{"id":474,"date":"2026-05-05T13:26:20","date_gmt":"2026-05-05T04:26:20","guid":{"rendered":"https:\/\/rmnw.jp\/teams\/?p=474"},"modified":"2026-05-05T13:26:22","modified_gmt":"2026-05-05T04:26:22","slug":"rm02-%e7%ac%ac%e4%ba%8c%e7%ab%a0-%e5%86%8d%e7%94%9f%e5%8c%bb%e7%99%82%e3%81%ae%e6%b3%95%e5%be%8b","status":"publish","type":"post","link":"https:\/\/rmnw.jp\/teams\/?p=474","title":{"rendered":"RM02. \u7b2c\u4e8c\u7ae0 \u518d\u751f\u533b\u7642\u306e\u6cd5\u5f8b"},"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=\"RM02. \u7b2c\u4e8c\u7ae0 \u518d\u751f\u533b\u7642\u306e\u6cd5\u5f8b\" width=\"500\" height=\"281\" src=\"https:\/\/www.youtube.com\/embed\/Hq5gB4sJDBA?feature=oembed\" frameborder=\"0\" allow=\"accelerometer; autoplay; clipboard-write; encrypted-media; gyroscope; picture-in-picture; web-share\" 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class=\"wp-block-heading\">\u518d\u751f\u533b\u7642\u7b49\u88fd\u54c1\u306e\u898f\u5236\u67a0\u7d44\u307f\u3068\u5e02\u5834\u52d5\u5411\uff1a\u30b0\u30ed\u30fc\u30d0\u30eb\u6bd4\u8f03\u3068\u5c06\u6765\u5c55\u671b\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\u65e5\u672c\u3001\u7c73\u56fd\u3001\u6b27\u5dde\u306b\u304a\u3051\u308b\u518d\u751f\u533b\u7642\u7b49\u88fd\u54c1\u306e\u6cd5\u7684\u898f\u5236\u3001\u627f\u8a8d\u30b7\u30b9\u30c6\u30e0\u3001\u88fd\u9020\u57fa\u6e96\u3001\u304a\u3088\u30732026\u5e74\u306b\u5411\u3051\u305f\u5e02\u5834\u52d5\u5411\u3092\u7db2\u7f85\u7684\u306b\u5206\u6790\u3057\u305f\u3082\u306e\u3067\u3042\u308b\u3002\u518d\u751f\u533b\u7642\u306f\u3001\u5f93\u6765\u306e\u533b\u85ac\u54c1\u3068\u306f\u7570\u306a\u308b\u300c\u7d30\u80de\u300d\u3084\u300c\u907a\u4f1d\u5b50\u300d\u3092\u6271\u3046\u305f\u3081\u3001\u5404\u56fd\u3067\u72ec\u81ea\u306e\u898f\u5236\u67a0\u7d44\u307f\u304c\u69cb\u7bc9\u3055\u308c\u3066\u3044\u308b\u3002<\/p>\n\n\n\n<p>\u4e3b\u8981\u306a\u30dd\u30a4\u30f3\u30c8\u306f\u4ee5\u4e0b\u306e\u901a\u308a\u3067\u3042\u308b\u3002<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>\u65e5\u672c\u306e\u72ec\u81ea\u6027\uff1a<\/strong> 2014\u5e74\u306e\u6cd5\u6539\u6b63\u306b\u3088\u308a\u3001\u4e16\u754c\u306b\u5148\u99c6\u3051\u3066\u300c\u6761\u4ef6\u53ca\u3073\u671f\u9650\u4ed8\u627f\u8a8d\u5236\u5ea6\u300d\u3092\u5c0e\u5165\u3002\u8fc5\u901f\u306a\u60a3\u8005\u30a2\u30af\u30bb\u30b9\u3068\u5373\u6642\u306e\u4fdd\u967a\u9069\u7528\u3092\u5f37\u307f\u3068\u3059\u308b\u4e00\u65b9\u3001\u671f\u9650\u5185\u306e\u6709\u52b9\u6027\u78ba\u8a8d\u3084\u9ad8\u984d\u306a\u516c\u7684\u533b\u7642\u8cbb\u306e\u652f\u51fa\u304c\u8ab2\u984c\u3068\u306a\u3063\u3066\u3044\u308b\u3002<\/li>\n\n\n\n<li><strong>\u30b0\u30ed\u30fc\u30d0\u30eb\u5e02\u5834\u306e\u62e1\u5927\uff1a<\/strong> 2025\u5e74\u672b\u304b\u30892026\u5e74\u306b\u304b\u3051\u3066\u627f\u8a8d\u54c1\u76ee\u6570\u306f\u5927\u5e45\u306b\u5897\u52a0\u3059\u308b\u898b\u8fbc\u307f\u3002\u65e5\u672c\u306f\u7d0422-23\u54c1\u76ee\u3001\u7c73\u56fd\u306f48\u54c1\u76ee\uff082025\u5e7412\u6708\u6642\u70b9\uff09\u306b\u9054\u3059\u308b\u3068\u4e88\u6e2c\u3055\u308c\u3066\u3044\u308b\u3002<\/li>\n\n\n\n<li><strong>\u6280\u8853\u306e\u30d1\u30e9\u30c0\u30a4\u30e0\u30b7\u30d5\u30c8\uff1a<\/strong> \u5f93\u6765\u306eex vivo\u6cbb\u7642\u304b\u3089\u3001CRISPR\/Cas9\u3092\u7528\u3044\u305fin vivo\u907a\u4f1d\u5b50\u6cbb\u7642\u3001mRNA\u3001\u3055\u3089\u306b\u306fN=1\uff08\u500b\u5225\u5316\u533b\u7642\uff09\u3078\u306e\u8ee2\u63db\u304c\u9032\u884c\u3057\u3066\u3044\u308b\u3002<\/li>\n\n\n\n<li><strong>\u5546\u7528\u5316\u306e\u58c1\uff1a<\/strong> \u6b27\u5dde\uff08EMA\uff09\u3067\u306f\u627f\u8a8d\u5f8c\u306e\u88fd\u54c1\u64a4\u9000\u304c\u7d0430%\u306b\u9054\u3057\u3066\u304a\u308a\u3001HTA\uff08\u533b\u7642\u6280\u8853\u8a55\u4fa1\uff09\u3084\u85ac\u4fa1\u4ea4\u6e09\u3001\u88fd\u9020\u30b3\u30b9\u30c8\u306e\u7ba1\u7406\u304c\u5e02\u5834\u5b58\u7d9a\u306e\u9375\u3068\u306a\u3063\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. \u65e5\u672c\u306e\u898f\u5236\u67a0\u7d44\u307f\u3068\u5e02\u5834\u5c55\u671b<\/h2>\n\n\n\n<h3 class=\"wp-block-heading\">1.1 \u6cd5\u7684\u57fa\u76e4\u3068\u627f\u8a8d\u30b7\u30b9\u30c6\u30e0<\/h3>\n\n\n\n<p>\u65e5\u672c\u306e\u518d\u751f\u533b\u7642\u306f\u3001\u4e3b\u306b\u300c\u518d\u751f\u533b\u7642\u7b49\u5b89\u5168\u6027\u78ba\u4fdd\u6cd5\u300d\u3068\u300c\u533b\u85ac\u54c1\u533b\u7642\u6a5f\u5668\u7b49\u6cd5\uff08\u85ac\u6a5f\u6cd5\uff09\u300d\u306e\u4e8c\u6bb5\u69cb\u3048\u3067\u898f\u5236\u3055\u308c\u3066\u3044\u308b\u3002<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>\u6761\u4ef6\u53ca\u3073\u671f\u9650\u4ed8\u627f\u8a8d\u5236\u5ea6\uff1a<\/strong> \u5747\u4e00\u3067\u306a\u3044\u518d\u751f\u533b\u7642\u7b49\u88fd\u54c1\u306e\u7279\u6027\u3092\u8003\u616e\u3057\u3001\u5b89\u5168\u6027\u304c\u78ba\u8a8d\u3055\u308c\u6709\u52b9\u6027\u304c\u63a8\u5b9a\u3055\u308c\u305f\u6bb5\u968e\u3067\u3001\u6700\u95777\u5e74\u306e\u671f\u9650\u4ed8\u304d\u3067\u65e9\u671f\u627f\u8a8d\u3059\u308b\u5236\u5ea6\u3002<\/li>\n\n\n\n<li><strong>\u4fdd\u967a\u9069\u7528\uff1a<\/strong> \u627f\u8a8d\u5f8c\u3001\u5373\u5ea7\u306b\u56fd\u6c11\u5065\u5eb7\u4fdd\u967a\uff08NHI\uff09\u306b\u9069\u7528\u3055\u308c\u308b\u70b9\u304c\u56fd\u969b\u7684\u306a\u6bd4\u8f03\u512a\u4f4d\u6027\u3068\u306a\u3063\u3066\u3044\u308b\u3002<\/li>\n<\/ul>\n\n\n\n<h3 class=\"wp-block-heading\">1.2 \u8fd1\u5e74\u306e\u627f\u8a8d\u52d5\u5411\u30682026\u5e74\u4e88\u6e2c<\/h3>\n\n\n\n<p>\u65e5\u672c\u306b\u304a\u3051\u308b\u627f\u8a8d\u54c1\u76ee\u6570\u306f\u30012026\u5e743\u6708\u307e\u3067\u306b22\u301c23\u54c1\u76ee\u306b\u9054\u3059\u308b\u898b\u8fbc\u307f\u3067\u3042\u308b\u3002<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>\u4e3b\u8981\u306a\u30de\u30a4\u30eb\u30b9\u30c8\u30fc\u30f3\uff1a<\/strong>\n<ul class=\"wp-block-list\">\n<li><strong>iPS\u7d30\u80de\u7531\u6765\u88fd\u54c1\uff1a<\/strong> CiRA\uff08\u4eac\u90fd\u5927\u5b66\uff09\u3084Heartseed\u793e\u306b\u3088\u308biPS\u7d30\u80de\u7531\u6765\u5fc3\u7b4b\u7d30\u80de\u7b49\u306e\u627f\u8a8d\u304c\u30012025\u5e74\u304b\u30892026\u5e74\u306b\u304b\u3051\u3066\u671f\u5f85\u3055\u308c\u3066\u3044\u308b\u3002<\/li>\n\n\n\n<li><strong>eCTD v4.0\uff1a<\/strong> PMDA\u306f\u4e16\u754c\u306b\u5148\u99c6\u3051\u3066\u53d7\u4ed8\u3092\u958b\u59cb\u3057\u3066\u304a\u308a\u30012026\u5e744\u6708\u4ee5\u964d\u306e\u7533\u8acb\u304b\u3089\u7fa9\u52d9\u5316\u3055\u308c\u308b\u4e88\u5b9a\u3067\u3042\u308b\u3002\u3053\u308c\u306f\u898f\u5236\u5b9f\u88c5\u306b\u304a\u3051\u308b\u7a00\u5c11\u306a\u5148\u884c\u4e8b\u4f8b\u3067\u3042\u308b\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\">2. \u7c73\u56fd\uff08FDA\uff09\u304a\u3088\u3073\u6b27\u5dde\uff08EMA\uff09\u306e\u898f\u5236\u69cb\u9020<\/h2>\n\n\n\n<h3 class=\"wp-block-heading\">2.1 \u7c73\u56fd\uff1aFDA\u306e\u6226\u7565\u7684\u518d\u7de8<\/h3>\n\n\n\n<p>FDA\u306f\u518d\u751f\u533b\u7642\uff08CGT: Cell and Gene Therapy\uff09\u306e\u6025\u5897\u306b\u5bfe\u5fdc\u3059\u308b\u305f\u3081\u3001\u898f\u5236\u4f53\u5236\u3092\u5f37\u5316\u3057\u3066\u3044\u308b\u3002<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>OTP\uff08Office of Therapeutic Products\uff09\u306e\u8a2d\u7acb\uff1a<\/strong> 2023\u5e74\u3001\u5f93\u6765\u306eOTAT\u304b\u3089OTP\u3078\u3068\u7d44\u7e54\u3092\u6607\u683c\u30fb\u62e1\u5145\u30026\u3064\u306e\u30ec\u30d3\u30e5\u30fc\u90e8\u9580\u3092\u64c1\u3057\u30012024\u5e74\u306b\u306f\u7d042,500\u4ef6\u306eIND\uff08\u6cbb\u9a13\u5c4a\uff09\u3092\u7ba1\u7406\u3059\u308b\u898f\u6a21\u306b\u9054\u3057\u3066\u3044\u308b\u3002<\/li>\n\n\n\n<li><strong>RMAT\uff08\u518d\u751f\u533b\u7642\u5148\u9032\u6cbb\u7642\uff09\u6307\u5b9a\uff1a<\/strong> 2017\u5e74\u304b\u30892025\u5e74\u307e\u3067\u306b\u7d2f\u8a08193\u4ef6\u304c\u8a8d\u5b9a\u3055\u308c\u3001\u305d\u306e\u3046\u306173%\u304c\u627f\u8a8d\u30d7\u30ed\u30bb\u30b9\u306b\u79fb\u884c\u3057\u3066\u3044\u308b\u3002<\/li>\n\n\n\n<li><strong>\u4e3b\u8981\u627f\u8a8d\u54c1\u76ee\uff082023-2024\uff09\uff1a<\/strong> \u30b2\u30ce\u30e0\u7de8\u96c6\u3092\u7528\u3044\u305f\u300cCasgevy\u300d\u3084\u300cLyfgenia\u300d\u3001TCR-T\u7d30\u80de\u7642\u6cd5\u306e\u300cTecelra\u300d\u306a\u3069\u304c\u76f8\u6b21\u3044\u3067\u627f\u8a8d\u3055\u308c\u305f\u3002<\/li>\n<\/ul>\n\n\n\n<h3 class=\"wp-block-heading\">2.2 \u6b27\u5dde\uff1aATMP\u898f\u5236\u3068\u5e02\u5834\u306e\u73fe\u5b9f<\/h3>\n\n\n\n<p>\u6b27\u5dde\u3067\u306f\u300cATMP\uff08\u5148\u9032\u533b\u7642\u533b\u85ac\u54c1\uff09\u300d\u3068\u3057\u30664\u3064\u306e\u30ab\u30c6\u30b4\u30ea\u30fc\uff08GTMP, sCTMP, TEP, \u8907\u5408ATMP\uff09\u3067\u5206\u985e\u30fb\u7ba1\u7406\u3055\u308c\u3066\u3044\u308b\u3002<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>Hospital Exemption\uff08\u75c5\u9662\u514d\u9664\uff09\uff1a<\/strong> \u7279\u5b9a\u306e\u65bd\u8a2d\u5185\u3067\u306e\u975e\u5b9a\u578b\u7684\u306a\u4f7f\u7528\u3092\u8a8d\u3081\u308b\u4f8b\u5916\u898f\u5b9a\u304c\u3042\u308b\u304c\u3001\u3053\u308c\u304c\u5546\u7528\u88fd\u54c1\u306e\u5e02\u5834\u6d78\u900f\u3092\u59a8\u3052\u308b\u8981\u56e0\u306b\u3082\u306a\u3063\u3066\u3044\u308b\u3002<\/li>\n\n\n\n<li><strong>\u5e02\u5834\u64a4\u9000\u306e\u73fe\u72b6\uff1a<\/strong> 2024\u5e7411\u6708\u6642\u70b9\u3067\u627f\u8a8d\u54c1\u76ee\u306f27\u54c1\u76ee\u3060\u304c\u3001\u627f\u8a8d\u5f8c\u306e\u64a4\u9000\u7387\u304c\u9ad8\u3044\u3002\n<ul class=\"wp-block-list\">\n<li><strong>\u64a4\u9000\u4e8b\u4f8b\uff1a<\/strong> Glybera\uff082017\u5e74\uff09\u3001Zynteglo\uff082022\u5e74\uff09\u3001Alofisel\uff082024\u5e7412\u6708\u898b\u8fbc\u307f\uff09\u306a\u3069\u3002<\/li>\n\n\n\n<li><strong>\u8981\u56e0\uff1a<\/strong> HTA\u306b\u3088\u308b\u7d4c\u6e08\u6027\u8a55\u4fa1\u306e\u53b3\u683c\u5316\u3001\u88fd\u9020\u30b3\u30b9\u30c8\u306b\u898b\u5408\u308f\u306a\u3044\u85ac\u4fa1\u8a2d\u5b9a\u3001\u78ba\u8a3c\u7684\u8a66\u9a13\uff08Phase III\uff09\u306e\u5931\u6557\u304c\u4e3b\u306a\u7406\u7531\u3067\u3042\u308b\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\">3. \u88fd\u9020\u7ba1\u7406\u3068\u54c1\u8cea\u4fdd\u8a3c\uff08GCTP\/CPC\uff09<\/h2>\n\n\n\n<h3 class=\"wp-block-heading\">3.1 GCTP\uff08\u518d\u751f\u533b\u7642\u7b49\u88fd\u54c1\u88fd\u9020\u7ba1\u7406\u53ca\u3073\u54c1\u8cea\u7ba1\u7406\u57fa\u6e96\uff09<\/h3>\n\n\n\n<p>\u518d\u751f\u533b\u7642\u7b49\u88fd\u54c1\u306e\u88fd\u9020\u306b\u306f\u3001\u5f93\u6765\u306e\u533b\u85ac\u54c1\uff08GMP\uff09\u3088\u308a\u3082\u53b3\u683c\u306aGCTP\u57fa\u6e96\u304c\u9069\u7528\u3055\u308c\u308b\u3002<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>\u69cb\u9020\u8a2d\u5099\uff08CPC\uff09\uff1a<\/strong> \u7121\u83cc\u72b6\u614b\u3092\u7dad\u6301\u3059\u308b\u305f\u3081\u306eCell Processing Center\uff08\u7d30\u80de\u57f9\u990a\u52a0\u5de5\u65bd\u8a2d\uff09\u306e\u8a2d\u7f6e\u304c\u5fc5\u9808\u3002<\/li>\n\n\n\n<li><strong>SOP\uff08\u6a19\u6e96\u4f5c\u696d\u624b\u9806\u66f8\uff09\uff1a<\/strong> SOP\u306b\u57fa\u3065\u304f\u30d0\u30ea\u30c7\u30fc\u30b7\u30e7\u30f3\u3001\u885b\u751f\u7ba1\u7406\u3001\u6587\u66f8\u5316\u304c\u53b3\u683c\u306b\u6c42\u3081\u3089\u308c\u308b\u3002<\/li>\n<\/ul>\n\n\n\n<h3 class=\"wp-block-heading\">3.2 \u65bd\u8a2d\u306e\u5f62\u614b\u3068\u9078\u629e<\/h3>\n\n\n\n<figure class=\"wp-block-table\"><table class=\"has-fixed-layout\"><tbody><tr><td>\u65bd\u8a2d\u30bf\u30a4\u30d7<\/td><td>\u7279\u5fb4<\/td><td>\u4e8b\u4f8b<\/td><\/tr><tr><td><strong>\u9662\u5185CPC<\/strong><\/td><td>\u75c5\u9662\u304c\u904b\u55b6\u3002\u7814\u7a76\u958b\u767a\u3084\u6cbb\u9a13\u306b\u5f37\u307f\u3002<\/td><td>\u6176\u61c9\u7fa9\u587e\u5927\u5b66\u75c5\u9662\uff08KHCPC\uff09\u306a\u3069<\/td><\/tr><tr><td><strong>\u5546\u7528CPC (\u5916\u90e8)<\/strong><\/td><td>\u5c02\u9580\u306e\u7b2c\u4e09\u8005\u6a5f\u95a2\u3002\u30b9\u30b1\u30fc\u30eb\u30e1\u30ea\u30c3\u30c8\u3068\u6a19\u6e96\u5316\u3055\u308c\u305f\u30d7\u30ed\u30bb\u30b9\u3092\u63d0\u4f9b\u3002<\/td><td>J-TEC\uff08\u30b8\u30e3\u30d1\u30f3\u30fb\u30c6\u30a3\u30c3\u30b7\u30e5\u30a8\u30f3\u30b8\u30cb\u30a2\u30ea\u30f3\u30b0\uff09\u306a\u3069<\/td><\/tr><\/tbody><\/table><\/figure>\n\n\n\n<h3 class=\"wp-block-heading\">3.3 \u65b0\u305f\u306a\u54c1\u8cea\u7ba1\u7406\u624b\u6cd5<\/h3>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>QbD (Quality by Design)\uff1a<\/strong> 2025\u5e743\u6708\u306b\u306fQ&amp;A\u306e\u767a\u51fa\u304c\u4e88\u5b9a\u3055\u308c\u3066\u304a\u308a\u3001AI\u3084\u30c7\u30b8\u30bf\u30eb\u30c4\u30a4\u30f3\u3092\u6d3b\u7528\u3057\u305f\u88fd\u9020\u30d7\u30ed\u30bb\u30b9\u306e\u6700\u9069\u5316\u304c\u8b70\u8ad6\u3055\u308c\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\">4. \u627f\u8a8d\u5236\u5ea6\u306e\u6bd4\u8f03\u3068\u8ab2\u984c<\/h2>\n\n\n\n<p>\u5404\u56fd\u306e\u6761\u4ef6\u4ed8\u627f\u8a8d\u5236\u5ea6\u306b\u306f\u3001\u904b\u7528\u306e\u5b9f\u614b\u306b\u5dee\u7570\u304c\u3042\u308b\u3002<\/p>\n\n\n\n<figure class=\"wp-block-table\"><table class=\"has-fixed-layout\"><tbody><tr><td>\u9805\u76ee<\/td><td>\u65e5\u672c (\u6761\u4ef6\u53ca\u3073\u671f\u9650\u4ed8)<\/td><td>\u7c73\u56fd (Accelerated Approval)<\/td><td>\u6b27\u5dde (Conditional Marketing 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class=\"wp-block-heading\">5. \u5c06\u6765\u306e\u6280\u8853\u30c8\u30ec\u30f3\u30c9\u3068\u898f\u5236\u306e\u65b9\u5411\u6027\uff082025-2026\uff09<\/h2>\n\n\n\n<h3 class=\"wp-block-heading\">5.1 \u6280\u8853\u7684\u8ee2\u63db\u70b9<\/h3>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>In vivo\u907a\u4f1d\u5b50\u6cbb\u7642\uff1a<\/strong> CRISPR\/Cas9\u306a\u3069\u306e\u30c4\u30fc\u30eb\u3092\u7528\u3044\u3001\u4f53\u5185\u3067\u76f4\u63a5\u907a\u4f1d\u5b50\u3092\u6539\u5909\u3059\u308b\u6cbb\u7642\u3002<\/li>\n\n\n\n<li><strong>N=1 \u500b\u5225\u5316\u533b\u7642\uff1a<\/strong> \u8d85\u5e0c\u5c11\u75be\u60a3\u306b\u5bfe\u3059\u308b\u3001\u60a3\u8005\u4e00\u4eba\u3072\u3068\u308a\u306b\u6700\u9069\u5316\u3055\u308c\u305f\u88fd\u54c1\uff08N=1\uff09\u306e\u898f\u5236\u67a0\u7d44\u307f\u304c\u691c\u8a0e\u3055\u308c\u3066\u3044\u308b\u3002<\/li>\n\n\n\n<li><strong>mRNA\u6280\u8853\uff1a<\/strong> 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