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\"\"<\/p>\n

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\"\"<\/p>\n

\u4e0d\u53ca\u9884\u671f\u7684\u5341\u5b57\u8def\u53e3\uff0c\u81f3\u6697\u8fd8\u662f\u9ece\u660e\u524d\uff1f<\/strong><\/p>\n

\u76f8\u8f83\u4e8e\u7814\u53d1\u7aef\u7684\u8d25\u8d70\u9ea6\u57ce\u4e0e\u6495\u4e0d\u5f00\u5546\u4e1a\u5316\u53e3\u5b50\u7684\u56f0\u5c40\uff0c\u5f62\u6210\u9c9c\u660e\u53cd\u5dee\u7684\u662f\u8fd9\u4e00\u9886\u57df\u4f9d\u7136\u6709\u70ed\u94b1\u4e0d\u65ad\u6d8c\u5165\u3002<\/p>\n

\u5f53\u7136\u8fd9\u4e5f\u79bb\u4e0d\u5f00\u8fc7\u53bb\u51e0\u5e74AAV \u57fa\u56e0\u7597\u6cd5\u7684\u7684\u786e\u786e\u53d6\u5f97\u4e86\u4e00\u7cfb\u5217\u4ee4\u4eba\u77a9\u76ee\u7684\u6210\u679c\u3002\u81ea20\u4e16\u7eaa60\u5e74\u4ee3\u9996\u6b21\u88ab\u53d1\u73b0\u4ee5\u6765\uff0cAAV\u4ece\u5b9e\u9a8c\u5ba4\u7684\u201c\u6f5c\u529b\u80a1\u201d\u6210\u4e3a\u57fa\u56e0\u6cbb\u7597\u9886\u57df\u8fdb\u5c55\u6700\u5feb\u7684\u7597\u6cd5\u4e4b\u4e00\uff0c\u591a\u4e2a\u4ea7\u54c1\u6210\u529f\u83b7\u6279\u4e0a\u5e02\uff0c\u6301\u7eed\u5728\u5e02\u573a\u4e0a\u6380\u8d77\u6295\u8d44\u70ed\u6f6e\u3002<\/p>\n

2012\u5e7411\u6708\uff0c\u7531UniQure\u516c\u53f8\u5f00\u53d1\u7684\u7528\u4e8e\u6cbb\u7597\u8102\u86cb\u767d\u8102\u80aa\u9176\u7f3a\u4e4f\u75c7 (LPLD) \u7684Glybera (alipogene tiparvovec) \u5728\u6b27\u76df\u83b7\u6279\uff0c\u6210\u4e3a\u9996\u4e2a\u6b63\u5f0f\u4e0a\u5e02\u7684AAV\u57fa\u56e0\u7597\u6cd5\u836f\u7269\uff0c\u4e5f\u5f00\u542f\u4e86\u57fa\u56e0\u7597\u6cd5\u7684\u65b0\u65f6\u4ee3\u3002<\/p>\n

\u622a\u81f3\u76ee\u524d\uff0c\u5168\u7403\u5df2\u67099\u6b3eAAV\u57fa\u56e0\u7597\u6cd5\u6b63\u5f0f\u83b7\u6279\uff0c\u7ed9\u65e0\u836f\u53ef\u7528\u7684\u7f55\u89c1\u75c5\u60a3\u8005\u5e26\u6765\u751f\u7684\u5e0c\u671b\u3002<\/p>\n

\u7b97\u5f97\u4e0a\u8fc4\u4eca\u4e3a\u6b62\u5546\u4e1a\u5316\u6700\u4e3a\u6210\u529f\u7684\u57fa\u56e0\u6cbb\u7597\u836f\u7269\uff0c\u8bfa\u534e\u7684Zolgensma\u8bde\u751f\u4e8e2019\u5e74\uff0c\u7528\u4e8e\u6cbb\u75972\u5c81\u4ee5\u4e0b\u7684\u5c0f\u513f\u5d74\u9ad3\u6027\u808c\u840e\u7f29\u75c7\uff08SMA\uff09\uff0c\u5176\u91c7\u7528 AAV9\u8f7d\u4f53\u9012\u9001SMN1\u57fa\u56e0\uff0c\u901a\u8fc7\u5355\u6b21\u9759\u8109\u6ce8\u5c04\u5c06\u529f\u80fd\u6027SMN1\u57fa\u56e0\u5bfc\u5165\u8fd0\u52a8\u795e\u7ecf\u5143\uff0c\u5b9e\u73b0\u957f\u671fSMN\u86cb\u767d\u8868\u8fbe\u3002\u81ea\u83b7\u6279\u4ee5\u6765\u9500\u552e\u989d\u9010\u5e74\u589e\u957f\uff0c\u57282021\u5e74\u5b9e\u73b0\u4e86\u7ea613.51\u4ebf\u7f8e\u5143\u7684\u9500\u552e\u989d\uff0c\u5e76\u5728\u968f\u540e\u51e0\u5e74\u5747\u4fdd\u6301\u8d8510\u4ebf\u7f8e\u5143\u7684\u5e74\u9500\u552e\u989d\uff0c\u6210\u4e3a\u9996\u6b3e\u8be5\u7c7b\u522b\u7684\u91cd\u78c5\u836f\u7269\u3002<\/p>\n

\u4e2d\u56fd\u516c\u53f8\u4e5f\u4e0d\u7518\u843d\u540e\u30022025\u5e744\u6708\uff0c\u4e2d\u56fd\u4fe1\u5ff5\u533b\u836f\u7684\u6ce2\u54cc\u8fbe\u53ef\u57fa\u6ce8\u5c04\u6db2\uff08\u5546\u54c1\u540d\uff1a\u4fe1\u7396\u51dd\uff0c\u7814\u53d1\u4ee3\u53f7\uff1aBBM-H901\uff09\u83b7\u6279\u4e0a\u5e02\uff0c\u7528\u4e8e\u6cbb\u7597\u8840\u53cb\u75c5 B\uff08\u5148\u5929\u6027\u51dd\u8840\u56e0\u5b50 \u2168 \u7f3a\u4e4f\u75c7\uff09\u6210\u5e74\u60a3\u8005\u3002\u8fd9\u662f\u56fd\u5185\u9996\u4e2a\u83b7\u6279\u4e0a\u5e02\u7684AAV\u57fa\u56e0\u6cbb\u7597\u836f\u7269\uff0c\u901a\u8fc7\u9759\u8109\u7ed9\u836f\u5c06\u4eba\u51dd\u8840\u56e0\u5b50IX\u57fa\u56e0\u5bfc\u5165\u8840\u53cb\u75c5B\u60a3\u8005\u4f53\u5185\u6301\u7eed\u8868\u8fbe\uff0c\u4ece\u800c\u63d0\u9ad8\u5e76\u957f\u671f\u7ef4\u6301\u60a3\u8005\u4f53\u5185\u51dd\u8840\u56e0\u5b50\u6c34\u5e73\uff0c\u4ee5\u671f\u8fbe\u5230 \u201c\u4e00\u6b21\u7ed9\u836f\u3001\u957f\u671f\u6709\u6548\u201d \u7684\u6cbb\u7597\u53ca\u9884\u9632\u51fa\u8840\u6548\u679c\u3002<\/p>\n

\u636e\u7edf\u8ba1\uff0c\u8fd1\u5e74\u6765\u5168\u7403 AAV \u57fa\u56e0\u7597\u6cd5\u9886\u57df\u7684\u6295\u8d44\u6301\u7eed\u589e\u957f\uff0c\u4f17\u591a\u836f\u4f01\u7eb7\u7eb7\u5e03\u5c40\uff0c\u6295\u5165\u5927\u91cf\u8d44\u91d1\u5f00\u5c55\u76f8\u5173\u7814\u53d1\u9879\u76ee\u3002\u4e00\u4e9b\u4e13\u6ce8\u4e8eAAV\u57fa\u56e0\u7597\u6cd5\u7684\u751f\u7269\u6280\u672f\u516c\u53f8\u5982Spark Therapeutics\u3001Regenxbio\u3001uniQure\u7b49\u5728\u8d44\u672c\u5e02\u573a\u5907\u53d7\u9752\u7750\uff0c\u80a1\u4ef7\u4e00\u8def\u6500\u5347\uff0c\u4e0d\u65ad\u83b7\u5f97\u9ad8\u989d\u878d\u8d44\u7528\u4e8e\u7814\u53d1\u7ba1\u7ebf\u7684\u62d3\u5c55\u548c\u4e34\u5e8a\u8bd5\u9a8c\u7684\u63a8\u8fdb\u3002\u5404\u5927\u5236\u836f\u5de8\u5934\u4e5f\u7eb7\u7eb7\u901a\u8fc7\u5408\u4f5c\u3001\u5e76\u8d2d\u7b49\u65b9\u5f0f\u6d89\u8db3AAV\u57fa\u56e0\u7597\u6cd5\u9886\u57df\uff0c\u8fd9\u4e9b\u4e3e\u63aa\u8fdb\u4e00\u6b65\u63a8\u52a8\u4e86AAV\u57fa\u56e0\u7597\u6cd5\u5e02\u573a\u7684\u53d1\u5c55\uff0c\u4f7f\u5176\u6210\u4e3a\u751f\u7269\u533b\u836f\u9886\u57df\u6700\u70ed\u95e8\u7684\u8d5b\u9053\u4e4b\u4e00\uff0c\u4e00\u65f6\u95f4\u98ce\u5149\u65e0\u9650\u3002<\/p>\n

\u867d\u7136\u4e0d\u80fd\u7b80\u5355\u7684\u7528\u201c\u7978\u516e\u798f\u4e4b\u6240\u501a\uff0c\u798f\u516e\u7978\u4e4b\u6240\u4f0f\u201d\u6765\u5f62\u5bb9\uff0c\u4f46AAV\u57fa\u56e0\u7597\u6cd5\u7684\u706b\u70ed\uff0c\u4e5f\u6210\u4e86\u5b55\u80b2\u201c\u5371\u673a\u201d\u7684\u6e29\u5e8a\u3002\u201c\u70ed\u94b1\u201d\u6d8c\u5165\u5e26\u6765\u7ba1\u7ebf\u4e95\u55b7\uff0c\u53ef\u968f\u7740\u7814\u53d1\u8fdb\u7a0b\u7684\u63a8\u8fdb\u4e43\u81f3\u5546\u4e1a\u5316\u65f6\uff0c\u8d8a\u6765\u8d8a\u591a\u53ef\u80fd\u201c\u4eba\u8d22\u4e24\u5931\u201d\u7684\u95ee\u9898\u4e5f\u66b4\u9732\u51fa\u6765\u3002<\/p>\n

\u4eca\u5e743\u6708\uff0cSarepta Therapeutics\u516c\u53f8\u7528\u4e8e\u6cbb\u7597\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\uff08DMD\uff09\u7684\u57fa\u56e0\u7597\u6cd5Elevidys\u51fa\u73b0\u9996\u4f8b\u6cbb\u7597\u76f8\u5173\u6b7b\u4ea1\u75c5\u4f8b\uff0c\u8be5\u60a3\u8005\u5728\u63a5\u53d7Elevidys\u6cbb\u7597\u540e\u56e0\u6025\u6027\u809d\u8870\u7aed\uff08ALF\uff09\u6b7b\u4ea1\u3002Sarepta\u80a1\u4ef7\u4e5f\u5e94\u58f0\u66b4\u8dcc27%\u3002<\/p>\n

Elevidys\u662f\u4e00\u6b3e\u57fa\u4e8e\u817a\u76f8\u5173\u75c5\u6bd2\u8f7d\u4f53\uff08AAVrh74\uff09\u7684\u91cd\u7ec4\u57fa\u56e0\u7597\u6cd5\uff0c\u7531Sarepta\u548c\u7f57\u6c0f\u8054\u5408\u5f00\u53d1\uff0c\u4e8e2023\u5e746\u6708\u83b7FDA\u6279\u51c6\u4e0a\u5e02\uff0c\u6210\u4e3a\u5168\u7403\u9996\u6b3e\u9488\u5bf9DMD\u7684\u57fa\u56e0\u7597\u6cd5\uff0c\u4ef7\u683c\u4e0e\u5176\u4ed6\u57fa\u56e0\u7597\u6cd5\u7c7b\u4f3c\uff0c\u90fd\u662f\u767e\u4e07\u7f8e\u5143\u7684\u5929\u4ef7\u3002<\/p>\n

\u6570\u636e\u663e\u793a\uff0c\u5728\u9ad8\u5242\u91cfAAV (\u8d85\u8fc72\u00d710\u2074vg\/kg) \u7ed9\u836f\u4e0b\uff0cElevidys\u6709\u53ef\u80fd\u5f15\u53d1\u80c6\u6c41\u6de4\u79ef\u6027\u809d\u708e\uff0c\u8fd9\u4e0e2020\u5e74\u5b89\u65af\u6cf0\u6765AT132\u6cbb\u7597X\u8fde\u9501\u808c\u7ba1\u808c\u75c5 (XLMTM) \u4e34\u5e8a\u8bd5\u9a8c\u4e2d\u51fa\u73b0\u76843\u4f8b\u6b7b\u4ea1\u4e8b\u4ef6\u6240\u63ed\u793a\u7684\u6570\u636e\u76f8\u7b26\u3002\u6b64\u59162022\u5e74\uff0c\u8bfa\u534e\u4e5f\u62a5\u544a\u4e86\u4e24\u540d\u60a3\u513f\u5728\u63a5\u53d7\u7528\u4e8e\u6cbb\u7597\u5d74\u9ad3\u6027\u808c\u840e\u7f29\u75c7\u7684Zolgensma\u6cbb\u7597\u540e\uff0c\u56e0\u6025\u6027\u809d\u529f\u80fd\u8870\u7aed\u800c\u53d1\u751f\u7684\u6b7b\u4ea1\u3002\u60a3\u8005\u6b7b\u4ea1\u4e8b\u4ef6\u518d\u6b21\u628a\u57fa\u56e0\u7597\u6cd5\u7684\u5b89\u5168\u6027\u95ee\u9898\u63a8\u4e0a\u4e86\u98ce\u53e3\u6d6a\u5c16\u3002<\/p>\n

\u6446\u5728AAV\u57fa\u56e0\u7597\u6cd5\uff0c\u6216\u8005\u8bf4\u6574\u4e2a\u57fa\u56e0\u6cbb\u7597\u9762\u524d\u7684\u53e6\u4e00\u4e2a\u9e3f\u6c9f\u5219\u662f\u65e0\u4eba\u4e70\u5355\u3002\u5c31\u50cf\u4ea7\u54c1\u83b7\u6279\u4e5f\u6482\u6311\u5b50\u4e0d\u5e72\u4e86\u7684\u5b87\u5b99\u5927\u836f\u5382\u8f89\u745e\u73b0\u8eab\u8bf4\u6cd5\u7684\u2014\u2014\u592a\u8d35\u4e86\uff0c\u533b\u751f\u548c\u60a3\u8005\u90fd\u4e0d\u611f\u5174\u8da3\u3002<\/p>\n

\u66f4\u662f\u6709\u57fa\u56e0\u7597\u6cd5\u9886\u57df\u5148\u9a71\u84dd\u9e1f\u751f\u7269\u8fd9\u79cd\uff0c\u8270\u96be\u652f\u6491\u5230\u5546\u4e1a\u5316\uff0c\u8fd8\u662f\u5148\u9a71\u53d8\u5148\u70c8\u3002\u4eca\u5e742\u6708\u84dd\u9e1f\u751f\u7269\u5ba3\u5e03\u88ab\u5168\u7403\u6295\u8d44\u516c\u53f8\u51ef\u96f7\uff08Carlyle\uff09\u548cSK Capital\u6536\u8d2d\uff0c\u4f30\u503c\u4ec5\u4e3a2900\u4e07\u7f8e\u5143\uff0c\u8981\u77e5\u9053\u84dd\u9e1f\u6614\u65e5\u5e02\u503c\u66fe\u9ad8\u8fbe\u767e\u4ebf\u7f8e\u5143\u3002<\/p>\n

\u8ba9\u84dd\u9e1f\u751f\u7269\u52a0\u901f\u201c\u6b7b\u4ea1\u201d\u7684\uff0c\u6b63\u662f\u4ea7\u54c1\u4e0a\u5e02\u540e\u4e0d\u4f73\u7684\u8868\u73b0\u3002\u84dd\u9e1f\u751f\u7269\u7684\u4e09\u6b3e\u57fa\u56e0\u6cbb\u7597\u4ea7\u54c1\uff0c\u5206\u522b\u9488\u5bf9\u5730\u4e2d\u6d77\u8d2b\u8840\u3001\u80be\u4e0a\u817a\u8111\u767d\u8d28\u8425\u517b\u4e0d\u826f\u548c\u9570\u72b6\u7ec6\u80de\u75c5\u3002\u4f46\u4ea7\u54c1\u65e0\u6cd5\u76c8\u5229\u6210\u4e86\u538b\u57ae\u84dd\u9e1f\u751f\u7269\u7684\u6700\u540e\u4e00\u6839\u7a3b\u8349\u3002<\/p>\n

\u6709\u5206\u6790\u6307\u51fa\uff0c\u5b89\u5168\u6027\u98ce\u9669\uff0c\u53e0\u52a0\u6bcf\u5242200\u4e07-300\u4e07\u7f8e\u5143\u7684\u5929\u4ef7\uff0c\u8ba9\u57fa\u56e0\u7597\u6cd5\u7684\u98ce\u9669\u6536\u76ca\u5e73\u8861\u6b63\u5728\u53d1\u751f\u6839\u672c\u6027\u6539\u53d8\uff0c\u4e0d\u7ba1\u662f\u4e34\u5e8a\u7814\u7a76\u7684\u6311\u6218\u4e0e\u5546\u4e1a\u5316\u56f0\u5c40\uff0c\u90fd\u6210\u4e86\u5176\u524d\u8fdb\u7684\u7eca\u811a\u77f3\u3002<\/p>\n

<\/p>\n","protected":false},"excerpt":{"rendered":"

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