{"id":85718,"date":"2026-01-06T03:36:57","date_gmt":"2026-01-05T18:36:57","guid":{"rendered":"https:\/\/www.marketresearch.co.jp\/insights\/huntingtons-disease-treatment-mordor\/"},"modified":"2026-04-03T01:35:46","modified_gmt":"2026-04-02T16:35:46","slug":"huntingtons-disease-treatment-mordor","status":"publish","type":"post","link":"https:\/\/www.marketresearch.co.jp\/insights\/huntingtons-disease-treatment-mordor\/","title":{"rendered":"\u30cf\u30f3\u30c1\u30f3\u30c8\u30f3\u75c5\u6cbb\u7642\u5e02\u5834\uff1a\u5e02\u5834\u898f\u6a21\u30fb\u30b7\u30a7\u30a2\u5206\u6790\u3001\u6210\u9577\u52d5\u5411\u30fb\u4e88\u6e2c (2025-2030\u5e74)"},"content":{"rendered":"<p><img decoding=\"async\" src=\"https:\/\/www.marketresearch.co.jp\/insights\/wp-content\/uploads\/2026\/01\/huntingtons-disease-therapy_1646747014936_Market_Players.webp\" alt=\"Chart\" class=\"aligncenter size-full\" \/><\/p>\n<p><img decoding=\"async\" src=\"https:\/\/www.marketresearch.co.jp\/insights\/wp-content\/uploads\/2026\/01\/huntingtons-disease-treatment-size-image-1751602393597.webp\" alt=\"Chart\" class=\"aligncenter size-full\" \/><\/p>\n<p class=\"toc-level-1 toc-content-heading\" id=\"toc-content-heading-0\">\n    1. \u306f\u3058\u3081\u306b<\/p>\n<ul>\n<li class=\"toc-level-2\">\n          1.1 \u8abf\u67fb\u306e\u524d\u63d0\u3068\u5e02\u5834\u306e\u5b9a\u7fa9<\/p>\n<\/li>\n<li class=\"toc-level-2\">\n          1.2 \u8abf\u67fb\u7bc4\u56f2<\/p>\n<\/li>\n<\/ul>\n<p class=\"toc-level-1 toc-content-heading\" id=\"toc-content-heading-1\">\n    2. \u8abf\u67fb\u65b9\u6cd5<\/p>\n<p class=\"toc-level-1 toc-content-heading\" id=\"toc-content-heading-2\">\n    3. \u30a8\u30b0\u30bc\u30af\u30c6\u30a3\u30d6\u30b5\u30de\u30ea\u30fc<\/p>\n<p class=\"toc-level-1 toc-content-heading\" id=\"toc-content-heading-3\">\n    4. \u5e02\u5834\u6982\u6cc1<\/p>\n<ul>\n<li class=\"toc-level-2\">\n          4.1 \u5e02\u5834\u6982\u8981<\/p>\n<\/li>\n<li class=\"toc-level-2\">\n          4.2 \u5e02\u5834\u306e\u63a8\u9032\u8981\u56e0<\/p>\n<ul>\n<li class=\"toc-level-3\">\n          4.2.1 \u9ad8\u9f62\u8005\u5c64\u306b\u304a\u3051\u308b\u767a\u751f\u7387\u306e\u5897\u52a0<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          4.2.2 HTT\u4f4e\u4e0b\u81e8\u5e8a\u30d1\u30a4\u30d7\u30e9\u30a4\u30f3\u306e\u753b\u671f\u7684\u306a\u9032\u6b69\u306e\u52a0\u901f<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          4.2.3 \u30a2\u30b8\u30a2\u306b\u304a\u3051\u308b1\u65e51\u56deVMAT2\u963b\u5bb3\u5264\u306e\u5546\u696d\u7684\u767a\u58f2<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          4.2.4 \u7c73\u56fd\/EU\u306b\u304a\u3051\u308b\u30d5\u30a1\u30b9\u30c8\u30c8\u30e9\u30c3\u30af\u304a\u3088\u3073\u5e0c\u5c11\u75be\u75c5\u7528\u533b\u85ac\u54c1\u6307\u5b9a<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          4.2.5 AI\u3092\u6d3b\u7528\u3057\u305f\u30d0\u30a4\u30aa\u30de\u30fc\u30ab\u30fc\u767a\u898b\u306b\u3088\u308b\u6cbb\u9a13\u6210\u529f\u7387\u306e\u5411\u4e0a<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          4.2.6 \u03b2\u906e\u65ad\u85ac\u306e\u518d\u5229\u7528\u306b\u3088\u308b\u75be\u60a3\u4fee\u98fe\u30b7\u30b0\u30ca\u30eb\u306e\u767a\u73fe<\/p>\n<\/li>\n<\/ul>\n<\/li>\n<li class=\"toc-level-2\">\n          4.3 \u5e02\u5834\u306e\u963b\u5bb3\u8981\u56e0<\/p>\n<ul>\n<li class=\"toc-level-3\">\n          4.3.1 \u907a\u4f1d\u5b50\u6cbb\u7642\u304a\u3088\u3073\u7d30\u80de\u6cbb\u7642\u306e\u8d85\u9ad8\u30b3\u30b9\u30c8<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          4.3.2 \u5317\u7c73\u304a\u3088\u3073EU\u4ee5\u5916\u306e\u5c02\u9580\u30bb\u30f3\u30bf\u30fc\u306e\u9650\u5b9a<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          4.3.3 \u5bfe\u75c7\u7642\u6cd5\u3068\u3057\u3066\u306e\u591a\u5264\u4f75\u7528\u7642\u6cd5\u3078\u306e\u4e0d\u5341\u5206\u306a\u9075\u5b88<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          4.3.4 \u795e\u7d4c\u7d44\u7e54\u306b\u304a\u3051\u308bCRISPR\u306e\u30c7\u30fc\u30bf\u5b89\u5168\u6027\u306b\u95a2\u3059\u308b\u61f8\u5ff5<\/p>\n<\/li>\n<\/ul>\n<\/li>\n<li class=\"toc-level-2\">\n          4.4 \u30dd\u30fc\u30bf\u30fc\u306e5\u3064\u306e\u529b<\/p>\n<ul>\n<li class=\"toc-level-3\">\n          4.4.1 \u65b0\u898f\u53c2\u5165\u8005\u306e\u8105\u5a01<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          4.4.2 \u8cb7\u3044\u624b\u306e\u4ea4\u6e09\u529b<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          4.4.3 \u4f9b\u7d66\u8005\u306e\u4ea4\u6e09\u529b<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          4.4.4 \u4ee3\u66ff\u54c1\u306e\u8105\u5a01<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          4.4.5 \u7af6\u4e89\u306e\u6fc0\u3057\u3055<\/p>\n<\/li>\n<\/ul>\n<\/li>\n<\/ul>\n<p class=\"toc-level-1 toc-content-heading\" id=\"toc-content-heading-4\">\n    5. \u5e02\u5834\u898f\u6a21\u3068\u6210\u9577\u4e88\u6e2c\uff08\u91d1\u984d\u3001\u7c73\u30c9\u30eb\uff09<\/p>\n<ul>\n<li class=\"toc-level-2\">\n          5.1 \u6cbb\u7642\u30bf\u30a4\u30d7\u5225<\/p>\n<ul>\n<li class=\"toc-level-3\">\n          5.1.1 \u5bfe\u75c7\u7642\u6cd5<\/p>\n<\/li>\n<li class=\"toc-level-4\">\n          5.1.1.1 VMAT2\u963b\u5bb3\u5264<\/p>\n<\/li>\n<li class=\"toc-level-4\">\n          5.1.1.2 \u6297\u30c9\u30d1\u30df\u30f3\u4f5c\u52d5\u85ac\uff08\u6297\u7cbe\u795e\u75c5\u85ac\uff09<\/p>\n<\/li>\n<li class=\"toc-level-4\">\n          5.1.1.3 \u6297\u3046\u3064\u85ac &amp; \u6297\u4e0d\u5b89\u85ac<\/p>\n<\/li>\n<li class=\"toc-level-4\">\n          5.1.1.4 \u6297\u3051\u3044\u308c\u3093\u85ac \/ \u305d\u306e\u4ed6<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          5.1.2 \u75be\u60a3\u4fee\u98fe\u7642\u6cd5<\/p>\n<\/li>\n<li class=\"toc-level-4\">\n          5.1.2.1 \u907a\u4f1d\u5b50\u30b5\u30a4\u30ec\u30f3\u30b7\u30f3\u30b0 \/ ASO<\/p>\n<\/li>\n<li class=\"toc-level-4\">\n          5.1.2.2 \u907a\u4f1d\u5b50\u6cbb\u7642\uff08AAV\u3001LNP\u306a\u3069\uff09<\/p>\n<\/li>\n<li class=\"toc-level-4\">\n          5.1.2.3 \u5e79\u7d30\u80de\u6cbb\u7642<\/p>\n<\/li>\n<li class=\"toc-level-4\">\n          5.1.2.4 \u4f4e\u5206\u5b50HTT\u4fee\u98fe\u85ac<\/p>\n<\/li>\n<\/ul>\n<\/li>\n<li class=\"toc-level-2\">\n          5.2 \u6295\u4e0e\u7d4c\u8def\u5225<\/p>\n<ul>\n<li class=\"toc-level-3\">\n          5.2.1 \u7d4c\u53e3<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          5.2.2 \u9759\u8108\u5185<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          5.2.3 \u9ac4\u8154\u5185<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          5.2.4 \u76ae\u4e0b<\/p>\n<\/li>\n<\/ul>\n<\/li>\n<li class=\"toc-level-2\">\n          5.3 \u6d41\u901a\u30c1\u30e3\u30cd\u30eb\u5225<\/p>\n<ul>\n<li class=\"toc-level-3\">\n          5.3.1 \u75c5\u9662\u85ac\u5c40<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          5.3.2 \u5c0f\u58f2\u85ac\u5c40<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          5.3.3 \u5c02\u9580\u85ac\u5c40 &amp; \u30aa\u30f3\u30e9\u30a4\u30f3\u85ac\u5c40<\/p>\n<\/li>\n<\/ul>\n<\/li>\n<li class=\"toc-level-2\">\n          5.4 \u75be\u60a3\u306e\u75c5\u671f\u5225<\/p>\n<ul>\n<li class=\"toc-level-3\">\n          5.4.1 \u524d\u5146\u671f \/ \u524d\u99c6\u671f<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          5.4.2 \u521d\u671f<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          5.4.3 \u4e2d\u671f<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          5.4.4 \u5f8c\u671f<\/p>\n<\/li>\n<\/ul>\n<\/li>\n<li class=\"toc-level-2\">\n          5.5 \u5730\u57df\u5225<\/p>\n<ul>\n<li class=\"toc-level-3\">\n          5.5.1 \u5317\u7c73<\/p>\n<\/li>\n<li class=\"toc-level-4\">\n          5.5.1.1 \u7c73\u56fd<\/p>\n<\/li>\n<li class=\"toc-level-4\">\n          5.5.1.2 \u30ab\u30ca\u30c0<\/p>\n<\/li>\n<li class=\"toc-level-4\">\n          5.5.1.3 \u30e1\u30ad\u30b7\u30b3<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          5.5.2 \u30e8\u30fc\u30ed\u30c3\u30d1<\/p>\n<\/li>\n<li class=\"toc-level-4\">\n          5.5.2.1 \u30c9\u30a4\u30c4<\/p>\n<\/li>\n<li class=\"toc-level-4\">\n          5.5.2.2 \u82f1\u56fd<\/p>\n<\/li>\n<li class=\"toc-level-4\">\n          5.5.2.3 \u30d5\u30e9\u30f3\u30b9<\/p>\n<\/li>\n<li class=\"toc-level-4\">\n          5.5.2.4 \u30a4\u30bf\u30ea\u30a2<\/p>\n<\/li>\n<li class=\"toc-level-4\">\n          5.5.2.5 \u30b9\u30da\u30a4\u30f3<\/p>\n<\/li>\n<li class=\"toc-level-4\">\n          5.5.2.6 \u305d\u306e\u4ed6\u306e\u30e8\u30fc\u30ed\u30c3\u30d1<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          5.5.3 \u30a2\u30b8\u30a2\u592a\u5e73\u6d0b<\/p>\n<\/li>\n<li class=\"toc-level-4\">\n          5.5.3.1 \u4e2d\u56fd<\/p>\n<\/li>\n<li class=\"toc-level-4\">\n          5.5.3.2 \u65e5\u672c<\/p>\n<\/li>\n<li class=\"toc-level-4\">\n          5.5.3.3 \u30a4\u30f3\u30c9<\/p>\n<\/li>\n<li class=\"toc-level-4\">\n          5.5.3.4 \u97d3\u56fd<\/p>\n<\/li>\n<li class=\"toc-level-4\">\n          5.5.3.5 \u30aa\u30fc\u30b9\u30c8\u30e9\u30ea\u30a2<\/p>\n<\/li>\n<li class=\"toc-level-4\">\n          5.5.3.6 \u305d\u306e\u4ed6\u306e\u30a2\u30b8\u30a2\u592a\u5e73\u6d0b<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          5.5.4 \u4e2d\u6771\u304a\u3088\u3073\u30a2\u30d5\u30ea\u30ab<\/p>\n<\/li>\n<li class=\"toc-level-4\">\n          5.5.4.1 GCC<\/p>\n<\/li>\n<li class=\"toc-level-4\">\n          5.5.4.2 \u5357\u30a2\u30d5\u30ea\u30ab<\/p>\n<\/li>\n<li class=\"toc-level-4\">\n          5.5.4.3 \u305d\u306e\u4ed6\u306e\u4e2d\u6771\u304a\u3088\u3073\u30a2\u30d5\u30ea\u30ab<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          5.5.5 \u5357\u7c73<\/p>\n<\/li>\n<li class=\"toc-level-4\">\n          5.5.5.1 \u30d6\u30e9\u30b8\u30eb<\/p>\n<\/li>\n<li class=\"toc-level-4\">\n          5.5.5.2 \u30a2\u30eb\u30bc\u30f3\u30c1\u30f3<\/p>\n<\/li>\n<li class=\"toc-level-4\">\n          5.5.5.3 \u305d\u306e\u4ed6\u306e\u5357\u7c73<\/p>\n<\/li>\n<\/ul>\n<\/li>\n<\/ul>\n<p class=\"toc-level-1 toc-content-heading\" id=\"toc-content-heading-5\">\n    6. \u7af6\u5408\u72b6\u6cc1<\/p>\n<ul>\n<li class=\"toc-level-2\">\n          6.1 \u5e02\u5834\u96c6\u4e2d\u5ea6<\/p>\n<\/li>\n<li class=\"toc-level-2\">\n          6.2 \u5e02\u5834\u30b7\u30a7\u30a2\u5206\u6790<\/p>\n<\/li>\n<li class=\"toc-level-2\">\n          6.3 \u4f01\u696d\u30d7\u30ed\u30d5\u30a1\u30a4\u30eb\uff08\u30b0\u30ed\u30fc\u30d0\u30eb\u30ec\u30d9\u30eb\u306e\u6982\u8981\u3001\u5e02\u5834\u30ec\u30d9\u30eb\u306e\u6982\u8981\u3001\u4e3b\u8981\u30bb\u30b0\u30e1\u30f3\u30c8\u3001\u5229\u7528\u53ef\u80fd\u306a\u8ca1\u52d9\u60c5\u5831\u3001\u6226\u7565\u60c5\u5831\u3001\u4e3b\u8981\u4f01\u696d\u306e\u5e02\u5834\u30e9\u30f3\u30af\/\u30b7\u30a7\u30a2\u3001\u88fd\u54c1&amp;\u30b5\u30fc\u30d3\u30b9\u3001\u304a\u3088\u3073\u6700\u8fd1\u306e\u52d5\u5411\u3092\u542b\u3080\uff09<\/p>\n<ul>\n<li class=\"toc-level-3\">\n          6.3.1 \u30d5\u30a1\u30a4\u30b6\u30fc\u682a\u5f0f\u4f1a\u793e<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          6.3.2 \u30c6\u30d0\u88fd\u85ac\u7523\u696d<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          6.3.3 \u30cb\u30e5\u30fc\u30ed\u30af\u30e9\u30a4\u30f3\u30fb\u30d0\u30a4\u30aa\u30b5\u30a4\u30a8\u30f3\u30b9<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          6.3.4 \u30a2\u30eb\u30ca\u30a4\u30e9\u30e0\u30fb\u30d5\u30a1\u30fc\u30de\u30b7\u30e5\u30fc\u30c6\u30a3\u30ab\u30eb\u30ba<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          6.3.5 \u30a2\u30a4\u30aa\u30cb\u30b9\u30fb\u30d5\u30a1\u30fc\u30de\u30b7\u30e5\u30fc\u30c6\u30a3\u30ab\u30eb\u30ba<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          6.3.6 \u30ed\u30b7\u30e5 \/ \u30b8\u30a7\u30cd\u30f3\u30c6\u30c3\u30af<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          6.3.7 \u30ce\u30d0\u30eb\u30c6\u30a3\u30b9AG<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          6.3.8 \u30e6\u30cb\u30ad\u30e5\u30a2<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          6.3.9 \u30b5\u30f3\u30ac\u30e2\u30fb\u30bb\u30e9\u30d4\u30e5\u30fc\u30c6\u30a3\u30af\u30b9<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          6.3.10 PTC\u30bb\u30e9\u30d4\u30e5\u30fc\u30c6\u30a3\u30af\u30b9<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          6.3.11 \u30d7\u30ea\u30ec\u30cb\u30a2\u30fb\u30bb\u30e9\u30d4\u30e5\u30fc\u30c6\u30a3\u30af\u30b9<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          6.3.12 \u30a6\u30a7\u30fc\u30d6\u30fb\u30e9\u30a4\u30d5\u30fb\u30b5\u30a4\u30a8\u30f3\u30b9<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          6.3.13 \u30bb\u30fc\u30b8\u30fb\u30bb\u30e9\u30d4\u30e5\u30fc\u30c6\u30a3\u30af\u30b9<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          6.3.14 \u30d0\u30a4\u30aa\u30b8\u30a7\u30f3\u682a\u5f0f\u4f1a\u793e<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          6.3.15 \u30eb\u30f3\u30c9\u30d9\u30c3\u30af<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          6.3.16 \u30a2\u30bc\u30d0\u30f3\u30fb\u30d5\u30a1\u30fc\u30de\u30b7\u30e5\u30fc\u30c6\u30a3\u30ab\u30eb\u30ba<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          6.3.17 \u30dc\u30a4\u30b8\u30e3\u30fc\u30fb\u30bb\u30e9\u30d4\u30e5\u30fc\u30c6\u30a3\u30af\u30b9<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          6.3.18 \u30a4\u30f3\u30c8\u30e9\u30d0\u30a4\u30aa<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          6.3.19 \u5354\u548c\u30ad\u30ea\u30f3<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          6.3.20 \u30f4\u30a1\u30af\u30b7\u30cb\u30c6\u30a3<\/p>\n<\/li>\n<\/ul>\n<\/li>\n<\/ul>\n<p class=\"toc-level-1 toc-content-heading\" id=\"toc-content-heading-6\">\n    7. \u5e02\u5834\u6a5f\u4f1a\u3068\u5c06\u6765\u5c55\u671b<\/p>\n","protected":false},"excerpt":{"rendered":"<p>\u30cf\u30f3\u30c1\u30f3\u30c8\u30f3\u75c5\u6cbb\u7642\u5e02\u5834\u306e\u6982\u8981<\/p>\n<p>\u5e02\u5834\u898f\u6a21\u3001\u30c8\u30ec\u30f3\u30c9\u3001\u30b7\u30a7\u30a2\u5206\u6790\uff082025\u5e74\uff5e2030\u5e74\uff09<\/p>\n<p>\u30cf\u30f3\u30c1\u30f3\u30c8\u30f3\u75c5\u6cbb\u7642\u5e02\u5834\u306f\u30012025\u5e74\u306b\u306f8.2\u5104\u7c73\u30c9\u30eb\u3068\u8a55\u4fa1\u3055\u308c\u30012030\u5e74\u307e\u3067\u306b12.2\u5104\u7c73\u30c9\u30eb\u306b\u9054\u3059\u308b\u3068\u4e88\u6e2c\u3055\u308c\u3066\u304a\u308a\u30012025\u5e74\u304b\u30892030\u5e74\u306b\u304b\u3051\u3066\u5e74\u5e73\u5747\u6210\u9577\u7387\uff08CAGR\uff098.23%\u3067\u6210\u9577\u3059\u308b\u898b\u8fbc\u307f\u3067\u3059\u3002\u3053\u306e\u5e02\u5834\u62e1\u5927\u306f\u3001\u753b\u671f\u7684\u306a\u907a\u4f1d\u5b50\u6cbb\u7642\u306e\u9032\u5c55\u3001\u8fc5\u901f\u306a\u898f\u5236\u627f\u8a8d\u7d4c\u8def\u3001\u6295\u8cc7\u5bb6\u306e\u4fe1\u983c\u306e\u9ad8\u307e\u308a\u306b\u652f\u3048\u3089\u308c\u3001\u5bfe\u75c7\u7642\u6cd5\u304b\u3089\u75be\u60a3\u4fee\u98fe\u7642\u6cd5\u3078\u306e\u69cb\u9020\u7684\u306a\u8ee2\u63db\u3092\u793a\u3057\u3066\u3044\u307e\u3059\u3002\u30ce\u30d0\u30eb\u30c6\u30a3\u30b9\u306b\u3088\u308bPTC 518\u306e29\u5104\u7c73\u30c9\u30eb\u3067\u306e\u8cb7\u53ce\u306e\u3088\u3046\u306a\u5927\u578b\u53d6\u5f15\u306f\u3001\u30cf\u30f3\u30c1\u30f3\u30c1\u30f3\u30bf\u30f3\u30d1\u30af\u8cea\u4f4e\u4e0b\u7642\u6cd5\u304c\u5b9f\u9a13\u6bb5\u968e\u306e\u6709\u671b\u6027\u304b\u3089\u5546\u696d\u7684\u73fe\u5b9f\u3078\u3068\u79fb\u884c\u3057\u3066\u3044\u308b\u3053\u3068\u3092\u660e\u78ba\u306b\u793a\u3057\u3066\u3044\u307e\u3059\u3002\u540c\u6642\u306b\u3001VMAT2\u963b\u5bb3\u5264\u306e\u53ce\u76ca\u306f\u5f15\u304d\u7d9a\u304d\u5897\u52a0\u3057\u3001\u30c7\u30b8\u30bf\u30eb\u30d0\u30a4\u30aa\u30de\u30fc\u30ab\u30fc\u306f\u6cbb\u9a13\u30c7\u30b6\u30a4\u30f3\u3092\u6539\u5584\u3057\u3001\u907a\u4f1d\u5b50\u6cbb\u7642\u306e\u30b3\u30b9\u30c8\u3092\u6291\u5236\u3059\u308b\u305f\u3081\u306e\u9769\u65b0\u7684\u306a\u8cc7\u91d1\u8abf\u9054\u30e2\u30c7\u30eb\u304c\u767b\u5834\u3057\u3066\u3044\u307e\u3059\u3002\u30d7\u30e9\u30c3\u30c8\u30d5\u30a9\u30fc\u30e0\u304c\u4ee3\u66ff\u30a8\u30f3\u30c9\u30dd\u30a4\u30f3\u30c8\u306e\u691c\u8a3c\u3092\u9054\u6210\u3057\u3001\u5c02\u9580\u30bb\u30f3\u30bf\u30fc\u304c\u5897\u52a0\u3057\u3001\u5bfe\u75c7\u7642\u6cd5\u3068\u75be\u60a3\u4fee\u98fe\u7642\u6cd5\u306e\u4e21\u65b9\u306e\u5229\u70b9\u3092\u7d50\u3073\u3064\u3051\u308b\u4f75\u7528\u7642\u6cd5\u304c\u5177\u4f53\u5316\u3059\u308b\u306b\u3064\u308c\u3066\u3001\u4eca\u5f8c\u306e\u52e2\u3044\u306f\u5f15\u304d\u7d9a\u304d\u5f37\u3044\u3068\u4e88\u60f3\u3055\u308c\u307e\u3059\u3002<\/p>\n<p>\u4e3b\u8981\u306a\u30ec\u30dd\u30fc\u30c8\u306e\u30dd\u30a4\u30f3\u30c8<\/p>\n<p>*   \u6cbb\u7642\u30bf\u30a4\u30d7\u5225: 2024\u5e74\u306b\u306f\u5bfe\u75c7\u7642\u6cd5\u304c\u30cf\u30f3\u30c1\u30f3\u30c8\u30f3\u75c5\u6cbb\u7642\u5e02\u5834\u30b7\u30a7\u30a2\u306e62.55%\u3092\u5360\u3081\u307e\u3057\u305f\u304c\u3001\u75be\u60a3\u4fee\u98fe\u7642\u6cd5\u306f2030\u5e74\u307e\u3067\u306b16.25%\u306eCAGR\u3067\u6210\u9577\u3059\u308b\u3068\u4e88\u6e2c\u3055\u308c\u3066\u3044\u307e\u3059\u3002<br \/>\n*   \u6295\u4e0e\u7d4c\u8def\u5225: 2024\u5e74\u306b\u306f\u7d4c\u53e3\u88fd\u54c1\u304c\u30cf\u30f3\u30c1\u30f3\u30c8\u30f3\u75c5\u6cbb\u7642\u5e02\u5834\u898f\u6a21\u306e54.53%\u3092\u5360\u3081\u307e\u3057\u305f\u3002\u9759\u8108\u5185\u6295\u4e0e\u306f2025\u5e74\u304b\u30892030\u5e74\u306b\u304b\u3051\u306611.85%\u306eCAGR\u3067\u62e1\u5927\u3059\u308b\u3068\u4e88\u6e2c\u3055\u308c\u3066\u3044\u307e\u3059\u3002<br \/>\n*   \u6d41\u901a\u30c1\u30e3\u30cd\u30eb\u5225: 2024\u5e74\u306b\u306f\u75c5\u9662\u85ac\u5c40\u304c\u53ce\u76ca\u30b7\u30a7\u30a2\u306e55.12%\u3092\u7dad\u6301\u3057\u307e\u3057\u305f\u304c\u3001\u5c02\u9580\u85ac\u5c40\u304a\u3088\u3073\u30aa\u30f3\u30e9\u30a4\u30f3\u85ac\u5c40\u306f2030\u5e74\u307e\u3067\u306b\u6700\u3082\u901f\u304415.31%\u306eCAGR\u3067\u6210\u9577\u3059\u308b\u6e96\u5099\u304c\u6574\u3063\u3066\u3044\u307e\u3059\u3002<br \/>\n*   \u75c5\u671f\u5225: 2024\u5e74\u306b\u306f\u65e9\u671f\u60a3\u8005\u304c\u30cf\u30f3\u30c1\u30f3\u30c8\u30f3\u75c5\u6cbb\u7642\u5e02\u5834\u898f\u6a21\u306e40.63%\u3092\u5360\u3081\u307e\u3057\u305f\u304c\u3001\u767a\u75c7\u524d\u30b3\u30db\u30fc\u30c8\u306f2030\u5e74\u307e\u3067\u306b16.7%\u306eCAGR\u3067\u6025\u5897\u3059\u308b\u3068\u4e88\u6e2c\u3055\u308c\u3066\u3044\u307e\u3059\u3002<br \/>\n*   \u5730\u57df\u5225: 2024\u5e74\u306b\u306f\u5317\u7c73\u304c\u30cf\u30f3\u30c1\u30f3\u30c8\u30f3\u75c5\u6cbb\u7642\u5e02\u5834\u30b7\u30a7\u30a2\u306e38.63%\u3092\u5360\u3081\u307e\u3057\u305f\u3002\u30a2\u30b8\u30a2\u592a\u5e73\u6d0b\u5730\u57df\u306f2030\u5e74\u307e\u3067\u306b14.87%\u306eCAGR\u3067\u6210\u9577\u3059\u308b\u3068\u4e88\u6e2c\u3055\u308c\u3066\u3044\u307e\u3059\u3002<\/p>\n<p>\u4e16\u754c\u306e\u30cf\u30f3\u30c1\u30f3\u30c8\u30f3\u75c5\u6cbb\u7642\u5e02\u5834\u306e\u30c8\u30ec\u30f3\u30c9\u3068\u6d1e\u5bdf<\/p>\n<p>\u5e02\u5834\u306e\u63a8\u9032\u8981\u56e0<\/p>\n<p>*   \u9ad8\u9f62\u8005\u4eba\u53e3\u306b\u304a\u3051\u308b\u767a\u751f\u7387\u306e\u5897\u52a0\uff08CAGR\u3078\u306e\u5f71\u97ff: +1.2%\uff09: \u8a3a\u65ad\u7bc4\u56f2\u306e\u6539\u5584\u306b\u3088\u308a\u3001\u3088\u308a\u591a\u304f\u306e\u6669\u671f\u767a\u75c7\u4f8b\u304c\u767a\u898b\u3055\u308c\u3001\u4e16\u754c\u7684\u306a\u5e73\u5747\u5bff\u547d\u306e\u5ef6\u9577\u304c\u60a3\u8005\u30d7\u30fc\u30eb\u3092\u62e1\u5927\u3057\u3066\u3044\u307e\u3059\u3002\u5317\u7c73\u3068\u6b27\u5dde\u306e\u30b3\u30db\u30fc\u30c8\u7814\u7a76\u3067\u306f\u30012030\u5e74\u307e\u3067\u6709\u75c5\u7387\u304c\u7740\u5b9f\u306b\u4e0a\u6607\u3059\u308b\u3068\u4e88\u6e2c\u3055\u308c\u3066\u304a\u308a\u3001\u30cf\u30f3\u30c1\u30f3\u30c8\u30f3\u75c5\u6cbb\u7642\u5e02\u5834\u306b\u6301\u7d9a\u7684\u306a\u91cf\u7684\u52e2\u3044\u3092\u3082\u305f\u3089\u3057\u3066\u3044\u307e\u3059\u3002<br \/>\n*   HTT\uff08\u30cf\u30f3\u30c1\u30f3\u30c1\u30f3\uff09\u30bf\u30f3\u30d1\u30af\u8cea\u4f4e\u4e0b\u81e8\u5e8a\u30d1\u30a4\u30d7\u30e9\u30a4\u30f3\u306e\u753b\u671f\u7684\u306a\u9032\u5c55\u306e\u52a0\u901f\uff08CAGR\u3078\u306e\u5f71\u97ff: +2.8%\uff09: \u8907\u6570\u306e\u30d7\u30e9\u30c3\u30c8\u30d5\u30a9\u30fc\u30e0\u304c\u3001\u5909\u7570\u578b\u30cf\u30f3\u30c1\u30f3\u30c1\u30f3\u30bf\u30f3\u30d1\u30af\u8cea\u306e\u6709\u610f\u306a\u6e1b\u5c11\u3092\u793a\u3057\u3066\u3044\u307e\u3059\u3002WVE-003\u306f\u91ce\u751f\u578b\u30bf\u30f3\u30d1\u30af\u8cea\u3092\u6e29\u5b58\u3057\u3064\u3064\u8133\u810a\u9ac4\u6db2\u4e2d\u306e\u30bf\u30f3\u30d1\u30af\u8cea\u309246%\u6e1b\u5c11\u3055\u305b\u3001\u4ee5\u524d\u306e\u5b89\u5168\u6027\u61f8\u5ff5\u306b\u5bfe\u51e6\u3057\u307e\u3057\u305f\u3002\u7d4c\u53e3PTC 518\u306f\u8840\u6db2\u4e2d\u306e\u30bf\u30f3\u30d1\u30af\u8cea\u3092\u6700\u592743%\u6e1b\u5c11\u3055\u305b\u300112\u30f6\u6708\u3067\u7dcf\u904b\u52d5\u30b9\u30b3\u30a2\u306e\u9032\u884c\u309270%\u4ee5\u4e0a\u9045\u3089\u305b\u307e\u3057\u305f\u3002\u30ce\u30d0\u30eb\u30c6\u30a3\u30b9\u304cPTC 518\u309229\u5104\u7c73\u30c9\u30eb\u3067\u8cb7\u53ce\u3057\u305f\u3053\u3068\u3067\u6295\u8cc7\u5bb6\u306e\u4fe1\u983c\u304c\u6025\u4e0a\u6607\u3057\u3001\u3053\u306e\u6cbb\u7642\u6cd5\u306e\u5546\u696d\u7684\u53ef\u80fd\u6027\u304c\u5177\u4f53\u5316\u3057\u307e\u3057\u305f\u3002\u7c73\u56fdFDA\u304c\u30cf\u30f3\u30c1\u30f3\u30c1\u30f3\u30bf\u30f3\u30d1\u30af\u8cea\u4f4e\u4e0b\u3092\u4ee3\u66ff\u30a8\u30f3\u30c9\u30dd\u30a4\u30f3\u30c8\u3068\u3057\u3066\u53d7\u3051\u5165\u308c\u308b\u7528\u610f\u304c\u3042\u308b\u3053\u3068\u306f\u3001\u30bf\u30a4\u30e0\u30e9\u30a4\u30f3\u3092\u77ed\u7e2e\u3057\u3001\u5358\u4e00\u30d7\u30e9\u30c3\u30c8\u30d5\u30a9\u30fc\u30e0\u306e\u30ea\u30b9\u30af\u3092\u8efd\u6e1b\u3057\u3001\u30cf\u30f3\u30c1\u30f3\u30c8\u30f3\u75c5\u6cbb\u7642\u5e02\u5834\u306e\u8ecc\u9053\u3092\u52a0\u901f\u3055\u305b\u3066\u3044\u307e\u3059\u3002<br \/>\n*   \u30a2\u30b8\u30a2\u306b\u304a\u3051\u308b1\u65e51\u56de\u6295\u4e0eVMAT2\u963b\u5bb3\u5264\u306e\u5546\u696d\u7684\u767a\u58f2\uff08CAGR\u3078\u306e\u5f71\u97ff: +1.5%\uff09: Teva\u3068\u6c5f\u8607\u6069\u83ef\u85ac\u696d\u306e\u63d0\u643a\u306b\u3088\u308a\u3001\u4e2d\u56fd\u3067AUSTEDO\u304c\u521d\u306e\u91cd\u6c34\u7d20\u5316\u85ac\u3068\u3057\u3066\u627f\u8a8d\u3055\u308c\u3001\u821e\u8e0f\u75c5\u7ba1\u7406\u3078\u306e\u30a2\u30af\u30bb\u30b9\u304c\u62e1\u5927\u3057\u3001\u5c06\u6765\u306e\u75be\u60a3\u4fee\u98fe\u7642\u6cd5\u306e\u5c0e\u5165\u306e\u8db3\u304c\u304b\u308a\u3068\u306a\u308a\u307e\u3057\u305f\u3002INGREZZA\u306f2024\u5e74\u306b23\u5104\u7c73\u30c9\u30eb\u306e\u53ce\u76ca\u3092\u4e0a\u3052\u3001\u56a5\u4e0b\u969c\u5bb3\u306e\u3042\u308b\u60a3\u8005\u5411\u3051\u306b\u6563\u5264\u304c\u6295\u4e0e\u3092\u5bb9\u6613\u306b\u3057\u3066\u3044\u307e\u3059\u3002\u30a2\u30b8\u30a2\u306e\u6709\u75c5\u7387\uff0810\u4e07\u4eba\u3042\u305f\u308a0.40\u4eba\uff09\u3068\u6b27\u5dde\u306e\u6709\u75c5\u7387\uff0810\u4e07\u4eba\u3042\u305f\u308a5.70\u4eba\uff09\u306e\u683c\u5dee\u306f\u3001\u5927\u898f\u6a21\u306a\u5e02\u5834\u30ad\u30e3\u30f3\u30da\u30fc\u30f3\u3088\u308a\u3082\u533b\u5e2b\u6559\u80b2\u306b\u91cd\u70b9\u3092\u7f6e\u3044\u305f\u30e2\u30c7\u30eb\u3092\u5fc5\u8981\u3068\u3057\u307e\u3059\u3002\u3057\u305f\u304c\u3063\u3066\u3001\u6226\u7565\u7684\u306a\u30a2\u30b8\u30a2\u3067\u306e\u767a\u58f2\u306f\u3001\u30cf\u30f3\u30c1\u30f3\u30c8\u30f3\u75c5\u6cbb\u7642\u5e02\u5834\u306e\u30d5\u30c3\u30c8\u30d7\u30ea\u30f3\u30c8\u3092\u6b27\u7c73\u306e\u4e3b\u8981\u5730\u57df\u4ee5\u5916\u306b\u3082\u62e1\u5927\u3057\u3066\u3044\u307e\u3059\u3002<br \/>\n*   \u7c73\u56fd\/\u6b27\u5dde\u306b\u304a\u3051\u308b\u8fc5\u901f\u627f\u8a8d\u304a\u3088\u3073\u5e0c\u5c11\u75be\u75c5\u7528\u533b\u85ac\u54c1\u6307\u5b9a\uff08CAGR\u3078\u306e\u5f71\u97ff: +1.8%\uff09: AMT-130\u306f\u3001\u4ee5\u524d\u306eRMAT\u304a\u3088\u3073\u5e0c\u5c11\u75be\u75c5\u7528\u533b\u85ac\u54c1\u6307\u5b9a\u306b\u7d9a\u304d\u30012025\u5e74\u306b\u753b\u671f\u7684\u306a\u6cbb\u7642\u6cd5\u6307\u5b9a\u3092\u7372\u5f97\u3057\u3001\u524d\u4f8b\u306e\u306a\u3044\u898f\u5236\u30b9\u30a4\u30fc\u30c8\u3092\u78ba\u7acb\u3057\u307e\u3057\u305f\u3002PTC 518\u306f\u8fc5\u901f\u627f\u8a8d\u6307\u5b9a\u3092\u53d7\u3051\u3001\u30d7\u30ea\u30c9\u30d4\u30b8\u30f3\u306f\u6b27\u5dde\u3067\u306e\u8ca9\u58f2\u5be9\u67fb\u306b\u5165\u308a\u3001\u30d0\u30a4\u30aa\u30de\u30fc\u30ab\u30fc\u306b\u57fa\u3065\u304f\u7533\u8acb\u66f8\u985e\u3092\u53d7\u3051\u5165\u308c\u308b\u5f53\u5c40\u306e\u610f\u6b32\u3092\u53cd\u6620\u3057\u3066\u3044\u307e\u3059\u3002ENROLL-HD\u5916\u90e8\u5bfe\u7167\u30c7\u30fc\u30bf\u3092\u4f7f\u7528\u3059\u308b\u3053\u3068\u306b\u95a2\u3059\u308b\u5408\u610f\u306f\u3001\u5805\u7262\u306a\u5b9f\u4e16\u754c\u30a8\u30d3\u30c7\u30f3\u30b9\u30d5\u30ec\u30fc\u30e0\u30ef\u30fc\u30af\u3092\u5c55\u958b\u3059\u308b\u4f01\u696d\u306b\u6709\u5229\u306a\u898f\u5236\u30d1\u30e9\u30c0\u30a4\u30e0\u30b7\u30d5\u30c8\u3092\u793a\u3057\u3066\u3044\u307e\u3059\u3002<br \/>\n*   AI\u3092\u6d3b\u7528\u3057\u305f\u30d0\u30a4\u30aa\u30de\u30fc\u30ab\u30fc\u767a\u898b\u306b\u3088\u308b\u6cbb\u9a13\u6210\u529f\u7387\u306e\u5411\u4e0a\uff08CAGR\u3078\u306e\u5f71\u97ff: +0.9%\uff09: AI\u3092\u6d3b\u7528\u3057\u305f\u30d0\u30a4\u30aa\u30de\u30fc\u30ab\u30fc\u767a\u898b\u306f\u3001\u6cbb\u9a13\u30c7\u30b6\u30a4\u30f3\u3092\u6539\u5584\u3057\u3001\u6210\u529f\u7387\u3092\u9ad8\u3081\u308b\u3053\u3068\u3067\u3001\u5e02\u5834\u306e\u6210\u9577\u306b\u8ca2\u732e\u3057\u3066\u3044\u307e\u3059\u3002<br \/>\n*   \u30d9\u30fc\u30bf\u906e\u65ad\u85ac\u306e\u518d\u5229\u7528\u304c\u75be\u60a3\u4fee\u98fe\u30b7\u30b0\u30ca\u30eb\u3092\u793a\u3059\uff08CAGR\u3078\u306e\u5f71\u97ff: +0.6%\uff09: \u30d9\u30fc\u30bf\u906e\u65ad\u85ac\u306e\u518d\u5229\u7528\u306f\u3001\u767a\u75c7\u524d\u4fdd\u56e0\u8005\u306b\u304a\u3044\u3066\u904b\u52d5\u75c7\u72b6\u306e\u30ea\u30b9\u30af\u309234%\u6e1b\u5c11\u3055\u305b\u3001\u5bfe\u75c7\u7642\u6cd5\u3068\u75be\u60a3\u4fee\u98fe\u7642\u6cd5\u306e\u30d1\u30e9\u30c0\u30a4\u30e0\u3092\u7d50\u3073\u3064\u3051\u3066\u3044\u307e\u3059\u3002<\/p>\n<p>\u5e02\u5834\u306e\u6291\u5236\u8981\u56e0<\/p>\n<p>*   \u907a\u4f1d\u5b50\u30fb\u7d30\u80de\u7642\u6cd5\u306e\u8d85\u9ad8\u30b3\u30b9\u30c8\uff08CAGR\u3078\u306e\u5f71\u97ff: -1.8%\uff09: \u60a3\u8005\u4e00\u4eba\u3042\u305f\u308a100\u4e07\u7c73\u30c9\u30eb\u3092\u8d85\u3048\u308b\u4fa1\u683c\u306f\u3001\u516c\u5e73\u306a\u30a2\u30af\u30bb\u30b9\u3092\u8105\u304b\u3057\u3066\u3044\u307e\u3059\u30021\u4e07\u7a2e\u985e\u306e\u5e0c\u5c11\u75be\u60a3\u306e\u3046\u3061\u3001FDA\u627f\u8a8d\u306e\u6cbb\u7642\u6cd5\u304c\u3042\u308b\u306e\u306f5%\u672a\u6e80\u3067\u3042\u308a\u3001\u511f\u9084\u306e\u8ab2\u984c\u3092\u6d6e\u304d\u5f6b\u308a\u306b\u3057\u3066\u3044\u307e\u3059\u3002\u652f\u6255\u8005\u306f\u4fa1\u5024\u30d9\u30fc\u30b9\u306e\u30e2\u30c7\u30eb\u3084\u518d\u4fdd\u967a\u3092\u6a21\u7d22\u3057\u3066\u3044\u307e\u3059\u304c\u3001\u65ad\u7247\u7684\u306a\u63a1\u7528\u306b\u3088\u308a\u3001\u591a\u304f\u306e\u5e02\u5834\u304c\u624b\u306e\u5c4a\u304b\u306a\u3044\u307e\u307e\u3067\u3042\u308a\u3001\u30cf\u30f3\u30c1\u30f3\u30c8\u30f3\u75c5\u6cbb\u7642\u5e02\u5834\u306b\u304a\u3051\u308b\u65e9\u671f\u5c0e\u5165\u3092\u9045\u3089\u305b\u3066\u3044\u307e\u3059\u3002<br \/>\n*   \u5317\u7c73\u304a\u3088\u3073\u6b27\u5dde\u4ee5\u5916\u306e\u5c02\u9580\u533b\u7642\u30bb\u30f3\u30bf\u30fc\u306e\u9650\u5b9a\u6027\uff08CAGR\u3078\u306e\u5f71\u97ff: -1.2%\uff09: \u5317\u7c73\u306e\u9ed2\u4eba\u53c2\u52a0\u8005\u306f\u8a3a\u65ad\u306b12\u30f6\u6708\u8fd1\u304f\u306e\u9045\u5ef6\u3092\u7d4c\u9a13\u3057\u3066\u304a\u308a\u3001\u5148\u9032\u7684\u306a\u30b7\u30b9\u30c6\u30e0\u306b\u304a\u3044\u3066\u3082\u4f53\u7cfb\u7684\u306a\u4e0d\u516c\u5e73\u304c\u6d6e\u304d\u5f6b\u308a\u306b\u306a\u3063\u3066\u3044\u307e\u3059\u3002\u65b0\u8208\u5730\u57df\u3067\u306f\u9ac4\u8154\u5185\u6295\u4e0e\u3084\u907a\u4f1d\u5b50\u6cbb\u7642\u65bd\u8a2d\u304c\u4e0d\u8db3\u3057\u3066\u3044\u308b\u3053\u3068\u304c\u591a\u304f\u3001\u9060\u9694\u533b\u7642\u306f\u4ee3\u66ff\u624b\u6bb5\u3067\u306f\u306a\u304f\u4e00\u6642\u7684\u306a\u63aa\u7f6e\u306b\u3068\u3069\u307e\u3063\u3066\u3044\u307e\u3059\u3002\u3057\u305f\u304c\u3063\u3066\u3001\u30a4\u30f3\u30d5\u30e9\u6295\u8cc7\u3068\u81e8\u5e8a\u533b\u306e\u30c8\u30ec\u30fc\u30cb\u30f3\u30b0\u306f\u3001\u4e16\u754c\u5e02\u5834\u306e\u6210\u719f\u306e\u305f\u3081\u306e\u524d\u63d0\u6761\u4ef6\u3067\u3042\u308a\u7d9a\u3051\u3066\u3044\u307e\u3059\u3002<br \/>\n*   \u5bfe\u75c7\u7642\u6cd5\u306e\u591a\u5264\u4f75\u7528\u306b\u5bfe\u3059\u308b\u670d\u85ac\u9075\u5b88\u306e\u6700\u9069\u5316\u4e0d\u8db3\uff08CAGR\u3078\u306e\u5f71\u97ff: -0.7%\uff09: \u5bfe\u75c7\u7642\u6cd5\u306e\u591a\u5264\u4f75\u7528\u306b\u5bfe\u3059\u308b\u670d\u85ac\u9075\u5b88\u306e\u8ab2\u984c\u306f\u3001\u6cbb\u7642\u52b9\u679c\u3092\u5236\u9650\u3057\u3001\u5e02\u5834\u306e\u6210\u9577\u3092\u6291\u5236\u3059\u308b\u53ef\u80fd\u6027\u304c\u3042\u308a\u307e\u3059\u3002<br \/>\n*   \u795e\u7d4c\u7d44\u7e54\u306b\u304a\u3051\u308bCRISPR\u306e\u30c7\u30fc\u30bf\u5b89\u5168\u6027\u306b\u95a2\u3059\u308b\u61f8\u5ff5\uff08CAGR\u3078\u306e\u5f71\u97ff: -0.9%\uff09: CRISPR\u306a\u3069\u306e\u30b2\u30ce\u30e0\u7de8\u96c6\u6280\u8853\u306e\u795e\u7d4c\u7d44\u7e54\u3078\u306e\u5fdc\u7528\u306b\u304a\u3051\u308b\u5b89\u5168\u6027\u306b\u95a2\u3059\u308b\u61f8\u5ff5\u306f\u3001\u9577\u671f\u7684\u306a\u958b\u767a\u3068\u63a1\u7528\u306b\u5f71\u97ff\u3092\u4e0e\u3048\u308b\u53ef\u80fd\u6027\u304c\u3042\u308a\u307e\u3059\u3002<\/p>\n<p>\u30bb\u30b0\u30e1\u30f3\u30c8\u5206\u6790<\/p>\n<p>*   \u6cbb\u7642\u30bf\u30a4\u30d7\u5225: \u75be\u60a3\u4fee\u98fe\u7642\u6cd5\u304c\u30a4\u30ce\u30d9\u30fc\u30b7\u30e7\u30f3\u3092\u727d\u5f15<br \/>\n    2024\u5e74\u306b\u306f\u5bfe\u75c7\u7642\u6cd5\u304c\u30cf\u30f3\u30c1\u30f3\u30c8\u30f3\u75c5\u6cbb\u7642\u5e02\u5834\u30b7\u30a7\u30a2\u306e62.55%\u3092\u7dad\u6301\u3057\u307e\u3057\u305f\u304c\u3001\u75be\u60a3\u4fee\u98fe\u85ac\u306f2030\u5e74\u307e\u3067\u306b16.25%\u306eCAGR\u3067\u3088\u308a\u901f\u304f\u6210\u9577\u3057\u3066\u3044\u307e\u3059\u3002\u3053\u306e\u8ee2\u63db\u306f\u3001\u7d4c\u53e3\u5c0f\u5206\u5b50\u3001\u30a2\u30f3\u30c1\u30bb\u30f3\u30b9\u30aa\u30ea\u30b4\u30cc\u30af\u30ec\u30aa\u30c1\u30c9\u3001RNA\u5e72\u6e09\u69cb\u7bc9\u7269\u3001\u304a\u3088\u3073AAV\u907a\u4f1d\u5b50\u6cbb\u7642\u304c\u53ce\u675f\u3059\u308b\u306b\u3064\u308c\u3066\u3001\u30cf\u30f3\u30c1\u30f3\u30c8\u30f3\u75c5\u6cbb\u7642\u5e02\u5834\u3092\u62e1\u5927\u3055\u305b\u3066\u3044\u307e\u3059\u3002INGREZZA\u306e2024\u5e74\u306e23\u5104\u7c73\u30c9\u30eb\u306e\u53ce\u76ca\u306f\u3001\u821e\u8e0f\u75c5\u7ba1\u7406\u306b\u5bfe\u3059\u308b\u5805\u8abf\u306a\u9700\u8981\u3092\u88cf\u4ed8\u3051\u3066\u3044\u308b\u4e00\u65b9\u3001AMT-130\u304c\u5916\u90e8\u5bfe\u7167\u3068\u6bd4\u8f03\u3057\u3066\u75be\u60a3\u9032\u884c\u309280%\u9045\u3089\u305b\u305f\u3053\u3068\u306f\u3001\u907a\u4f1d\u5b50\u6cbb\u7642\u306e\u7834\u58ca\u7684\u306a\u53ef\u80fd\u6027\u3092\u793a\u3057\u3066\u3044\u307e\u3059\u3002\u30d9\u30fc\u30bf\u906e\u65ad\u85ac\u306e\u518d\u5229\u7528\u306f\u3001\u767a\u75c7\u524d\u4fdd\u56e0\u8005\u306b\u304a\u3044\u3066\u904b\u52d5\u75c7\u72b6\u306e\u30ea\u30b9\u30af\u309234%\u6e1b\u5c11\u3055\u305b\u3001\u5bfe\u75c7\u7642\u6cd5\u3068\u75be\u60a3\u4fee\u98fe\u7642\u6cd5\u306e\u30d1\u30e9\u30c0\u30a4\u30e0\u3092\u7d50\u3073\u3064\u3051\u307e\u3057\u305f\u3002\u4f75\u7528\u7642\u6cd5\u304c\u5177\u4f53\u5316\u3059\u308b\u306b\u3064\u308c\u3066\u3001\u958b\u767a\u8005\u306f\u30b7\u30fc\u30b1\u30f3\u30b9\u3001\u5b89\u5168\u6027\u30e2\u30cb\u30bf\u30ea\u30f3\u30b0\u3001\u304a\u3088\u3073\u652f\u6255\u8005\u3078\u306e\u30a8\u30d3\u30c7\u30f3\u30b9\u30d1\u30c3\u30b1\u30fc\u30b8\u3092\u6700\u9069\u5316\u3059\u308b\u5fc5\u8981\u304c\u3042\u308a\u307e\u3059\u3002<br \/>\n    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  \u6295\u4e0e\u7d4c\u8def\u5225: \u9759\u8108\u5185\u6295\u4e0e\u304c\u52e2\u3044\u3092\u5897\u3059<br \/>\n    2024\u5e74\u306b\u306f\u7d4c\u53e3\u88fd\u5264\u304c\u30cf\u30f3\u30c1\u30f3\u30c8\u30f3\u75c5\u6cbb\u7642\u5e02\u5834\u898f\u6a21\u306e54.53%\u3092\u5360\u3081\u3001\u9ad8\u3044\u670d\u85ac\u9075\u5b88\u3068\u30d7\u30e9\u30a4\u30de\u30ea\u30b1\u30a2\u3067\u306e\u51e6\u65b9\u30d1\u30bf\u30fc\u30f3\u3068\u4e00\u81f4\u3057\u3066\u3044\u307e\u3059\u3002\u9759\u8108\u5185\u30bb\u30b0\u30e1\u30f3\u30c8\u306f11.85%\u306eCAGR\u3067\u6210\u9577\u3057\u3066\u304a\u308a\u30011\u56de\u9650\u308a\u306e\u907a\u4f1d\u5b50\u6cbb\u7642\u3084\u3001\u6b63\u78ba\u306a\u4e2d\u67a2\u795e\u7d4c\u7cfb\u5206\u5e03\u3092\u5fc5\u8981\u3068\u3059\u308b\u53cd\u5fa9\u7684\u306a\u30a2\u30f3\u30c1\u30bb\u30f3\u30b9\u6ce8\u5165\u306b\u3088\u3063\u3066\u4fc3\u9032\u3055\u308c\u3066\u3044\u307e\u3059\u3002\u9ac4\u8154\u5185\u6295\u4e0e\u306f\u30cb\u30c3\u30c1\u3067\u3059\u304c\u3001\u5168\u8eab\u66dd\u9732\u306a\u3057\u306b\u8133\u810a\u9ac4\u6db2\u3078\u306e\u30a2\u30af\u30bb\u30b9\u3092\u5fc5\u8981\u3068\u3059\u308b\u30a2\u30ec\u30eb\u9078\u629e\u7684\u30aa\u30ea\u30b4\u30cc\u30af\u30ec\u30aa\u30c1\u30c9\u306b\u3068\u3063\u3066\u4e0d\u53ef\u6b20\u3067\u3059\u3002<br \/>\n    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  \u6d41\u901a\u30c1\u30e3\u30cd\u30eb\u5225: \u5c02\u9580\u85ac\u5c40\u304c\u6210\u9577\u3092\u6349\u3048\u308b<br \/>\n    2024\u5e74\u306b\u306f\u75c5\u9662\u85ac\u5c40\u304c\u53ce\u76ca\u306e55.12%\u3092\u5360\u3081\u3001\u907a\u4f1d\u5b50\u6cbb\u7642\u306e\u958b\u59cb\u3068\u6025\u6027\u795e\u7d4c\u7cbe\u795e\u30a4\u30d9\u30f3\u30c8\u306e\u7ba1\u7406\u3092\u53cd\u6620\u3057\u3066\u3044\u307e\u3059\u3002\u5c02\u9580\u85ac\u5c40\u304a\u3088\u3073\u30aa\u30f3\u30e9\u30a4\u30f3\u85ac\u5c40\u306f\u3001\u60a3\u8005\u3078\u306e\u76f4\u63a5\u7269\u6d41\u3001\u670d\u85ac\u9075\u5b88\u5206\u6790\u3001\u304a\u3088\u3073\u6210\u679c\u30d9\u30fc\u30b9\u306e\u511f\u9084\u7ba1\u7406\u306b\u652f\u3048\u3089\u308c\u3001\u6700\u3082\u5f37\u304415.31%\u306eCAGR\u3092\u9054\u6210\u3059\u308b\u3068\u4e88\u6e2c\u3055\u308c\u3066\u3044\u307e\u3059\u3002\u8abf\u5264\u85ac\u5c40\u306f\u7d4c\u53e3\u5bfe\u75c7\u7642\u6cd5\u85ac\u306e\u51e6\u65b9\u3092\u4f9b\u7d66\u3057\u7d9a\u3051\u3066\u3044\u307e\u3059\u304c\u3001\u670d\u85ac\u4e0d\u9075\u5b88\u3084\u526f\u4f5c\u7528\u30d1\u30bf\u30fc\u30f3\u3092\u7279\u5b9a\u3059\u308b\u305f\u3081\u306e\u30c7\u30b8\u30bf\u30eb\u30c4\u30fc\u30eb\u3092\u7d71\u5408\u3057\u3066\u3044\u307e\u3059\u3002<br \/>\n    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  \u75c5\u671f\u5225: \u767a\u75c7\u524d\u30bb\u30b0\u30e1\u30f3\u30c8\u304c\u30a4\u30ce\u30d9\u30fc\u30b7\u30e7\u30f3\u3092\u727d\u5f15<br \/>\n    2024\u5e74\u306e\u53ce\u76ca\u306e40.63%\u306f\u65e9\u671f\u767a\u75c7\u304c\u5360\u3081\u307e\u3057\u305f\u304c\u3001\u30d0\u30a4\u30aa\u30de\u30fc\u30ab\u30fc\u99c6\u52d5\u578b\u30b9\u30af\u30ea\u30fc\u30cb\u30f3\u30b0\u304c\u767a\u75c7\u524d\u30ad\u30e3\u30ea\u30a2\u3092\u65e9\u671f\u306b\u7279\u5b9a\u3059\u308b\u305f\u3081\u3001\u767a\u75c7\u524d\u30bb\u30b0\u30e1\u30f3\u30c8\u306f2030\u5e74\u307e\u3067\u306b16.7%\u306eCAGR\u3067\u6210\u9577\u3059\u308b\u8ecc\u9053\u306b\u4e57\u3063\u3066\u3044\u307e\u3059\u3002\u4e88\u9632\u7684\u4ecb\u5165\u306e\u305f\u3081\u306e\u30cf\u30f3\u30c1\u30f3\u30c8\u30f3\u75c5\u6cbb\u7642\u5e02\u5834\u898f\u6a21\u306f\u30012030\u5e74\u307e\u3067\u306b2.5\u5104\u7c73\u30c9\u30eb\u3092\u8d85\u3048\u308b\u3068\u4e88\u60f3\u3055\u308c\u3066\u3044\u307e\u3059\u3002\u4f53\u7d30\u80deCAG\u30ea\u30d4\u30fc\u30c8\u30a2\u30c3\u30bb\u30a4\u3001\u795e\u7d4c\u30d5\u30a3\u30e9\u30e1\u30f3\u30c8\u8efd\u9396\u306e\u4e0a\u6607\u3001\u304a\u3088\u3073\u30c7\u30b8\u30bf\u30eb\u904b\u52d5\u30bb\u30f3\u30b5\u30fc\u306e\u8aad\u307f\u53d6\u308a\u306f\u3001\u6cbb\u9a13\u767b\u9332\u3092\u5145\u5b9f\u3055\u305b\u3001\u3088\u308a\u65e9\u671f\u306e\u6cbb\u7642\u30a6\u30a3\u30f3\u30c9\u30a6\u3092\u691c\u8a3c\u3057\u307e\u3059\u3002<br \/>\n    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  \u5317\u7c73: 2024\u5e74\u306b\u306f\u4e16\u754c\u306e\u53ce\u76ca\u306e38.63%\u3092\u5360\u3081\u3001\u8fc5\u901f\u627f\u8a8d\u7d4c\u8def\u306b\u95a2\u3059\u308bFDA\u306e\u30ea\u30fc\u30c0\u30fc\u30b7\u30c3\u30d7\u3001\u96c6\u4e2d\u3057\u305f\u5c02\u9580\u30af\u30ea\u30cb\u30c3\u30af\u3001\u304a\u3088\u3073VMAT2\u963b\u5bb3\u5264\u306e\u5f37\u529b\u306a\u652f\u6255\u8005\u63a1\u7528\u306b\u652f\u3048\u3089\u308c\u3066\u3044\u307e\u3059\u3002\u3057\u304b\u3057\u3001\u9ed2\u4eba\u4eba\u53e3\u306b\u304a\u3051\u308b\u8a3a\u65ad\u306e\u9045\u5ef6\u306f\u3001\u7d76\u5bfe\u7684\u306a\u6210\u9577\u306e\u53ef\u80fd\u6027\u3092\u6291\u5236\u3059\u308b\u4f53\u7cfb\u7684\u306a\u4e0d\u516c\u5e73\u3092\u6d6e\u304d\u5f6b\u308a\u306b\u3057\u3066\u3044\u307e\u3059\u3002\u30ab\u30ca\u30c0\u3068\u30e1\u30ad\u30b7\u30b3\u306f\u7c73\u56fd\u306e\u30bb\u30f3\u30bf\u30fc\u3068\u5354\u529b\u3057\u3066\u907a\u4f1d\u5b50\u6cbb\u7642\u6cbb\u9a13\u3078\u306e\u30a2\u30af\u30bb\u30b9\u3092\u62e1\u5927\u3057\u3001\u56fd\u5883\u3092\u8d8a\u3048\u305f\u7d39\u4ecb\u30c1\u30e3\u30cd\u30eb\u3092\u80b2\u6210\u3057\u3066\u30cf\u30f3\u30c1\u30f3\u30c8\u30f3\u75c5\u6cbb\u7642\u5e02\u5834\u3092\u7740\u5b9f\u306b\u62e1\u5927\u3057\u3066\u3044\u307e\u3059\u3002<br \/>\n*   \u6b27\u5dde: \u898f\u5236\u306e\u8abf\u548c\u3092\u6d3b\u7528\u3057\u3066\u75be\u60a3\u4fee\u98fe\u85ac\u306e\u7533\u8acb\u3092\u8fc5\u901f\u5316\u3057\u3066\u304a\u308a\u3001EMA\u306f2025\u5e74\u4e0b\u534a\u671f\u306e\u767a\u58f2\u306b\u5411\u3051\u3066\u30d7\u30ea\u30c9\u30d4\u30b8\u30f3\u3092\u5be9\u67fb\u3057\u3066\u3044\u307e\u3059\u3002\u591a\u56fd\u7c4d\u30b3\u30f3\u30bd\u30fc\u30b7\u30a2\u30e0\u306f\u30cf\u30f3\u30c1\u30f3\u30c8\u30f3\u30b5\u30dd\u30fc\u30c8\u30a2\u30d7\u30ea\u3092\u958b\u767a\u3057\u3001\u4e0d\u5747\u4e00\u306a\u30af\u30ea\u30cb\u30c3\u30af\u5bc6\u5ea6\u3092\u76f8\u6bba\u3059\u308b\u8cbb\u7528\u5bfe\u52b9\u679c\u306e\u9ad8\u3044\u30c7\u30b8\u30bf\u30eb\u30a2\u30a6\u30c8\u30ea\u30fc\u30c1\u306e\u4f8b\u3092\u793a\u3057\u3066\u3044\u307e\u3059\u3002\u30c9\u30a4\u30c4\u3001\u82f1\u56fd\u3001\u30d5\u30e9\u30f3\u30b9\u304c\u6cbb\u9a13\u30b9\u30dd\u30f3\u30b5\u30fc\u30b7\u30c3\u30d7\u3092\u30ea\u30fc\u30c9\u3057\u3001\u6771\u6b27\u306e\u8fd1\u4ee3\u5316\u3055\u308c\u305f\u795e\u7d4c\u5b66\u30a4\u30f3\u30d5\u30e9\u306f2\u6841\u306e\u6210\u9577\u3092\u7d04\u675f\u3057\u3066\u3044\u307e\u3059\u3002\u591a\u69d8\u306a\u511f\u9084\u30b7\u30b9\u30c6\u30e0\u306f\u3001\u691c\u8a3c\u3055\u308c\u308c\u3070\u4ed6\u306e\u5730\u57df\u306b\u79fb\u884c\u3059\u308b\u53ef\u80fd\u6027\u306e\u3042\u308b\u30d1\u30a4\u30ed\u30c3\u30c8\u6210\u679c\u30d9\u30fc\u30b9\u5951\u7d04\u3092\u4fc3\u9032\u3057\u3066\u3044\u307e\u3059\u3002<br \/>\n*   \u30a2\u30b8\u30a2\u592a\u5e73\u6d0b: 2030\u5e74\u307e\u3067\u306b14.87%\u306eCAGR\u3067\u6700\u3082\u6025\u901f\u306b\u6210\u9577\u3057\u3066\u3044\u308b\u5730\u57df\u3067\u3042\u308a\u3001\u4e2d\u56fd\u521d\u306e\u91cd\u6c34\u7d20\u5316\u85ac\u306e\u627f\u8a8d\u3068\u65e5\u672c\u306e\u6d17\u7df4\u3055\u308c\u305f\u5e0c\u5c11\u75be\u60a3\u767b\u9332\u304c\u727d\u5f15\u3057\u3066\u3044\u307e\u3059\u3002\u97d3\u56fd\u306e\u5c02\u9580\u5b66\u4f1a\u306f\u5730\u5143\u306e\u81e8\u5e8a\u533b\u306e\u30c8\u30ec\u30fc\u30cb\u30f3\u30b0\u3092\u9032\u3081\u3001\u30aa\u30fc\u30b9\u30c8\u30e9\u30ea\u30a2\u306e\u898f\u5236\u4e26\u884c\u51e6\u7406\u306f\u56fd\u969b\u7814\u7a76\u3092\u52a0\u901f\u3055\u305b\u3066\u3044\u307e\u3059\u3002\u6709\u75c5\u7387\u304c\u4f4e\u3044\u3053\u3068\u304b\u3089\u3001\u533b\u5e2b\u4e3b\u5c0e\u306e\u60a3\u8005\u30de\u30c3\u30d4\u30f3\u30b0\u3001\u5c02\u9580\u30bb\u30f3\u30bf\u30fc\u306e\u8a8d\u5b9a\u3001\u304a\u3088\u3073\u9060\u9694\u30e2\u30cb\u30bf\u30ea\u30f3\u30b0\u3068\u3044\u3063\u305f\u304d\u3081\u7d30\u3084\u304b\u306a\u95a2\u4e0e\u304c\u5fc5\u8981\u3068\u3055\u308c\u3001\u30a4\u30f3\u30d5\u30e9\u304c\u9045\u308c\u3066\u3044\u308b\u5730\u57df\u306e\u30ae\u30e3\u30c3\u30d7\u3092\u57cb\u3081\u3066\u3044\u307e\u3059\u3002\u3053\u308c\u3089\u306e\u53d6\u308a\u7d44\u307f\u306f\u3001\u65b0\u8208\u7d4c\u6e08\u56fd\u5168\u4f53\u3067\u30cf\u30f3\u30c1\u30f3\u30c8\u30f3\u75c5\u6cbb\u7642\u5e02\u5834\u306e\u5b58\u5728\u611f\u3092\u96c6\u5408\u7684\u306b\u9ad8\u3081\u3066\u3044\u307e\u3059\u3002<br \/>\n*   \u30e9\u30c6\u30f3\u30a2\u30e1\u30ea\u30ab\u3001\u4e2d\u6771\u3001\u30a2\u30d5\u30ea\u30ab: 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518\u304c\u30ce\u30d0\u30eb\u30c6\u30a3\u30b9\u306b29\u5104\u7c73\u30c9\u30eb\u3067\u8b72\u6e21\u3055\u308c\u305f\u3053\u3068\u306f\u3001\u5927\u624b\u4f01\u696d\u304c\u7b2c\u4e00\u539f\u7406\u7814\u7a76\u3088\u308a\u3082\u30ea\u30b9\u30af\u304c\u8efd\u6e1b\u3055\u308c\u305f\u7b2c2\u76f8\u8cc7\u7523\u3092\u6c42\u3081\u308b\u610f\u6b32\u3092\u793a\u3057\u3066\u3044\u307e\u3059\u3002<br \/>\n\u30a2\u30ec\u30eb\u9078\u629e\u7684\u4f4e\u4e0b\u3001\u5c0f\u5206\u5b50\u306e\u6d78\u900f\u6027\u3001\u304a\u3088\u3073\u8010\u4e45\u6027\u306e\u3042\u308bAAV\u30da\u30a4\u30ed\u30fc\u30c9\u304c\u4e3b\u8981\u306a\u6280\u8853\u7af6\u4e89\u3092\u5f62\u6210\u3057\u3066\u3044\u307e\u3059\u3002\u30c7\u30ea\u30d0\u30ea\u30fc\u306f\u30dd\u30fc\u30c8\u30d5\u30a9\u30ea\u30aa\u3092\u5dee\u5225\u5316\u3057\u307e\u3059\u3002\u9ac4\u8154\u5185\u30aa\u30ea\u30b4\u30cc\u30af\u30ec\u30aa\u30c1\u30c9\u306f\u30011\u56de\u9650\u308a\u306e\u9759\u8108\u5185\u30d9\u30af\u30bf\u30fc\u3084\u6bce\u65e5\u306e\u7d4c\u53e3\u30e2\u30b8\u30e5\u30ec\u30fc\u30bf\u30fc\u3068\u306f\u5bfe\u7167\u7684\u3067\u3059\u3002\u7af6\u4e89\u512a\u4f4d\u6027\u306f\u3001\u30d0\u30a4\u30aa\u30de\u30fc\u30ab\u30fc\u306e\u6d17\u7df4\u5ea6\u3001\u5b9f\u4e16\u754c\u306e\u30a8\u30d3\u30c7\u30f3\u30b9\u6226\u7565\u3001\u304a\u3088\u3073\u511f\u9084\u4ea4\u6e09\u306e\u57fa\u790e\u3068\u306a\u308b\u60a3\u8005\u4e2d\u5fc3\u306e\u30b5\u30fc\u30d3\u30b9\u30e2\u30c7\u30eb\u304b\u3089\u307e\u3059\u307e\u3059\u751f\u307e\u308c\u3066\u3044\u307e\u3059\u3002<br \/>\n\u672a\u958b\u62d3\u306e\u6a5f\u4f1a\u306b\u306f\u3001\u75bc\u75db\u7ba1\u7406\u306e\u4f75\u7528\u7642\u6cd5\u3001\u30d9\u30fc\u30bf\u906e\u65ad\u85ac\u306e\u518d\u5229\u7528\u3001\u304a\u3088\u3073\u30c7\u30b8\u30bf\u30eb\u6cbb\u7642\u88dc\u52a9\u5264\u304c\u542b\u307e\u308c\u307e\u3059\u3002Biogen\u306e\u65b0\u3057\u3044\u30b9\u30d7\u30e9\u30a4\u30b7\u30f3\u30b0\u30e2\u30b8\u30e5\u30ec\u30fc\u30bf\u30fc\u7279\u8a31\u306f\u3001\u6b21\u4e16\u4ee3\u30bf\u30fc\u30b2\u30c6\u30a3\u30f3\u30b0\u306e\u7d99\u7d9a\u7684\u306a\u8ffd\u6c42\u3092\u793a\u5506\u3057\u3066\u3044\u307e\u3059\u3002\u30d1\u30a4\u30d7\u30e9\u30a4\u30f3\u306e\u6df7\u96d1\u304c\u30a8\u30b9\u30ab\u30ec\u30fc\u30c8\u3057\u3066\u3044\u308b\u306b\u3082\u304b\u304b\u308f\u3089\u305a\u3001\u30cf\u30f3\u30c1\u30f3\u30c8\u30f3\u75c5\u6cbb\u7642\u5e02\u5834\u306f\u3001\u753b\u671f\u7684\u306a\u751f\u7269\u5b66\u3068\u5b9f\u7528\u7684\u306a\u6d41\u901a\u304a\u3088\u3073\u30a8\u30d3\u30c7\u30f3\u30b9\u30d5\u30ec\u30fc\u30e0\u30ef\u30fc\u30af\u3092\u7d44\u307f\u5408\u308f\u305b\u305f\u4f01\u696d\u306b\u5831\u3044\u3066\u3044\u307e\u3059\u3002<\/p>\n<p>\u30cf\u30f3\u30c1\u30f3\u30c8\u30f3\u75c5\u6cbb\u7642\u696d\u754c\u306e\u4e3b\u8981\u4f01\u696d<\/p>\n<p>*   \u30d5\u30a1\u30a4\u30b6\u30fc\u682a\u5f0f\u4f1a\u793e<br \/>\n*   \u30a2\u30eb\u30ca\u30a4\u30e9\u30e0\u30fb\u30d5\u30a1\u30fc\u30de\u30b7\u30e5\u30fc\u30c6\u30a3\u30ab\u30eb\u30ba\u30fb\u30a4\u30f3\u30af<br \/>\n*   \u30c6\u30d0\u30fb\u30d5\u30a1\u30fc\u30de\u30b7\u30e5\u30fc\u30c6\u30a3\u30ab\u30eb\u30fb\u30a4\u30f3\u30c0\u30b9\u30c8\u30ea\u30fc\u30ba\u30fb\u30ea\u30df\u30c6\u30c3\u30c9<br \/>\n*   \u30cb\u30e5\u30fc\u30ed\u30af\u30e9\u30a4\u30f3\u30fb\u30d0\u30a4\u30aa\u30b5\u30a4\u30a8\u30f3\u30b7\u30ba\u30fb\u30a4\u30f3\u30af<br \/>\n*   \u30a2\u30a4\u30aa\u30cb\u30b9\u30fb\u30d5\u30a1\u30fc\u30de\u30b7\u30e5\u30fc\u30c6\u30a3\u30ab\u30eb\u30ba<\/p>\n<p>\u6700\u8fd1\u306e\u696d\u754c\u52d5\u5411<\/p>\n<p>*   2025\u5e745\u6708: PTC\u30bb\u30e9\u30d4\u30e5\u30fc\u30c6\u30a3\u30af\u30b9\u306f\u3001PIVOT-HD\u7b2c2\u76f8\u8a66\u9a13\u306e\u6210\u529f\u3092\u5831\u544a\u3057\u3001\u6709\u610f\u306a\u30cf\u30f3\u30c1\u30f3\u30c1\u30f3\u30bf\u30f3\u30d1\u30af\u8cea\u6e1b\u5c11\u3068\u826f\u597d\u306a\u5b89\u5168\u6027\u30d7\u30ed\u30d5\u30a1\u30a4\u30eb\u3092\u5b9f\u73fe\u3057\u307e\u3057\u305f\u3002<br \/>\n*   2025\u5e744\u6708: uniQure\u306f\u3001\u5916\u90e8\u5bfe\u7167\u3068\u6bd4\u8f03\u3057\u3066\u75be\u60a3\u9032\u884c\u309280%\u9045\u3089\u305b\u305f\u5f8c\u3001AMT-130\u306b\u5bfe\u3057\u3066FDA\u306e\u753b\u671f\u7684\u306a\u6cbb\u7642\u6cd5\u6307\u5b9a\u3092\u53d7\u3051\u307e\u3057\u305f\u3002<\/p>\n<p>\u3053\u306e\u30ec\u30dd\u30fc\u30c8\u306f\u3001\u30cf\u30f3\u30c1\u30f3\u30c8\u30f3\u75c5\u6cbb\u7642\u5e02\u5834\u306b\u95a2\u3059\u308b\u5305\u62ec\u7684\u306a\u5206\u6790\u3092\u63d0\u4f9b\u3057\u3066\u3044\u307e\u3059\u3002\u30cf\u30f3\u30c1\u30f3\u30c8\u30f3\u75c5\u306f\u3001\u4e3b\u306b30\u6b73\u304b\u308950\u6b73\u306e\u9593\u306b\u767a\u75c7\u3057\u3001\u8133\u306e\u795e\u7d4c\u7d30\u80de\u306e\u5909\u6027\u3092\u5f15\u304d\u8d77\u3053\u3059\u907a\u4f1d\u6027\u75be\u60a3\u3067\u3042\u308a\u3001\u672c\u30ec\u30dd\u30fc\u30c8\u3067\u306f\u5546\u696d\u7684\u306b\u5229\u7528\u53ef\u80fd\u306a\u69d8\u3005\u306a\u6cbb\u7642\u6cd5\u3092\u5bfe\u8c61\u3068\u3057\u3066\u3044\u307e\u3059\u3002<\/p>\n<p>\u5e02\u5834\u306e\u6982\u8981\u3068\u4e88\u6e2c<br \/>\n\u30cf\u30f3\u30c1\u30f3\u30c8\u30f3\u75c5\u6cbb\u7642\u5e02\u5834\u306f\u30012025\u5e74\u306b\u306f0.82\u5104\u7c73\u30c9\u30eb\u306e\u4fa1\u5024\u304c\u3042\u308b\u3068\u8a55\u4fa1\u3055\u308c\u3066\u304a\u308a\u30012030\u5e74\u307e\u3067\u306b\u306f1.22\u5104\u7c73\u30c9\u30eb\u306b\u9054\u3059\u308b\u3068\u4e88\u6e2c\u3055\u308c\u3066\u3044\u307e\u3059\u3002\u7279\u306b\u3001\u75be\u60a3\u4fee\u98fe\u7642\u6cd5\u306f2030\u5e74\u307e\u3067\u306b\u5e74\u5e73\u5747\u6210\u9577\u7387\uff08CAGR\uff0916.25%\u3067\u6700\u3082\u901f\u3044\u6210\u9577\u3092\u9042\u3052\u308b\u3068\u898b\u8fbc\u307e\u308c\u3066\u304a\u308a\u3001\u5bfe\u75c7\u7642\u6cd5\u3092\u4e0a\u56de\u308b\u52e2\u3044\u3067\u3059\u3002\u6295\u4e0e\u7d4c\u8def\u5225\u3067\u306f\u3001\u907a\u4f1d\u5b50\u6cbb\u7642\u3084\u30a2\u30f3\u30c1\u30bb\u30f3\u30b9\u30aa\u30ea\u30b4\u30cc\u30af\u30ec\u30aa\u30c1\u30c9\uff08ASO\uff09\u304c\u5236\u5fa1\u3055\u308c\u305f\u5168\u8eab\u307e\u305f\u306f\u8133\u810a\u9ac4\u3078\u306e\u9001\u9054\u3092\u5fc5\u8981\u3068\u3059\u308b\u3053\u3068\u304b\u3089\u3001\u9759\u8108\u5185\u6295\u4e0e\u30bb\u30b0\u30e1\u30f3\u30c8\u304c11.85%\u306eCAGR\u3067\u6210\u9577\u3092\u727d\u5f15\u3057\u3066\u3044\u307e\u3059\u3002\u5730\u57df\u5225\u3067\u306f\u3001\u30a2\u30b8\u30a2\u592a\u5e73\u6d0b\u5730\u57df\u304c\u6226\u7565\u7684\u30d1\u30fc\u30c8\u30ca\u30fc\u30b7\u30c3\u30d7\u3068\u898f\u5236\u306e\u8abf\u548c\u306b\u5f8c\u62bc\u3057\u3055\u308c\u30012030\u5e74\u307e\u3067\u306b14.87%\u306eCAGR\u3067\u6700\u3082\u9ad8\u3044\u6210\u9577\u6f5c\u5728\u529b\u3092\u793a\u3057\u3066\u3044\u307e\u3059\u3002<\/p>\n<p>\u5e02\u5834\u306e\u4fc3\u9032\u8981\u56e0<br \/>\n\u5e02\u5834\u306e\u6210\u9577\u3092\u4fc3\u9032\u3059\u308b\u4e3b\u306a\u8981\u56e0\u306f\u4ee5\u4e0b\u306e\u901a\u308a\u3067\u3059\u3002<br \/>\n*   \u9ad8\u9f62\u8005\u5c64\u306b\u304a\u3051\u308b\u767a\u751f\u7387\u306e\u5897\u52a0\uff1a \u9ad8\u9f62\u8005\u4eba\u53e3\u306e\u5897\u52a0\u306b\u4f34\u3044\u3001\u30cf\u30f3\u30c1\u30f3\u30c8\u30f3\u75c5\u306e\u767a\u751f\u7387\u3082\u4e0a\u6607\u3057\u3066\u304a\u308a\u3001\u6cbb\u7642\u85ac\u306e\u9700\u8981\u304c\u9ad8\u307e\u3063\u3066\u3044\u307e\u3059\u3002<br \/>\n*   HTT\u4f4e\u4e0b\u81e8\u5e8a\u30d1\u30a4\u30d7\u30e9\u30a4\u30f3\u306e\u753b\u671f\u7684\u306a\u9032\u5c55\uff1a \u30cf\u30f3\u30c1\u30f3\u30c8\u30f3\u75c5\u306e\u539f\u56e0\u907a\u4f1d\u5b50\u3067\u3042\u308b\u30cf\u30f3\u30c1\u30f3\u30bf\u30f3\u30bf\u30f3\u30d1\u30af\u8cea\uff08HTT\uff09\u306e\u30ec\u30d9\u30eb\u3092\u4f4e\u4e0b\u3055\u305b\u308b\u3053\u3068\u3092\u76ee\u7684\u3068\u3057\u305f\u81e8\u5e8a\u30d1\u30a4\u30d7\u30e9\u30a4\u30f3\u306b\u304a\u3044\u3066\u3001\u753b\u671f\u7684\u306a\u9032\u5c55\u304c\u898b\u3089\u308c\u3066\u3044\u307e\u3059\u3002<br \/>\n*   \u30a2\u30b8\u30a2\u3067\u306e1\u65e51\u56deVMAT2\u963b\u5bb3\u5264\u306e\u5546\u696d\u7684\u767a\u58f2\uff1a \u30a2\u30b8\u30a2\u5730\u57df\u306b\u304a\u3051\u308b1\u65e51\u56de\u6295\u4e0e\u306eVMAT2\u963b\u5bb3\u5264\u306e\u5546\u696d\u7684\u767a\u58f2\u304c\u3001\u5e02\u5834\u306e\u62e1\u5927\u306b\u8ca2\u732e\u3057\u3066\u3044\u307e\u3059\u3002<br \/>\n*   \u7c73\u56fd\/EU\u3067\u306e\u30d5\u30a1\u30b9\u30c8\u30c8\u30e9\u30c3\u30af\u304a\u3088\u3073\u30aa\u30fc\u30d5\u30a1\u30f3\u6307\u5b9a\uff1a \u7c73\u56fd\u304a\u3088\u3073\u6b27\u5dde\u9023\u5408\uff08EU\uff09\u306b\u304a\u3051\u308b\u30d5\u30a1\u30b9\u30c8\u30c8\u30e9\u30c3\u30af\u304a\u3088\u3073\u30aa\u30fc\u30d5\u30a1\u30f3\u6307\u5b9a\u304c\u3001\u65b0\u898f\u6cbb\u7642\u6cd5\u306e\u958b\u767a\u3068\u627f\u8a8d\u3092\u52a0\u901f\u3055\u305b\u3066\u3044\u307e\u3059\u3002<br \/>\n*   AI\u3092\u6d3b\u7528\u3057\u305f\u30d0\u30a4\u30aa\u30de\u30fc\u30ab\u30fc\u767a\u898b\u306b\u3088\u308b\u6cbb\u9a13\u6210\u529f\u7387\u306e\u5411\u4e0a\uff1a \u4eba\u5de5\u77e5\u80fd\uff08AI\uff09\u3092\u6d3b\u7528\u3057\u305f\u30d0\u30a4\u30aa\u30de\u30fc\u30ab\u30fc\u306e\u767a\u898b\u304c\u3001\u81e8\u5e8a\u8a66\u9a13\u306e\u6210\u529f\u7387\u3092\u9ad8\u3081\u3001\u65b0\u85ac\u958b\u767a\u3092\u4fc3\u9032\u3057\u3066\u3044\u307e\u3059\u3002<br \/>\n*   \u75be\u60a3\u4fee\u98fe\u30b7\u30b0\u30ca\u30eb\u3092\u793a\u3059\u30d9\u30fc\u30bf\u30d6\u30ed\u30c3\u30ab\u30fc\u306e\u518d\u5229\u7528\uff1a \u65e2\u5b58\u85ac\u3067\u3042\u308b\u30d9\u30fc\u30bf\u30d6\u30ed\u30c3\u30ab\u30fc\u304c\u75be\u60a3\u4fee\u98fe\u30b7\u30b0\u30ca\u30eb\u3092\u793a\u3059\u53ef\u80fd\u6027\u304c\u793a\u3055\u308c\u3066\u304a\u308a\u3001\u65b0\u305f\u306a\u6cbb\u7642\u9078\u629e\u80a2\u3068\u3057\u3066\u6ce8\u76ee\u3055\u308c\u3066\u3044\u307e\u3059\u3002<\/p>\n<p>\u5e02\u5834\u306e\u6291\u5236\u8981\u56e0<br \/>\n\u4e00\u65b9\u3067\u3001\u5e02\u5834\u306e\u6210\u9577\u3092\u59a8\u3052\u308b\u53ef\u80fd\u6027\u306e\u3042\u308b\u8981\u56e0\u3082\u5b58\u5728\u3057\u307e\u3059\u3002<br \/>\n*   \u907a\u4f1d\u5b50\u30fb\u7d30\u80de\u7642\u6cd5\u306e\u8d85\u9ad8\u30b3\u30b9\u30c8\uff1a 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\u30cf\u30f3\u30c1\u30f3\u30c8\u30f3\u75c5\u306e\u8a3a\u65ad\u3068\u6cbb\u7642\u306b\u306f\u5c02\u9580\u7684\u306a\u77e5\u8b58\u3068\u8a2d\u5099\u304c\u5fc5\u8981\u3067\u3059\u304c\u3001\u5317\u7c73\u304a\u3088\u3073EU\u4ee5\u5916\u306e\u5730\u57df\u3067\u306f\u5c02\u9580\u30bb\u30f3\u30bf\u30fc\u304c\u9650\u3089\u308c\u3066\u3044\u307e\u3059\u3002<br \/>\n*   \u5bfe\u75c7\u7642\u6cd5\u3068\u3057\u3066\u306e\u591a\u5264\u4f75\u7528\u7642\u6cd5\u306e\u9075\u5b88\u7387\u306e\u4f4e\u3055\uff1a \u5bfe\u75c7\u7642\u6cd5\u3068\u3057\u3066\u8907\u6570\u306e\u85ac\u5264\u3092\u4f75\u7528\u3059\u308b\u5834\u5408\u3001\u60a3\u8005\u306e\u670d\u85ac\u9075\u5b88\u7387\u304c\u4f4e\u3044\u3053\u3068\u304c\u8ab2\u984c\u3068\u306a\u3063\u3066\u3044\u307e\u3059\u3002<br \/>\n*   \u795e\u7d4c\u7d44\u7e54\u306b\u304a\u3051\u308bCRISPR\u306e\u30c7\u30fc\u30bf\u5b89\u5168\u6027\u306b\u95a2\u3059\u308b\u61f8\u5ff5\uff1a 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