{"id":64794,"date":"2025-12-25T14:22:16","date_gmt":"2025-12-25T05:22:16","guid":{"rendered":"https:\/\/www.marketresearch.co.jp\/insights\/alpha-mannosidosis-market-mordor\/"},"modified":"2026-09-22T04:11:24","modified_gmt":"2026-09-21T19:11:24","slug":"alpha-mannosidosis-market-mordor","status":"publish","type":"post","link":"https:\/\/www.marketresearch.co.jp\/insights\/alpha-mannosidosis-market-mordor\/","title":{"rendered":"\u30a2\u30eb\u30d5\u30a1\u30fb\u30de\u30f3\u30ce\u30b7\u30c9\u30fc\u30b7\u30b9\u5e02\u5834\u898f\u6a21\u30fb\u30b7\u30a7\u30a2\u5206\u6790 &#8211; \u6210\u9577\u30c8\u30ec\u30f3\u30c9\u3068\u4e88\u6e2c (2025-2030\u5e74)"},"content":{"rendered":"<p><img decoding=\"async\" src=\"https:\/\/www.marketresearch.co.jp\/insights\/wp-content\/uploads\/2025\/12\/alpha-mannosidosis-market-1753555075527-major-players.webp\" alt=\"Chart\" class=\"aligncenter size-full\" \/><\/p>\n<p><img decoding=\"async\" src=\"https:\/\/www.marketresearch.co.jp\/insights\/wp-content\/uploads\/2025\/12\/alpha-mannosidosis-market-size-image-1753472263894.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 &amp; \u5e02\u5834\u306e\u5b9a\u7fa9<\/p>\n<\/li>\n<li class=\"toc-level-2\">\n          1.2 \u8abf\u67fb\u306e\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\u89b3<\/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 Lamzede (Velmanase Alfa) ERT\u306e\u627f\u8a8d\u5f8c\u63a1\u7528\u306e\u5897\u52a0<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          4.2.2 \u30e9\u30a4\u30bd\u30be\u30fc\u30e0\u75c5\u306e\u65b0\u751f\u5150\u30b9\u30af\u30ea\u30fc\u30cb\u30f3\u30b0\u8a66\u9a13\u306e\u5897\u52a0<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          4.2.3 \u4eba\u9053\u7684\u898b\u5730\u304b\u3089\u306e\u4f7f\u7528\u304a\u3088\u3073\u65e9\u671f\u30a2\u30af\u30bb\u30b9\u30d7\u30ed\u30b0\u30e9\u30e0\u306e\u62e1\u5927<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          4.2.4 \u907a\u4f1d\u5b50\u6cbb\u7642\u30d1\u30a4\u30d7\u30e9\u30a4\u30f3\u306e\u30d6\u30ec\u30fc\u30af\u30b9\u30eb\u30fc\uff08AAV\u304a\u3088\u3073LNP\u30d7\u30e9\u30c3\u30c8\u30d5\u30a9\u30fc\u30e0\uff09<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          4.2.5 EU\u306b\u304a\u3051\u308b\u8907\u6570\u56fd\u9593\u3067\u306e\u5e0c\u5c11\u75be\u75c5\u7528\u533b\u85ac\u54c1\u511f\u9084\u306e\u8abf\u548c<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          4.2.6 AI\u3092\u6d3b\u7528\u3057\u305f\u8d85\u5e0c\u5c11\u75be\u60a3\u60a3\u8005\u767a\u898b\u30a2\u30eb\u30b4\u30ea\u30ba\u30e0<\/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 \u9ad8\u984d\u306a\u5e74\u9593\u6cbb\u7642\u8cbb\u3068\u4fa1\u683c\u8a2d\u5b9a\u3078\u306e\u53cd\u767a<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          4.3.2 \u73fe\u5728\u306eERT\u306e\u8840\u6db2\u8133\u95a2\u9580\u900f\u904e\u6027\u306e\u9650\u754c<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          4.3.3 \u9577\u671f\u7684\u306a\u5b9f\u4e16\u754c\u5b89\u5168\u6027\u30c7\u30fc\u30bf\u306e\u4e0d\u8db3<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          4.3.4 \u9aa8\u9ac4\u30c9\u30ca\u30fc\u9069\u5408\u306e\u7af6\u4e89\u306b\u3088\u308bHSCT\u63a1\u7528\u306e\u5236\u9650<\/p>\n<\/li>\n<\/ul>\n<\/li>\n<li class=\"toc-level-2\">\n          4.4 \u30b5\u30d7\u30e9\u30a4\u30c1\u30a7\u30fc\u30f3\u5206\u6790<\/p>\n<\/li>\n<li class=\"toc-level-2\">\n          4.5 \u898f\u5236\u74b0\u5883<\/p>\n<\/li>\n<li class=\"toc-level-2\">\n          4.6 \u6280\u8853\u7684\u5c55\u671b<\/p>\n<\/li>\n<li class=\"toc-level-2\">\n          4.7 \u30dd\u30fc\u30bf\u30fc\u306e5\u3064\u306e\u529b\u5206\u6790<\/p>\n<ul>\n<li class=\"toc-level-3\">\n          4.7.1 \u4f9b\u7d66\u8005\u306e\u4ea4\u6e09\u529b<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          4.7.2 \u8cb7\u3044\u624b\u306e\u4ea4\u6e09\u529b<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          4.7.3 \u65b0\u898f\u53c2\u5165\u306e\u8105\u5a01<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          4.7.4 \u4ee3\u66ff\u54c1\u306e\u8105\u5a01<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          4.7.5 \u7af6\u4e89\u4e0a\u306e\u5bfe\u6297\u95a2\u4fc2<\/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 \u9175\u7d20\u88dc\u5145\u7642\u6cd5<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          5.1.2 \u9020\u8840\u5e79\u7d30\u80de\u79fb\u690d<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          5.1.3 \u907a\u4f1d\u5b50\u6cbb\u7642<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          5.1.4 \u652f\u6301\u7642\u6cd5 &amp; \u88dc\u52a9\u7642\u6cd5<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          5.1.5 \u6cbb\u9a13\u85ac\u7406\u5b66\u7684\u30b7\u30e3\u30da\u30ed\u30f3<\/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 \u9759\u8108\u5185\u6ce8\u5165<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          5.2.2 \u5916\u79d1\u7684\u79fb\u690d\u6295\u4e0e<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          5.2.3 \u9ac4\u8154\u5185 \/ \u4e2d\u67a2\u795e\u7d4c\u7cfb\u76f4\u63a5\u9001\u9054<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          5.2.4 \u5168\u8eab\u6027\u30a6\u30a4\u30eb\u30b9\u30d9\u30af\u30bf\u30fc\u9001\u9054<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          5.2.5 \u305d\u306e\u4ed6\u306e\u7d4c\u8def<\/p>\n<\/li>\n<\/ul>\n<\/li>\n<li class=\"toc-level-2\">\n          5.3 \u5730\u57df\u5225<\/p>\n<ul>\n<li class=\"toc-level-3\">\n          5.3.1 \u5317\u7c73<\/p>\n<\/li>\n<li class=\"toc-level-4\">\n          5.3.1.1 \u7c73\u56fd<\/p>\n<\/li>\n<li class=\"toc-level-4\">\n          5.3.1.2 \u30ab\u30ca\u30c0<\/p>\n<\/li>\n<li class=\"toc-level-4\">\n          5.3.1.3 \u30e1\u30ad\u30b7\u30b3<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          5.3.2 \u30e8\u30fc\u30ed\u30c3\u30d1<\/p>\n<\/li>\n<li class=\"toc-level-4\">\n          5.3.2.1 \u30c9\u30a4\u30c4<\/p>\n<\/li>\n<li class=\"toc-level-4\">\n          5.3.2.2 \u30a4\u30ae\u30ea\u30b9<\/p>\n<\/li>\n<li class=\"toc-level-4\">\n          5.3.2.3 \u30d5\u30e9\u30f3\u30b9<\/p>\n<\/li>\n<li class=\"toc-level-4\">\n          5.3.2.4 \u30a4\u30bf\u30ea\u30a2<\/p>\n<\/li>\n<li class=\"toc-level-4\">\n          5.3.2.5 \u30b9\u30da\u30a4\u30f3<\/p>\n<\/li>\n<li class=\"toc-level-4\">\n          5.3.2.6 \u305d\u306e\u4ed6\u306e\u30e8\u30fc\u30ed\u30c3\u30d1<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          5.3.3 \u30a2\u30b8\u30a2\u592a\u5e73\u6d0b<\/p>\n<\/li>\n<li class=\"toc-level-4\">\n          5.3.3.1 \u4e2d\u56fd<\/p>\n<\/li>\n<li class=\"toc-level-4\">\n          5.3.3.2 \u65e5\u672c<\/p>\n<\/li>\n<li class=\"toc-level-4\">\n          5.3.3.3 \u30a4\u30f3\u30c9<\/p>\n<\/li>\n<li class=\"toc-level-4\">\n          5.3.3.4 \u97d3\u56fd<\/p>\n<\/li>\n<li class=\"toc-level-4\">\n          5.3.3.5 \u30aa\u30fc\u30b9\u30c8\u30e9\u30ea\u30a2<\/p>\n<\/li>\n<li class=\"toc-level-4\">\n          5.3.3.6 \u305d\u306e\u4ed6\u306e\u30a2\u30b8\u30a2\u592a\u5e73\u6d0b<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          5.3.4 \u4e2d\u6771 &amp; \u30a2\u30d5\u30ea\u30ab<\/p>\n<\/li>\n<li class=\"toc-level-4\">\n          5.3.4.1 GCC<\/p>\n<\/li>\n<li class=\"toc-level-4\">\n          5.3.4.2 \u5357\u30a2\u30d5\u30ea\u30ab<\/p>\n<\/li>\n<li class=\"toc-level-4\">\n          5.3.4.3 \u305d\u306e\u4ed6\u306e\u4e2d\u6771 &amp; \u30a2\u30d5\u30ea\u30ab<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          5.3.5 \u5357\u7c73<\/p>\n<\/li>\n<li class=\"toc-level-4\">\n          5.3.5.1 \u30d6\u30e9\u30b8\u30eb<\/p>\n<\/li>\n<li class=\"toc-level-4\">\n          5.3.5.2 \u30a2\u30eb\u30bc\u30f3\u30c1\u30f3<\/p>\n<\/li>\n<li class=\"toc-level-4\">\n          5.3.5.3 \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\u60c5\u52e2<\/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 (\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\u30fb\u30b5\u30fc\u30d3\u30b9\u3001\u304a\u3088\u3073\u6700\u8fd1\u306e\u52d5\u5411\u3092\u542b\u3080)<\/p>\n<ul>\n<li class=\"toc-level-3\">\n          6.3.1 Chiesi Farmaceutici S.p.A.<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          6.3.2 Sobi (Swedish Orphan Biovitrum)<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          6.3.3 Orchard Therapeutics plc<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          6.3.4 Avrobio Inc.<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          6.3.5 Ultragenyx Pharmaceutical Inc.<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          6.3.6 Regenxbio Inc.<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          6.3.7 Rocket Pharmaceuticals Inc.<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          6.3.8 M6P Therapeutics<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          6.3.9 SmartPharm Therapeutics<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          6.3.10 CSL Behring LLC<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          6.3.11 Takeda Pharmaceutical Company Ltd.<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          6.3.12 JCR Pharmaceuticals Co., Ltd.<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          6.3.13 Leadiant Biosciences Inc.<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          6.3.14 ArmaGen Inc.<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          6.3.15 Sangamo Therapeutics<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          6.3.16 Astellas Gene Therapies<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          6.3.17 Bluebird Bio Inc.<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          6.3.18 Genethon<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          6.3.19 Passage Bio<\/p>\n<\/li>\n<li class=\"toc-level-3\">\n          6.3.20 Denali Therapeutics<\/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>\u300c\u30a2\u30eb\u30d5\u30a1\u30de\u30f3\u30ce\u30b7\u30c9\u30fc\u30b7\u30b9\u5e02\u5834\u898f\u6a21\u3001\u30b7\u30a7\u30a2\u30012030\u5e74\u6210\u9577\u30c8\u30ec\u30f3\u30c9\u30ec\u30dd\u30fc\u30c8\u300d\u306e\u5e02\u5834\u6982\u8981\u306b\u3064\u3044\u3066\u3001\u8a73\u7d30\u306b\u8981\u7d04\u3044\u305f\u3057\u307e\u3059\u3002<\/p>\n<p>&#8212;<\/p>\n<p># \u30a2\u30eb\u30d5\u30a1\u30de\u30f3\u30ce\u30b7\u30c9\u30fc\u30b7\u30b9\u5e02\u5834\u6982\u8981\uff1a\u6210\u9577\u30c8\u30ec\u30f3\u30c9\u3068\u4e88\u6e2c\uff082025\u5e74\uff5e2030\u5e74\uff09<\/p>\n<p>\u672c\u30ec\u30dd\u30fc\u30c8\u306f\u3001\u30a2\u30eb\u30d5\u30a1\u30de\u30f3\u30ce\u30b7\u30c9\u30fc\u30b7\u30b9\u5e02\u5834\u306e\u73fe\u72b6\u3068\u5c06\u6765\u306e\u5c55\u671b\u3092\u8a73\u7d30\u306b\u5206\u6790\u3057\u305f\u3082\u306e\u3067\u3059\u3002\u30a2\u30eb\u30d5\u30a1\u30de\u30f3\u30ce\u30b7\u30c9\u30fc\u30b7\u30b9\u306f\u3001\u30e9\u30a4\u30bd\u30bd\u30fc\u30e0\u84c4\u7a4d\u75c7\u306e\u4e00\u7a2e\u3067\u3042\u308a\u3001\u5e0c\u5c11\u75be\u60a3\u3067\u3042\u308b\u305f\u3081\u3001\u305d\u306e\u6cbb\u7642\u6cd5\u3084\u5e02\u5834\u52d5\u5411\u306f\u7279\u5b9a\u306e\u8981\u56e0\u306b\u5927\u304d\u304f\u5de6\u53f3\u3055\u308c\u307e\u3059\u3002<\/p>\n<p>1. \u5e02\u5834\u306e\u5168\u4f53\u50cf<\/p>\n<p>\u30a2\u30eb\u30d5\u30a1\u30de\u30f3\u30ce\u30b7\u30c9\u30fc\u30b7\u30b9\u5e02\u5834\u306f\u30012025\u5e74\u306b\u306f4\u51043,310\u4e07\u7c73\u30c9\u30eb\u3068\u63a8\u5b9a\u3055\u308c\u30012030\u5e74\u306b\u306f9\u51043,850\u4e07\u7c73\u30c9\u30eb\u306b\u9054\u3059\u308b\u3068\u4e88\u6e2c\u3055\u308c\u3066\u3044\u307e\u3059\u3002\u4e88\u6e2c\u671f\u9593\uff082025\u5e74\uff5e2030\u5e74\uff09\u306b\u304a\u3051\u308b\u5e74\u5e73\u5747\u6210\u9577\u7387\uff08CAGR\uff09\u306f16.40%\u3068\u3001\u4e8c\u6841\u6210\u9577\u304c\u898b\u8fbc\u307e\u308c\u3066\u3044\u307e\u3059\u3002\u3053\u306e\u529b\u5f37\u3044\u6210\u9577\u306f\u3001\u4e3b\u306b\u30d9\u30eb\u30de\u30ca\u30fc\u30bc\u30a2\u30eb\u30d5\u30a1\uff08velmanase alfa\uff09\u306e\u627f\u8a8d\u5f8c\u9700\u8981\u306e\u5897\u52a0\u3001\u907a\u4f1d\u5b50\u6cbb\u7642\u306e\u30d1\u30a4\u30d7\u30e9\u30a4\u30f3\u9032\u5c55\u3001\u305d\u3057\u3066\u65b0\u751f\u5150\u30b9\u30af\u30ea\u30fc\u30cb\u30f3\u30b0\u30d7\u30ed\u30b0\u30e9\u30e0\u306e\u62e1\u5927\u306b\u3088\u3063\u3066\u727d\u5f15\u3055\u308c\u3066\u3044\u307e\u3059\u3002<\/p>\n<p>\u8a3a\u65ad\u52aa\u529b\u306e\u5f37\u5316\u306b\u3088\u308a\u3001\u75c7\u72b6\u767a\u73fe\u5f8c\u304b\u3089\u75c7\u72b6\u767a\u73fe\u524d\u6bb5\u968e\u3067\u306e\u767a\u898b\u3078\u3068\u30b7\u30d5\u30c8\u3057\u3066\u304a\u308a\u3001\u3053\u308c\u306b\u3088\u308a\u7d045,000\u4eba\u3068\u3055\u308c\u308b\u8a3a\u65ad\u6e08\u307f\u60a3\u8005\u306e\u5bfe\u8c61\u30d7\u30fc\u30eb\u304c\u62e1\u5927\u3057\u3066\u3044\u307e\u3059\u3002\u5e02\u5834\u306e\u7af6\u4e89\u306f\u6fc0\u5316\u3057\u3066\u304a\u308a\u3001\u5358\u56de\u6cbb\u7642\u306e\u907a\u4f1d\u5b50\u6cbb\u7642\u304c\u3001\u73fe\u5728\u306e\u53ce\u76ca\u3092\u652f\u914d\u3057\u3066\u3044\u308b\u6162\u6027\u7684\u306a\u8f38\u6db2\u30e2\u30c7\u30eb\u306b\u6311\u6226\u3057\u3066\u3044\u307e\u3059\u3002\u30e8\u30fc\u30ed\u30c3\u30d1\u306f\u65e9\u671f\u306e\u511f\u9084\u5236\u5ea6\u306b\u3088\u308a\u5148\u884c\u8005\u5229\u76ca\u3092\u4eab\u53d7\u3057\u3066\u3044\u307e\u3059\u304c\u30012024\u5e74\u306e\u7c73\u56fdFDA\u306b\u3088\u308bLamzede\u627f\u8a8d\u5f8c\u3001\u5317\u7c73\u304c\u5168\u4f53\u306e\u6210\u9577\u3092\u727d\u5f15\u3057\u3066\u3044\u307e\u3059\u3002\u3057\u304b\u3057\u3001\u5e74\u9593\u6cbb\u7642\u8cbb\u304c\u4f9d\u7136\u3068\u3057\u3066\u9ad8\u984d\u3067\u3042\u308b\u305f\u3081\u3001\u4fa1\u683c\u306b\u654f\u611f\u306a\u5730\u57df\u3067\u306e\u77ed\u671f\u7684\u306a\u666e\u53ca\u306f\u6291\u5236\u3055\u308c\u3066\u304a\u308a\u3001\u30e1\u30fc\u30ab\u30fc\u306f\u4fa1\u5024\u30d9\u30fc\u30b9\u306e\u5951\u7d04\u3084\u60a3\u8005\u652f\u63f4\u57fa\u91d1\u306e\u6a21\u7d22\u3092\u4f59\u5100\u306a\u304f\u3055\u308c\u3066\u3044\u307e\u3059\u3002<\/p>\n<p>\u5e02\u5834\u306e\u96c6\u4e2d\u5ea6\u306f\u300c\u9ad8\u3044\u300d\u3068\u8a55\u4fa1\u3055\u308c\u3066\u304a\u308a\u3001\u5c11\u6570\u306e\u4e3b\u8981\u30d7\u30ec\u30a4\u30e4\u30fc\u304c\u5e02\u5834\u3092\u30ea\u30fc\u30c9\u3057\u3066\u3044\u308b\u72b6\u6cc1\u3067\u3059\u3002\u5730\u57df\u5225\u3067\u306f\u3001\u5317\u7c73\u304c\u6700\u5927\u306e\u5e02\u5834\u3067\u3042\u308a\u3001\u30a2\u30b8\u30a2\u592a\u5e73\u6d0b\u5730\u57df\u304c\u6700\u3082\u6025\u901f\u306b\u6210\u9577\u3059\u308b\u5e02\u5834\u3068\u4e88\u6e2c\u3055\u308c\u3066\u3044\u307e\u3059\u3002<\/p>\n<p>2. \u4e3b\u8981\u306a\u30ec\u30dd\u30fc\u30c8\u306e\u30dd\u30a4\u30f3\u30c8<\/p>\n<p>*   \u6cbb\u7642\u30bf\u30a4\u30d7\u5225: 2024\u5e74\u306b\u304a\u3044\u3066\u3001\u9175\u7d20\u88dc\u5145\u7642\u6cd5\uff08ERT\uff09\u304c\u30a2\u30eb\u30d5\u30a1\u30de\u30f3\u30ce\u30b7\u30c9\u30fc\u30b7\u30b9\u5e02\u5834\u30b7\u30a7\u30a2\u306e82.4%\u3092\u5360\u3081\u307e\u3057\u305f\u3002\u4e00\u65b9\u3001\u907a\u4f1d\u5b50\u6cbb\u7642\u306f2030\u5e74\u307e\u3067\u306b18.4%\u306eCAGR\u3067\u6025\u6210\u9577\u3059\u308b\u3068\u4e88\u6e2c\u3055\u308c\u3066\u3044\u307e\u3059\u3002<br \/>\n*   \u6295\u4e0e\u7d4c\u8def\u5225: 2024\u5e74\u306b\u304a\u3044\u3066\u3001\u9759\u8108\u5185\u8f38\u6db2\u304c\u30a2\u30eb\u30d5\u30a1\u30de\u30f3\u30ce\u30b7\u30c9\u30fc\u30b7\u30b9\u5e02\u5834\u898f\u6a21\u306e81.8%\u3092\u5360\u3081\u307e\u3057\u305f\u3002\u5168\u8eab\u6027\u30a6\u30a4\u30eb\u30b9\u30d9\u30af\u30bf\u30fc\u9001\u9054\u306f\u30012030\u5e74\u307e\u3067\u306b17.3%\u306eCAGR\u3067\u6210\u9577\u3059\u308b\u3068\u4e88\u6e2c\u3055\u308c\u3066\u3044\u307e\u3059\u3002<br \/>\n*   \u5730\u57df\u5225: 2024\u5e74\u306b\u304a\u3044\u3066\u3001\u30e8\u30fc\u30ed\u30c3\u30d1\u304c\u53ce\u76ca\u306e41.3%\u3092\u5360\u3081\u307e\u3057\u305f\u3002\u3057\u304b\u3057\u3001\u5317\u7c73\u306f2025\u5e74\uff5e2030\u5e74\u306e\u671f\u9593\u306b15.7%\u306eCAGR\u3067\u6700\u3082\u901f\u3044\u6210\u9577\u3092\u9042\u3052\u308b\u3068\u4e88\u6e2c\u3055\u308c\u3066\u3044\u307e\u3059\u3002<\/p>\n<p>3. \u5e02\u5834\u306e\u63a8\u9032\u8981\u56e0\u3068\u5f71\u97ff<\/p>\n<p>\u5e02\u5834\u306e\u6210\u9577\u3092\u5f8c\u62bc\u3057\u3059\u308b\u4e3b\u306a\u8981\u56e0\u306f\u4ee5\u4e0b\u306e\u901a\u308a\u3067\u3059\u3002<\/p>\n<p>*   \u30d9\u30eb\u30de\u30ca\u30fc\u30bc\u30a2\u30eb\u30d5\u30a1ERT\u306e\u627f\u8a8d\u5f8c\u666e\u53ca\uff08CAGR\u3078\u306e\u5f71\u97ff: +3.20%\uff09:<br \/>\n    2024\u5e746\u6708\u306e\u7c73\u56fdFDA\u306b\u3088\u308bLamzede\u627f\u8a8d\u306f\u3001\u3053\u308c\u307e\u3067\u8f38\u5165\u3084\u30b3\u30f3\u30d1\u30c3\u30b7\u30e7\u30cd\u30fc\u30c8\u30e6\u30fc\u30b9\u306b\u983c\u3063\u3066\u3044\u305f\u7c73\u56fd\u60a3\u8005\u3078\u306e\u5546\u696d\u7684\u30a2\u30af\u30bb\u30b9\u3092\u53ef\u80fd\u306b\u3057\u307e\u3057\u305f\u3002\u81e8\u5e8a\u8a66\u9a13\u30c7\u30fc\u30bf\u3067\u306f\u300152\u9031\u3067\u8840\u6e05\u30aa\u30ea\u30b4\u7cd6\u304c77.6%\u6e1b\u5c11\u3057\u305f\u3053\u3068\u304c\u793a\u3055\u308c\u3066\u304a\u308a\u3001SPARKLE\u30ec\u30b8\u30b9\u30c8\u30ea\u304c15\u5e74\u9593\u306e\u30a2\u30a6\u30c8\u30ab\u30e0\u3092\u8ffd\u8de1\u3059\u308b\u3053\u3068\u3067\u3001\u51e6\u65b9\u533b\u306e\u4fe1\u983c\u3092\u5f37\u5316\u3057\u3066\u3044\u307e\u3059\u3002\u5c0f\u5150\u671f\u65e9\u671f\u306e\u6cbb\u7642\u958b\u59cb\u306f\u8074\u899a\u3084\u514d\u75ab\u30d7\u30ed\u30d5\u30a1\u30a4\u30eb\u306e\u6539\u5584\u3068\u76f8\u95a2\u3057\u3066\u304a\u308a\u3001\u8a3a\u65ad\u5f8c\u65e9\u671f\u306e\u6cbb\u7642\u3092\u63a8\u5968\u3059\u308b\u30ac\u30a4\u30c9\u30e9\u30a4\u30f3\u306e\u66f4\u65b0\u304c\u9032\u3093\u3067\u3044\u307e\u3059\u3002\u9031\u3054\u3068\u306e\u8f38\u6db2\u306b\u306f\u5c02\u9580\u65bd\u8a2d\u304c\u5fc5\u8981\u3067\u3059\u304c\u3001\u5728\u5b85\u30b1\u30a2\u30d7\u30ed\u30b0\u30e9\u30e0\u306e\u62e1\u5927\u304c\u5b89\u5b9a\u3057\u305f\u60a3\u8005\u306e\u7269\u6d41\u8ca0\u62c5\u3092\u8efd\u6e1b\u3057\u3066\u3044\u307e\u3059\u3002\u3053\u308c\u306b\u3088\u308a\u3001\u7c73\u56fd\u3067\u306e\u6025\u901f\u306a\u666e\u53ca\u304c\u30a2\u30eb\u30d5\u30a1\u30de\u30f3\u30ce\u30b7\u30c9\u30fc\u30b7\u30b9\u5e02\u5834\u306b\u77ed\u671f\u7684\u306a\u30dc\u30ea\u30e5\u30fc\u30e0\u3092\u52a0\u3048\u3066\u3044\u307e\u3059\u3002<\/p>\n<p>*   \u30e9\u30a4\u30bd\u30bd\u30fc\u30e0\u84c4\u7a4d\u75c7\u306e\u65b0\u751f\u5150\u30b9\u30af\u30ea\u30fc\u30cb\u30f3\u30b0\u30d1\u30a4\u30ed\u30c3\u30c8\u30d7\u30ed\u30b0\u30e9\u30e0\u306e\u5897\u52a0\uff08CAGR\u3078\u306e\u5f71\u97ff: +2.80%\uff09:<br \/>\n    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  \u907a\u4f1d\u5b50\u6cbb\u7642\u30d1\u30a4\u30d7\u30e9\u30a4\u30f3\u306e\u30d6\u30ec\u30fc\u30af\u30b9\u30eb\u30fc\uff08CAGR\u3078\u306e\u5f71\u97ff: +2.10%\uff09:<br \/>\n    \u4ed6\u306e\u9069\u5fdc\u75c7\u30678\u3064\u306e\u30a2\u30c7\u30ce\u968f\u4f34\u30a6\u30a4\u30eb\u30b9\uff08AAV\uff09\u907a\u4f1d\u5b50\u6cbb\u7642\u304c\u898f\u5236\u5f53\u5c40\u306e\u627f\u8a8d\u3092\u5f97\u3066\u304a\u308a\u3001\u8d85\u5e0c\u5c11\u75be\u60a3\u8a2d\u5b9a\u306b\u304a\u3051\u308b\u30d7\u30e9\u30c3\u30c8\u30d5\u30a9\u30fc\u30e0\u306e\u5b89\u5168\u6027\u3068\u88fd\u9020\u306e\u30ea\u30b9\u30af\u3092\u4f4e\u6e1b\u3057\u3066\u3044\u307e\u3059\u3002\u8102\u8cea\u30ca\u30ce\u7c92\u5b50\uff08LNP\uff09mRNA\u69cb\u9020\u306f\u3001\u30b2\u30ce\u30e0\u7d71\u5408\u306a\u3057\u306b\u518d\u6295\u4e0e\u53ef\u80fd\u306a\u767a\u73fe\u3092\u53ef\u80fd\u306b\u3057\u3001\u6b8b\u5b58\u3059\u308b\u30d9\u30af\u30bf\u30fc\u514d\u75ab\u306e\u61f8\u5ff5\u306b\u5bfe\u51e6\u3059\u308b\u8a2d\u8a08\u3067\u3059\u3002M6P 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\u30d9\u30eb\u30de\u30ca\u30fc\u30bc\u30a2\u30eb\u30d5\u30a1\u306f\u4f53\u6027\u30aa\u30ea\u30b4\u7cd6\u306e\u84c4\u7a4d\u3092\u30af\u30ea\u30a2\u3057\u307e\u3059\u304c\u3001\u795e\u7d4c\u7d44\u7e54\u306b\u306f\u5230\u9054\u305b\u305a\u3001\u8a8d\u77e5\u6a5f\u80fd\u306e\u4f4e\u4e0b\u3092\u672a\u89e3\u6c7a\u306e\u307e\u307e\u306b\u3057\u3066\u3044\u307e\u3059\u3002\u9020\u8840\u5e79\u7d30\u80de\u79fb\u690d\uff08HSCT\uff09\u306f\u9175\u7d20\u3092\u8840\u6db2\u8133\u95a2\u9580\u3092\u8d8a\u3048\u3066\u9001\u9054\u3067\u304d\u307e\u3059\u304c\u3001\u524d\u51e6\u7f6e\u306e\u6bd2\u6027\u3084\u30c9\u30ca\u30fc\u30de\u30c3\u30c1\u30f3\u30b0\u306e\u8ab2\u984c\u3092\u4f34\u3046\u305f\u3081\u3001\u91cd\u75c7\u306e\u5c0f\u5150\u75c7\u4f8b\u306b\u9650\u5b9a\u3055\u308c\u307e\u3059\u3002\u53d7\u5bb9\u4f53\u4ecb\u5728\u6027\u8f38\u9001\u3084\u9ac4\u8154\u5185\u8f38\u6db2\u306b\u95a2\u3059\u308b\u63a2\u7d22\u7684\u7814\u7a76\u306f\u524d\u81e8\u5e8a\u6bb5\u968e\u306b\u3068\u3069\u307e\u3063\u3066\u304a\u308a\u3001\u795e\u7d4c\u5b66\u7684\u8ca0\u62c5\u306f\u307b\u3068\u3093\u3069\u7ba1\u7406\u3055\u308c\u3066\u3044\u307e\u305b\u3093\u3002\u4f53\u6027\u7684\u306a\u6539\u5584\u304c\u8a8d\u77e5\u6a5f\u80fd\u306e\u9000\u884c\u3092\u6b62\u3081\u3089\u308c\u306a\u3044\u5834\u5408\u3001\u4ecb\u8b77\u8005\u306f\u4e0d\u6e80\u3092\u8868\u660e\u3057\u3001ERT\u306e\u9075\u5b88\u7387\u3092\u4f4e\u4e0b\u3055\u305b\u308b\u53ef\u80fd\u6027\u304c\u3042\u308a\u307e\u3059\u3002CNS\u6d78\u900f\u6027\u30bd\u30ea\u30e5\u30fc\u30b7\u30e7\u30f3\u304c\u306a\u3051\u308c\u3070\u3001\u30a2\u30eb\u30d5\u30a1\u30de\u30f3\u30ce\u30b7\u30c9\u30fc\u30b7\u30b9\u5e02\u5834\u306f\u795e\u7d4c\u4fdd\u8b77\u7684\u30a2\u30a6\u30c8\u30ab\u30e0\u306b\u5185\u5728\u3059\u308b\u6f5c\u5728\u7684\u4fa1\u5024\u3092\u5931\u3046\u3053\u3068\u306b\u306a\u308a\u307e\u3059\u3002<\/p>\n<p>* 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2024\u5e74\u3001\u9175\u7d20\u88dc\u5145\u7642\u6cd5\uff08ERT\uff09\u30bb\u30b0\u30e1\u30f3\u30c8\u306f\u30a2\u30eb\u30d5\u30a1\u30de\u30f3\u30ce\u30b7\u30c9\u30fc\u30b7\u30b9\u5e02\u5834\u306e82.4%\u3092\u5360\u3081\u3001\u30d9\u30eb\u30de\u30ca\u30fc\u30bc\u30a2\u30eb\u30d5\u30a1\u306e\u5e02\u5834\u5148\u884c\u8005\u3068\u3057\u3066\u306e\u512a\u4f4d\u6027\u304c\u78ba\u7acb\u3055\u308c\u3066\u3044\u307e\u3059\u3002\u4e00\u65b9\u30016\u3064\u306e\u5c0f\u5150\u75be\u60a3\u6307\u5b9a\u3068\u30d9\u30f3\u30c1\u30e3\u30fc\u30ad\u30e3\u30d4\u30bf\u30eb\u306e\u6d41\u5165\u306b\u652f\u3048\u3089\u308c\u305f\u907a\u4f1d\u5b50\u6cbb\u7642\u306f\u30012030\u5e74\u307e\u3067\u306b18.4%\u306eCAGR\u3092\u8a18\u9332\u3059\u308b\u3068\u4e88\u6e2c\u3055\u308c\u3066\u304a\u308a\u3001\u5e02\u5834\u306b\u7834\u58ca\u7684\u306a\u5909\u5316\u3092\u3082\u305f\u3089\u3059\u53ef\u80fd\u6027\u304c\u3042\u308a\u307e\u3059\u3002\u9020\u8840\u5e79\u7d30\u80de\u79fb\u690d\uff08HSCT\uff09\u306f\u3001\u91cd\u75c7\u306e\u795e\u7d4c\u5b66\u7684\u8868\u73fe\u578b\u3092\u6301\u3064\u60a3\u8005\u306b\u3068\u3063\u3066\u81e8\u5e8a\u7684\u610f\u7fa9\u3092\u4fdd\u6301\u3057\u3066\u304a\u308a\u30012000\u5e74\u4ee5\u964d\u306e\u751f\u5b58\u7387\u306f86%\u3068\u30012000\u5e74\u4ee5\u524d\u306e64%\u3068\u6bd4\u8f03\u3057\u3066\u6539\u5584\u3057\u3066\u3044\u307e\u3059\u3002\u8074\u899a\u3001\u514d\u75ab\u3001\u7406\u5b66\u7642\u6cd5\u3092\u542b\u3080\u652f\u6301\u7684\u306a\u591a\u5206\u91ce\u30b1\u30a2\u306f\u3001\u6a19\u6e96\u30d7\u30ed\u30c8\u30b3\u30eb\u304c\u6210\u719f\u3059\u308b\u306b\u3064\u308c\u3066\u62e1\u5927\u3057\u3066\u3044\u307e\u3059\u3002\u85ac\u7406\u5b66\u7684\u30b7\u30e3\u30da\u30ed\u30f3\u5019\u88dc\u306f\u3001\u30df\u30b9\u30d5\u30a9\u30fc\u30eb\u30c9\u3057\u305f\u30e9\u30a4\u30bd\u30bd\u30fc\u30e0\u9175\u7d20\u306e\u4fee\u6b63\u3092\u76ee\u6307\u3057\u3066\u3044\u307e\u3059\u304c\u3001\u307e\u3060\u4e3b\u8981\u306a\u81e8\u5e8a\u8a66\u9a13\u306b\u306f\u81f3\u3063\u3066\u3044\u307e\u305b\u3093\u3002<br \/>\n    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\/>\n    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Therapeutics\u306f\u3001\u6c38\u7d9a\u7684\u306a\u30b2\u30ce\u30e0\u7de8\u96c6\u3092\u56de\u907f\u3057\u306a\u304c\u3089\u767a\u73fe\u3092\u5ef6\u9577\u3067\u304d\u308b\u907a\u4f1d\u5b50\u30b3\u30fc\u30c9\u5316\u9175\u7d20\u30d7\u30e9\u30c3\u30c8\u30d5\u30a9\u30fc\u30e0\u3092\u8ffd\u6c42\u3057\u3066\u3044\u307e\u3059\u3002Kyowa Kirin\u5098\u4e0b\u3068\u306a\u3063\u305fOrchard Therapeutics\u306f\u3001Lenmeldy\u306e\u767a\u58f2\u304b\u3089\u5546\u696d\u7684\u5c02\u9580\u77e5\u8b58\u3092\u3082\u305f\u3089\u3057\u3001\u8cb7\u53ce\u4f01\u696d\u304c\u88fd\u9020\u304a\u3088\u3073\u5e02\u5834\u30a2\u30af\u30bb\u30b9\u80fd\u529b\u3092\u76f8\u4e92\u306b\u6d3b\u7528\u3067\u304d\u308b\u7acb\u5834\u306b\u3042\u308a\u307e\u3059\u3002<\/p>\n<p>\u6226\u7565\u7684\u63d0\u643a\u3082\u6d3b\u767a\u5316\u3057\u3066\u3044\u307e\u3059\u3002Sobi\u306fIonis\u3068\u306e\u63d0\u643a\u3092\u62e1\u5927\u3057\u3001\u5e0c\u5c11\u75be\u60a3\u3078\u306e\u30ea\u30fc\u30c1\u3092\u6df1\u3081\u307e\u3057\u305f\u3002REGENXBIO\u306fNippon Shinyaku\u3068\u63d0\u643a\u3057\u3001\u30a2\u30eb\u30d5\u30a1\u30de\u30f3\u30ce\u30b7\u30c9\u30fc\u30b7\u30b9\u5019\u88dc\u3068\u88fd\u9020\u30d7\u30e9\u30c3\u30c8\u30d5\u30a9\u30fc\u30e0\u3092\u5171\u6709\u3059\u308bMPS\u907a\u4f1d\u5b50\u6cbb\u7642\u306e\u5546\u696d\u5316\u3092\u9032\u3081\u3066\u3044\u307e\u3059\u3002Chiesi\u306e2024\u5e74\u306e\u7814\u7a76\u52a9\u6210\u91d1\u30a4\u30cb\u30b7\u30a2\u30c1\u30d6\u306f\u3001\u30e9\u30a4\u30bd\u30bd\u30fc\u30e0\u79d1\u5b66\u306b\u304a\u3051\u308b\u5b66\u8853\u7684\u30d6\u30ec\u30fc\u30af\u30b9\u30eb\u30fc\u3092\u4fc3\u9032\u3059\u308b\u3053\u3068\u3092\u76ee\u7684\u3068\u3057\u3066\u304a\u308a\u3001\u907a\u4f1d\u5b50\u6cbb\u7642\u306b\u3088\u308b\u7834\u58ca\u306b\u5bfe\u3059\u308b\u5148\u5236\u7684\u306a\u9632\u5fa1\u3092\u793a\u5506\u3057\u3066\u3044\u307e\u3059\u3002<\/p>\n<p>\u30db\u30ef\u30a4\u30c8\u30b9\u30da\u30fc\u30b9\u7af6\u4e89\u306f\u3001CNS\u6d78\u900f\u6027\u30e2\u30c0\u30ea\u30c6\u30a3\u306b\u96c6\u4e2d\u3057\u3066\u3044\u307e\u3059\u3002\u8aac\u5f97\u529b\u306e\u3042\u308b\u795e\u7d4c\u8a8d\u77e5\u4e0a\u306e\u5229\u76ca\u3092\u793a\u3059\u4f01\u696d\u306f\u3001\u30d9\u30eb\u30de\u30ca\u30fc\u30bc\u30a2\u30eb\u30d5\u30a1\u306e\u4f53\u6027\u306e\u307f\u306e\u7de9\u548c\u304b\u3089\u6025\u901f\u306b\u30b7\u30a7\u30a2\u3092\u7372\u5f97\u3059\u308b\u53ef\u80fd\u6027\u304c\u3042\u308a\u307e\u3059\u3002\u30e1\u30fc\u30ab\u30fc\u306e\u6210\u529f\u306f\u3001\u751f\u5316\u5b66\u7684\u30a8\u30f3\u30c9\u30dd\u30a4\u30f3\u30c8\u306e\u308f\u305a\u304b\u306a\u9055\u3044\u3067\u306f\u306a\u304f\u3001\u6301\u7d9a\u6027\u3001\u5229\u4fbf\u6027\u3001\u795e\u7d4c\u5b66\u7684\u6709\u52b9\u6027\u306b\u304a\u3051\u308b\u5dee\u5225\u5316\u306b\u304b\u304b\u3063\u3066\u3044\u307e\u3059\u3002\u30d1\u30a4\u30d7\u30e9\u30a4\u30f3\u306e\u591a\u69d8\u6027\u304c\u5897\u3059\u306b\u3064\u308c\u3066\u3001\u8907\u6570\u306e\u30e9\u30a4\u30bd\u30bd\u30fc\u30e0\u84c4\u7a4d\u75c7\u306b\u308f\u305f\u308b\u30dd\u30fc\u30c8\u30d5\u30a9\u30ea\u30aa\u30ec\u30d9\u30eb\u306e\u5c02\u9580\u77e5\u8b58\u304c\u3001\u652f\u6255\u8005\u3084\u898f\u5236\u5f53\u5c40\u3068\u306e\u4ea4\u6e09\u529b\u3092\u307e\u3059\u307e\u3059\u6c7a\u5b9a\u3059\u308b\u3067\u3057\u3087\u3046\u3002\u3053\u308c\u3089\u306e\u8981\u56e0\u304c\u8907\u5408\u7684\u306b\u3001\u30a2\u30eb\u30d5\u30a1\u30de\u30f3\u30ce\u30b7\u30c9\u30fc\u30b7\u30b9\u5e02\u5834\u306e\u30c0\u30a4\u30ca\u30df\u30c3\u30af\u306a\u7af6\u4e89\u72b6\u6cc1\u3092\u5f62\u6210\u3057\u3066\u3044\u307e\u3059\u3002<\/p>\n<p>8. \u30a2\u30eb\u30d5\u30a1\u30de\u30f3\u30ce\u30b7\u30c9\u30fc\u30b7\u30b9\u696d\u754c\u306e\u4e3b\u8981\u30ea\u30fc\u30c0\u30fc<\/p>\n<p>*   Chiesi Farmaceutici S.p.A.<br \/>\n*   Sobi<br \/>\n*   Orchard Therapeutics plc<br \/>\n*   Avrobio Inc.<br \/>\n*   Ultragenyx Pharmaceutical Inc.<br \/>\n    \uff08*\u514d\u8cac\u4e8b\u9805\uff1a\u4e3b\u8981\u30d7\u30ec\u30a4\u30e4\u30fc\u306f\u7279\u5b9a\u306e\u9806\u5e8f\u3067\u4e26\u3079\u3089\u308c\u3066\u3044\u307e\u305b\u3093\uff09<\/p>\n<p>9. \u6700\u8fd1\u306e\u696d\u754c\u52d5\u5411<\/p>\n<p>*   2025\u5e743\u6708: Sobi\u306fIonis Pharmaceuticals\u3068\u306e\u63d0\u643a\u3092\u62e1\u5927\u3057\u3001\u7c73\u56fd\u4ee5\u5916\u3067\u306e\u30aa\u30ec\u30b6\u30eb\u30bb\u30f3\u306e\u5546\u696d\u5316\u3092\u542b\u3081\u308b\u3053\u3068\u306b\u306a\u308a\u307e\u3057\u305f\u3002<br \/>\n*   2025\u5e741\u6708: REGENXBIO\u306fNippon Shinyaku\u30681\u51041,000\u4e07\u7c73\u30c9\u30eb\u306e\u63d0\u643a\u3092\u7d50\u3073\u3001RGX-121\u304a\u3088\u3073\u95a2\u9023\u3059\u308b\u30e9\u30a4\u30bd\u30bd\u30fc\u30e0\u84c4\u7a4d\u75c7\u907a\u4f1d\u5b50\u6cbb\u7642\u3092\u5bfe\u8c61\u3068\u3057\u307e\u3057\u305f\u3002<br \/>\n*   2024\u5e746\u6708: FDA\u306fLamzede\u3092\u627f\u8a8d\u3057\u3001\u7c73\u56fd\u306e\u30a2\u30eb\u30d5\u30a1\u30de\u30f3\u30ce\u30b7\u30c9\u30fc\u30b7\u30b9\u60a3\u8005\u3078\u306e\u9175\u7d20\u88dc\u5145\u7642\u6cd5\u30a2\u30af\u30bb\u30b9\u3092\u958b\u59cb\u3057\u307e\u3057\u305f\u3002<br \/>\n*   2024\u5e741\u6708: Kyowa Kirin\u306fOrchard Therapeutics\u306e\u8cb7\u53ce\u3092\u5b8c\u4e86\u3057\u3001\u5e0c\u5c11\u75be\u60a3\u907a\u4f1d\u5b50\u6cbb\u7642\u30d7\u30e9\u30c3\u30c8\u30d5\u30a9\u30fc\u30e0\u3092\u78ba\u4fdd\u3057\u307e\u3057\u305f\u3002<\/p>\n<p>&#8212;<\/p>\n<p>\u3053\u306e\u8981\u7d04\u306f\u3001\u63d0\u4f9b\u3055\u308c\u305f\u300cMarket Overview\u300d\u306e\u5185\u5bb9\u3092\u7db2\u7f85\u3057\u3001\u8a73\u7d30\u304b\u3064\u4e01\u5be7\u306a\u65e5\u672c\u8a9e\u3067\u8a18\u8ff0\u3044\u305f\u3057\u307e\u3057\u305f\u3002<\/p>\n<p>\u3053\u306e\u30ec\u30dd\u30fc\u30c8\u306f\u3001\u300c\u30b0\u30ed\u30fc\u30d0\u30eb\u03b1-\u30de\u30f3\u30ce\u30b7\u30c9\u30fc\u30b7\u30b9\u5e02\u5834\u300d\u306b\u95a2\u3059\u308b\u8a73\u7d30\u306a\u5206\u6790\u3092\u63d0\u4f9b\u3057\u3066\u3044\u307e\u3059\u3002\u5e02\u5834\u306e\u5b9a\u7fa9\u3001\u8abf\u67fb\u7bc4\u56f2\u3001\u8abf\u67fb\u65b9\u6cd5\u3001\u30a8\u30b0\u30bc\u30af\u30c6\u30a3\u30d6\u30b5\u30de\u30ea\u30fc\u3001\u5e02\u5834\u6982\u6cc1\u3001\u5e02\u5834\u898f\u6a21\u3068\u6210\u9577\u4e88\u6e2c\u3001\u7af6\u4e89\u74b0\u5883\u3001\u5e02\u5834\u6a5f\u4f1a\u3068\u5c06\u6765\u5c55\u671b\u3068\u3044\u3063\u305f\u4e3b\u8981\u306a\u30bb\u30af\u30b7\u30e7\u30f3\u3067\u69cb\u6210\u3055\u308c\u3066\u3044\u307e\u3059\u3002<\/p>\n<p>\u5e02\u5834\u306e\u6982\u8981\u3068\u52d5\u5411<br \/>\n\u03b1-\u30de\u30f3\u30ce\u30b7\u30c9\u30fc\u30b7\u30b9\u306f\u3001\u4e16\u754c\u306750\u4e07\uff5e100\u4e07\u51fa\u751f\u306b1\u4eba\u3068\u3044\u3046\u7a00\u306a\u767a\u751f\u7387\u3092\u6301\u3064\u8d85\u5e0c\u5c11\u75be\u60a3\u3067\u3042\u308a\u3001\u73fe\u5728\u8a3a\u65ad\u3055\u308c\u3066\u3044\u308b\u60a3\u8005\u6570\u306f5,000\u4eba\u672a\u6e80\u3068\u3055\u308c\u3066\u3044\u307e\u3059\u3002<\/p>\n<p>\u5e02\u5834\u306e\u6210\u9577\u3092\u727d\u5f15\u3059\u308b\u4e3b\u306a\u8981\u56e0\u3068\u3057\u3066\u306f\u3001Lamzede (Velmanase Alfa) \u9175\u7d20\u88dc\u5145\u7642\u6cd5 (ERT) \u306e\u627f\u8a8d\u5f8c\u306e\u5229\u7528\u5897\u52a0\u3001\u30e9\u30a4\u30bd\u30bd\u30fc\u30e0\u75c5\u306b\u5bfe\u3059\u308b\u65b0\u751f\u5150\u30b9\u30af\u30ea\u30fc\u30cb\u30f3\u30b0\u30d1\u30a4\u30ed\u30c3\u30c8\u30d7\u30ed\u30b0\u30e9\u30e0\u306e\u62e1\u5927\u3001\u30b3\u30f3\u30d1\u30c3\u30b7\u30e7\u30cd\u30fc\u30c8\u4f7f\u7528\u304a\u3088\u3073\u65e9\u671f\u30a2\u30af\u30bb\u30b9\u30d7\u30ed\u30b0\u30e9\u30e0\u306e\u5c55\u958b\u304c\u6319\u3052\u3089\u308c\u307e\u3059\u3002\u307e\u305f\u3001AAV\u304a\u3088\u3073LNP\u30d7\u30e9\u30c3\u30c8\u30d5\u30a9\u30fc\u30e0\u3092\u7528\u3044\u305f\u907a\u4f1d\u5b50\u6cbb\u7642\u30d1\u30a4\u30d7\u30e9\u30a4\u30f3\u306b\u304a\u3051\u308b\u753b\u671f\u7684\u306a\u9032\u5c55\u3001EU\u306b\u304a\u3051\u308b\u5e0c\u5c11\u75be\u75c5\u7528\u533b\u85ac\u54c1\u306e\u511f\u9084\u306b\u95a2\u3059\u308b\u591a\u56fd\u9593\u3067\u306e\u8abf\u548c\u3001AI\u3092\u6d3b\u7528\u3057\u305f\u8d85\u5e0c\u5c11\u75be\u60a3\u60a3\u8005\u767a\u898b\u30a2\u30eb\u30b4\u30ea\u30ba\u30e0\u306e\u767b\u5834\u3082\u5e02\u5834\u62e1\u5927\u306b\u5bc4\u4e0e\u3057\u3066\u3044\u307e\u3059\u3002<\/p>\n<p>\u4e00\u65b9\u3067\u3001\u5e02\u5834\u306e\u6210\u9577\u3092\u6291\u5236\u3059\u308b\u8981\u56e0\u3082\u5b58\u5728\u3057\u307e\u3059\u3002\u5e74\u9593\u6cbb\u7642\u8cbb\u304c\u9ad8\u984d\u3067\u3042\u308b\u3053\u3068\u306b\u3088\u308b\u4fa1\u683c\u3078\u306e\u53cd\u767a\u3001\u73fe\u884c\u306e\u9175\u7d20\u88dc\u5145\u7642\u6cd5\u304c\u8840\u6db2\u8133\u95a2\u9580\u3092\u5341\u5206\u306b\u900f\u904e\u3057\u306a\u3044\u3053\u3068\u3001\u9577\u671f\u7684\u306a\u5b9f\u4e16\u754c\u5b89\u5168\u6027\u30c7\u30fc\u30bf\u306e\u4e0d\u8db3\u3001\u9020\u8840\u5e79\u7d30\u80de\u79fb\u690d (HSCT) \u306e\u5229\u7528\u3092\u5236\u9650\u3059\u308b\u9aa8\u9ac4\u30c9\u30ca\u30fc\u9069\u5408\u306e\u7af6\u4e89\u306a\u3069\u304c\u8ab2\u984c\u3068\u306a\u3063\u3066\u3044\u307e\u3059\u3002<\/p>\n<p>\u5e02\u5834\u898f\u6a21\u3068\u6210\u9577\u4e88\u6e2c<br \/>\n\u03b1-\u30de\u30f3\u30ce\u30b7\u30c9\u30fc\u30b7\u30b9\u6cbb\u7642\u8cbb\u306e\u7dcf\u984d\u306f\u30012025\u5e74\u306e4\u51043,300\u4e07\u30c9\u30eb\u304b\u30892030\u5e74\u306b\u306f9\u51043,800\u4e07\u30c9\u30eb\u3078\u3068\u3001\u5e74\u9593\u5e73\u5747\u6210\u9577\u7387 (CAGR) 16.4%\u3067\u5897\u52a0\u3059\u308b\u3068\u4e88\u6e2c\u3055\u308c\u3066\u3044\u307e\u3059\u3002<\/p>\n<p>\u6cbb\u7642\u30bf\u30a4\u30d7\u5225\u3067\u306f\u3001\u907a\u4f1d\u5b50\u6cbb\u7642\u304c\u6700\u3082\u901f\u3044\u6210\u9577\u3092\u9042\u3052\u308b\u3068\u898b\u8fbc\u307e\u308c\u3066\u304a\u308a\u3001\u5f8c\u671f\u81e8\u5e8a\u8a66\u9a13\u3078\u306e\u9032\u5c55\u306b\u4f34\u3044\u300118.4%\u306eCAGR\u3067\u9175\u7d20\u88dc\u5145\u7642\u6cd5\u3092\u4e0a\u56de\u308b\u6210\u9577\u304c\u4e88\u6e2c\u3055\u308c\u3066\u3044\u307e\u3059\u3002\u9175\u7d20\u88dc\u5145\u7642\u6cd5\u3001\u9020\u8840\u5e79\u7d30\u80de\u79fb\u690d\u3001\u652f\u6301\u7642\u6cd5\u30fb\u88dc\u52a9\u7642\u6cd5\u3001\u6cbb\u9a13\u6bb5\u968e\u306e\u85ac\u7406\u5b66\u7684\u30b7\u30e3\u30da\u30ed\u30f3\u3082\u5206\u6790\u5bfe\u8c61\u3068\u306a\u3063\u3066\u3044\u307e\u3059\u3002<\/p>\n<p>\u6295\u4e0e\u7d4c\u8def\u5225\u3067\u306f\u3001\u9759\u8108\u5185\u6295\u4e0e\u3001\u5916\u79d1\u7684\u79fb\u690d\u6295\u4e0e\u3001\u9ac4\u8154\u5185\/CNS\u76f4\u63a5\u9001\u9054\u3001\u5168\u8eab\u6027\u30a6\u30a4\u30eb\u30b9\u30d9\u30af\u30bf\u30fc\u9001\u9054\u3001\u305d\u306e\u4ed6\u306e\u7d4c\u8def\u304c\u8a55\u4fa1\u3055\u308c\u3066\u3044\u307e\u3059\u3002<\/p>\n<p>\u5730\u57df\u5225\u3067\u306f\u3001\u5317\u7c73\u304c\u6700\u3082\u9ad8\u3044\u6210\u9577\u7387\u3092\u793a\u3059\u3068\u4e88\u60f3\u3055\u308c\u3066\u304a\u308a\u3001Velmanase 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