528\u306e\u76e3\u8996\uff1a<\/strong> AAV\uff08\u30a2\u30c7\u30ce\u968f\u4f34\u30a6\u30a4\u30eb\u30b9\uff09\u30d9\u30af\u30bf\u30fc\u3092\u7528\u3044\u305f\u6cbb\u7642\u306b\u304a\u3044\u3066\u3001\u8840\u6813\u6027\u5fae\u5c0f\u8840\u7ba1\u75c7\uff08TMA\uff09\u3084\u5fc3\u7b4b\u708e\u3001\u809d\u6bd2\u6027\u306a\u3069\u306e\u91cd\u7be4\u306a\u526f\u4f5c\u7528\u304c\u5831\u544a\u3055\u308c\u3066\u304a\u308a\u3001\u591a\u304f\u306e\u88fd\u54c1\u306b\u300c\u67a0\u7d44\u307f\u8b66\u544a\uff08Boxed Warning\uff09\u300d\u304c\u9069\u7528\u3055\u308c\u3066\u3044\u308b\u3002<\/li>\n\n\n\n<li><strong>\u5546\u696d\u5316\u306e\u969c\u58c1\u3068\u5236\u5ea6\u6539\u6b63\uff1a<\/strong> \u6570\u5104\u5186\u898f\u6a21\u306e\u8d85\u9ad8\u984d\u306a\u85ac\u5264\u8cbb\u3068\u3001\u9577\u671f\u7684\u306a\u6709\u52b9\u6027\u30fb\u5b89\u5168\u6027\u306e\u4e0d\u78ba\u5b9f\u6027\u304c\u666e\u53ca\u306e\u59a8\u3052\u3068\u306a\u3063\u3066\u304a\u308a\u30012024\u5e74\u306e\u85ac\u6a5f\u6cd5\u6539\u6b63\u3092\u542b\u3080\u898f\u5236\u67a0\u7d44\u307f\u306e\u6574\u5099\u304c\u9032\u884c\u3057\u3066\u3044\u308b\u3002<\/li>\n<\/ul>\n\n\n\n<p>&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8211;<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">1. \u810a\u9ac4\u6027\u7b4b\u840e\u7e2e\u75c7\uff08SMA\uff09\u306b\u304a\u3051\u308b\u9032\u5c55<\/h2>\n\n\n\n<p>SMA\u306f\u3001SMN1\u907a\u4f1d\u5b50\u306e\u6b20\u640d\u306b\u3088\u308a\u904b\u52d5\u30cb\u30e5\u30fc\u30ed\u30f3\u304c\u5909\u6027\u3057\u3001\u7b4b\u840e\u7e2e\u3092\u6765\u3059\u75be\u60a3\u3067\u3042\u308b\u3002\u907a\u4f1d\u5b50\u6cbb\u7642\u306e\u5c0e\u5165\u306b\u3088\u308a\u3001\u6cbb\u7642\u6210\u7e3e\u304c\u98db\u8e8d\u7684\u306b\u5411\u4e0a\u3057\u3066\u3044\u308b\u3002<\/p>\n\n\n\n<h3 class=\"wp-block-heading\">\u4e3b\u8981\u306a\u6cbb\u7642\u85ac\u3068\u81e8\u5e8a\u30a8\u30d3\u30c7\u30f3\u30b9<\/h3>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>Zolgensma\uff08\u30be\u30eb\u30b2\u30f3\u30b9\u30de\uff09\uff1a<\/strong> AAV9\u30d9\u30af\u30bf\u30fc\u3092\u7528\u3044\u305f\u907a\u4f1d\u5b50\u7f6e\u63db\u7642\u6cd5\u3002\n<ul class=\"wp-block-list\">\n<li><strong>SPR1NT\u8a66\u9a13\uff1a<\/strong> \u65b0\u751f\u5150\u30b9\u30af\u30ea\u30fc\u30cb\u30f3\u30b0\u3067\u767a\u898b\u3055\u308c\u305f\u767a\u75c7\u524d\u306e\u60a3\u8005\u3092\u5bfe\u8c61\u3068\u3057\u305f\u8a66\u9a13\u3002SMN2\u30b3\u30d4\u30fc\u65702\u306e\u7fa4\u3067\u306f\u300114\/15\u540d\u304c\u4ecb\u52a9\u306a\u3057\u306e\u81ea\u7acb\u6b69\u884c\u3092\u9054\u6210\u3057\u3001\u767a\u75c7\u524d\u6cbb\u7642\u306e\u5727\u5012\u7684\u306a\u512a\u4f4d\u6027\u304c\u793a\u3055\u308c\u305f\u3002<\/li>\n<\/ul>\n<\/li>\n\n\n\n<li><strong>Itvisma\uff1a<\/strong> 21kg\u4ee5\u4e0a\u306e\u60a3\u8005\u3092\u4e3b\u306a\u5bfe\u8c61\u3068\u3059\u308b\u65b0\u85ac\u3002STEER\u8a66\u9a13\uff08Phase 3\uff09\u306b\u304a\u3044\u3066\u3001HFMSE\u30b9\u30b3\u30a2\u306e\u6709\u610f\u306a\u6539\u5584\uff08+1.88\u70b9\uff09\u304c\u78ba\u8a8d\u3055\u308c\u3066\u3044\u308b\u3002<\/li>\n<\/ul>\n\n\n\n<h3 class=\"wp-block-heading\">\u5b89\u5168\u6027\u3068\u8b66\u544a\u4e8b\u9805<\/h3>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>Boxed Warning\uff1a<\/strong> Zolgensma\u306b\u306f\u3001\u6025\u6027\u809d\u4e0d\u5168\u3084\u8840\u6813\u6027\u5fae\u5c0f\u8840\u7ba1\u75c7\uff08TMA\uff09\u306e\u30ea\u30b9\u30af\u306b\u95a2\u3059\u308b\u8b66\u544a\u304c\u306a\u3055\u308c\u3066\u3044\u308b\u30022022\u5e74\u306b\u306fTMA\u306b\u3088\u308b\u6b7b\u4ea1\u4f8b\u3082\u5831\u544a\u3055\u308c\u3066\u304a\u308a\u3001\u6295\u4e0e\u5f8c\u306e\u53b3\u91cd\u306a\u30e2\u30cb\u30bf\u30ea\u30f3\u30b0\u304c\u4e0d\u53ef\u6b20\u3067\u3042\u308b\u3002<\/li>\n<\/ul>\n\n\n\n<p>&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8211;<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">2. \u30c7\u30e5\u30b7\u30a7\u30f3\u30cc\u578b\u7b4b\u30b8\u30b9\u30c8\u30ed\u30d5\u30a3\u30fc\uff08DMD\uff09<\/h2>\n\n\n\n<p>DMD\u306f\u3001\u30b8\u30b9\u30c8\u30ed\u30d5\u30a3\u30f3\u907a\u4f1d\u5b50\u306e\u5909\u7570\u306b\u3088\u308b\u9032\u884c\u6027\u306e\u7b4b\u75be\u60a3\u3067\u3042\u308b\u3002<\/p>\n\n\n\n<h3 class=\"wp-block-heading\">\u6cbb\u7642\u306e\u73fe\u72b6<\/h3>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>Elevidys\uff08\u30a8\u30ec\u30d3\u30c7\u30a3\u30b9\uff09\uff1a<\/strong> \u77ed\u7e2e\u578b\u30b8\u30b9\u30c8\u30ed\u30d5\u30a3\u30f3\uff08\u30de\u30a4\u30af\u30ed\u30b8\u30b9\u30c8\u30ed\u30d5\u30a3\u30f3\uff09\u3092\u767a\u73fe\u3055\u305b\u308bAAV\u907a\u4f1d\u5b50\u6cbb\u7642\u3002\n<ul class=\"wp-block-list\">\n<li><strong>EMBARK\u8a66\u9a13\uff08Phase 3\uff09\uff1a<\/strong> \u4e3b\u8981\u8a55\u4fa1\u9805\u76ee\u3067\u3042\u308bNSAA\u5408\u8a08\u30b9\u30b3\u30a2\u3067\u306f\u30d7\u30e9\u30bb\u30dc\u7fa4\u3068\u6709\u610f\u5dee\u304c\u51fa\u306a\u304b\u3063\u305f\u3082\u306e\u306e\u3001Time to Rise\uff08\u8d77\u7acb\u6642\u9593\uff09\u306a\u3069\u306e\u526f\u6b21\u9805\u76ee\u3067\u6709\u610f\u306a\u6539\u5584\u3092\u793a\u3057\u3001FDA\u306b\u3088\u308b\u627f\u8a8d\u7dad\u6301\u30fb\u62e1\u5927\u306e\u6839\u62e0\u3068\u306a\u3063\u305f\u3002<\/li>\n<\/ul>\n<\/li>\n\n\n\n<li><strong>deramiocel (CAP-1002)\uff1a<\/strong> \u5fc3\u7403\u7531\u6765\u7d30\u80de\uff08CDC\uff09\u3092\u7528\u3044\u305f\u6cbb\u7642\u6cd5\u3002\u5fc3\u6a5f\u80fd\uff08LVEF\uff09\u306e\u7dad\u6301\u52b9\u679c\u304c\u671f\u5f85\u3055\u308c\u3066\u304a\u308a\u30012026\u5e74\u306ePDUFA\uff08FDA\u5be9\u67fb\u671f\u9650\uff09\u3092\u63a7\u3048\u3066\u3044\u308b\u3002<\/li>\n<\/ul>\n\n\n\n<p>&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8211;<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">3. \u8840\u53cb\u75c5A\u578b\u304a\u3088\u3073B\u578b<\/h2>\n\n\n\n<p>\u8840\u53cb\u75c5\u6cbb\u7642\u306f\u3001\u5f93\u6765\u306e\u5b9a\u671f\u88dc\u5145\u7642\u6cd5\u304b\u3089\u3001\u5358\u56de\u6295\u4e0e\u3067\u9577\u671f\u52b9\u679c\u3092\u72d9\u3046\u907a\u4f1d\u5b50\u6cbb\u7642\u3078\u306e\u79fb\u884c\u671f\u306b\u3042\u308b\u3002<\/p>\n\n\n\n<h3 class=\"wp-block-heading\">\u88fd\u54c1\u3068\u7d4c\u6e08\u7684\u5074\u9762<\/h3>\n\n\n\n<figure class=\"wp-block-table\"><table class=\"has-fixed-layout\"><tbody><tr><td>\u75be\u60a3\u578b<\/td><td>\u88fd\u54c1\u540d<\/td><td>\u88fd\u9020\u5143<\/td><td>\u7279\u5fb4\u30fb\u72b6\u6cc1<\/td><\/tr><tr><td><strong>\u8840\u53cb\u75c5A<\/strong><\/td><td><strong>Roctavian<\/strong><\/td><td>BioMarin<\/td><td>AAV5\u30d9\u30af\u30bf\u30fc\u4f7f\u7528\u3002\u5e02\u5834\u6d78\u900f\u306f\u7de9\u3084\u304b\u3067\u3042\u308a\u3001\u8ca9\u58f2\u4e0d\u632f\u306b\u3088\u308b\u518d\u69cb\u7bc9\u304c\u9032\u884c\u4e2d\u3002<\/td><\/tr><tr><td><strong>\u8840\u53cb\u75c5B<\/strong><\/td><td><strong>Hemgenix<\/strong><\/td><td>CSL Behring<\/td><td>Padua\u5909\u7570FIX\u907a\u4f1d\u5b50\u3092\u4f7f\u7528\u3002\u5358\u56de\u6295\u4e0e\u3067\u51fa\u8840\u7387\u3092\u5927\u5e45\u306b\u6539\u5584\u3002<\/td><\/tr><tr><td><strong>\u8840\u53cb\u75c5B<\/strong><\/td><td><strong>Beqvez<\/strong><\/td><td>Pfizer<\/td><td>2024\u5e74\u306bFDA\u627f\u8a8d\u3002\u7d043.5\u30df\u30ea\u30aa\u30f3\u30c0\u30eb\u306e\u9ad8\u984d\u8a2d\u5b9a\u3002<\/td><\/tr><\/tbody><\/table><\/figure>\n\n\n\n<p>&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8211;<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">4. \u4e2d\u67a2\u795e\u7d4c\u30fb\u4ee3\u8b1d\u6027\u75be\u60a3\uff08AADC\u6b20\u640d\u75c7\u304a\u3088\u3073\u30ab\u30ca\u30d0\u30f3\u75c5\uff09<\/h2>\n\n\n\n<p>\u6975\u3081\u3066\u5e0c\u5c11\u306a\u75be\u60a3\u306b\u304a\u3044\u3066\u3082\u3001\u5c40\u6240\u6295\u4e0e\u3092\u4e2d\u5fc3\u3068\u3057\u305f\u907a\u4f1d\u5b50\u6cbb\u7642\u304c\u6210\u679c\u3092\u4e0a\u3052\u3066\u3044\u308b\u3002<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>AADC\u6b20\u640d\u75c7\uff08\u82b3\u9999\u65cfL-\u30a2\u30df\u30ce\u9178\u8131\u70ad\u9178\u9175\u7d20\u6b20\u640d\u75c7\uff09\uff1a<\/strong>\n<ul class=\"wp-block-list\">\n<li><strong>Kebilidi\uff1a<\/strong> \u8133\u5185\u306e\u88ab\u6bbb\uff08putamen\uff09\u3078\u76f4\u63a5\u6295\u4e0e\u3059\u308bAAV2\u907a\u4f1d\u5b50\u6cbb\u7642\u30022024\u5e7411\u6708\u306bFDA\u627f\u8a8d\u3002\u3053\u308c\u307e\u3067\u5bdd\u305f\u304d\u308a\u3067\u3042\u3063\u305f\u60a3\u8005\u304c\u3001\u904b\u52d5\u6a5f\u80fd\uff08\u982d\u90e8\u306e\u4fdd\u6301\u3084\u81ea\u7acb\u8d70\u884c\uff09\u3092\u7372\u5f97\u3059\u308b\u5287\u7684\u306a\u6539\u5584\u304c\u5831\u544a\u3055\u308c\u3066\u3044\u308b\u3002<\/li>\n<\/ul>\n<\/li>\n\n\n\n<li><strong>\u30ab\u30ca\u30d0\u30f3\u75c5\uff08Canavan Disease\uff09\uff1a<\/strong>\n<ul class=\"wp-block-list\">\n<li><strong>MYR-101 (Myrtelle)\uff1a<\/strong> \u30aa\u30ea\u30b4\u30c7\u30f3\u30c9\u30ed\u30b5\u30a4\u30c8\u3092\u6a19\u7684\u3068\u3057\u305fAAV\u6cbb\u7642\u3002<\/li>\n\n\n\n<li><strong>BBP-812 (Aspa\/BridgeBio)\uff1a<\/strong> AAV9\u30d9\u30af\u30bf\u30fc\u306b\u3088\u308b\u5168\u8eab\u6295\u4e0e\u3002NAA\uff08N-\u30a2\u30bb\u30c1\u30eb\u30a2\u30b9\u30d1\u30e9\u30ae\u30f3\u9178\uff09\u306e\u6e1b\u5c11\u3092\u30b5\u30ed\u30b2\u30fc\u30c8\u30a8\u30f3\u30c9\u30dd\u30a4\u30f3\u30c8\u3068\u3057\u3066\u958b\u767a\u304c\u9032\u3080\u3002<\/li>\n<\/ul>\n<\/li>\n<\/ul>\n\n\n\n<p>&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8212;&#8211;<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">5. \u898f\u5236\u30fb\u5236\u5ea6\u7684\u67a0\u7d44\u307f\u3068\u4eca\u5f8c\u306e\u5c55\u671b<\/h2>\n\n\n\n<h3 class=\"wp-block-heading\">\u65e5\u672c\u306b\u304a\u3051\u308b\u5236\u5ea6\u6539\u6b63\uff082024\u5e74\u85ac\u6a5f\u6cd5\u6539\u6b63\uff09<\/h3>\n\n\n\n<p>2024\u5e74\u306e\u6539\u6b63\u3067\u306f\u3001\u7279\u306b <strong>in vivo\u907a\u4f1d\u5b50\u6cbb\u7642<\/strong> \u306e\u7279\u6027\u306b\u5408\u308f\u305b\u305f\u898f\u5236\u6574\u5099\u304c\u9032\u3081\u3089\u308c\u3066\u3044\u308b\u3002<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>ex vivo\u6cbb\u7642\u3068\u306e\u533a\u5206\uff1a<\/strong> \u7d30\u80de\u3092\u4f53\u5916\u3067\u52a0\u5de5\u3059\u308bex vivo\u6cbb\u7642\u306b\u5bfe\u3057\u3001\u30d9\u30af\u30bf\u30fc\u3092\u76f4\u63a5\u4f53\u5185\u306b\u6295\u4e0e\u3059\u308bin vivo\u6cbb\u7642\u306e\u81e8\u5e8a\u5b9f\u614b\u306b\u5373\u3057\u305f\u8a55\u4fa1\u57fa\u6e96\u304c\u8a2d\u3051\u3089\u308c\u305f\u3002<\/li>\n\n\n\n<li><strong>\u9577\u671f\u30d5\u30a9\u30ed\u30fc\u30a2\u30c3\u30d7\uff1a<\/strong> AAV\u30d9\u30af\u30bf\u30fc\u3092\u7528\u3044\u305f\u6cbb\u7642\u3067\u306f\u30015\u301c15\u5e74\u7a0b\u5ea6\u306e\u9577\u671f\u89b3\u5bdf\u304cFDA\u7b49\u306e\u30ac\u30a4\u30c9\u30e9\u30a4\u30f3\u3067\u6c42\u3081\u3089\u308c\u3066\u304a\u308a\u3001\u65e5\u672c\u3067\u3082\u540c\u69d8\u306e\u5b89\u5168\u76e3\u8996\u4f53\u5236\u304c\u6574\u5099\u3055\u308c\u3064\u3064\u3042\u308b\u3002<\/li>\n<\/ul>\n\n\n\n<h3 class=\"wp-block-heading\">\u5546\u696d\u5316\u306e\u8ab2\u984c<\/h3>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>\u30b3\u30b9\u30c8\u3068\u30a2\u30af\u30bb\u30b9\uff1a<\/strong> 1\u5264\u6570\u5104\u5186\u3068\u3044\u3046\u4fa1\u683c\u8a2d\u5b9a\uff08\u4f8b\uff1aZolgensma 2.3M\u30c9\u30eb\u3001Hemgenix 3.5M\u30c9\u30eb\uff09\u306b\u5bfe\u3057\u3001\u533b\u7642\u7d4c\u6e08\u7684\u8a55\u4fa1\uff08ICER\u7b49\uff09\u306b\u57fa\u3065\u3044\u305f\u652f\u6255\u5236\u5ea6\u3084\u3001\u6210\u679c\u9023\u52d5\u578b\u652f\u6255\u3044\u306e\u691c\u8a0e\u304c\u5fc5\u8981\u3068\u306a\u3063\u3066\u3044\u308b\u3002<\/li>\n\n\n\n<li><strong>\u88fd\u9020\u80fd\u529b\u306e\u62e1\u5927\uff1a<\/strong> \u5e0c\u5c11\u75be\u60a3\u304b\u3089\u3088\u308a\u60a3\u8005\u6570\u306e\u591a\u3044\u75be\u60a3\u3078\u306e\u9069\u5fdc\u62e1\u5927\u306b\u5411\u3051\u3001\u30d9\u30af\u30bf\u30fc\u88fd\u9020\u306e\u30b9\u30b1\u30fc\u30eb\u30a2\u30c3\u30d7\u304c\u91cd\u8981\u8ab2\u984c\u3068\u306a\u3063\u3066\u3044\u308b\u3002<\/li>\n<\/ul>\n\n\n\n<h3 class=\"wp-block-heading\">\u7d50\u8ad6<\/h3>\n\n\n\n<p>\u5c0f\u5150\u30fb\u907a\u4f1d\u6027\u75be\u60a3\u306b\u5bfe\u3059\u308b\u907a\u4f1d\u5b50\u6cbb\u7642\u306f\u3001\u81e8\u5e8a\u7684\u6210\u529f\u3092\u53ce\u3081\u3064\u3064\u3042\u308b\u4e00\u65b9\u3067\u3001\u9ad8\u984d\u306a\u6cbb\u7642\u8cbb\u3001\u88fd\u9020\u306e\u8907\u96d1\u6027\u3001\u305d\u3057\u3066\u9577\u671f\u7684\u306a\u5b89\u5168\u6027\u306e\u4e0d\u78ba\u5b9f\u6027\u3068\u3044\u3046\u300c3\u3064\u306e\u58c1\u300d\u306b\u76f4\u9762\u3057\u3066\u3044\u308b\u3002\u4eca\u5f8c\u306f\u3001\u65b0\u751f\u5150\u30b9\u30af\u30ea\u30fc\u30cb\u30f3\u30b0\u306e\u6a19\u6e96\u5316\u3068\u3001\u3053\u308c\u3089\u5148\u7aef\u533b\u7642\u3092\u652f\u3048\u308b\u6301\u7d9a\u53ef\u80fd\u306a\u533b\u7642\u5236\u5ea6\u306e\u69cb\u7bc9\u304c\u4e0d\u53ef\u6b20\u3067\u3042\u308b\u3002<\/p>\n","protected":false},"excerpt":{"rendered":"\u5c0f\u5150\u79d1\u30fb\u907a\u4f1d\u6027\u75be\u60a3\u306b\u304a\u3051\u308b\u907a\u4f1d\u5b50\u6cbb\u7642\u3068\u518d\u751f\u533b\u7642\uff1a\u52d5\u5411\u3068\u8ab2\u984c\u306b\u95a2\u3059\u308b\u30d6\u30ea\u30fc\u30d5\u30a3\u30f3\u30b0\u6587\u66f8 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