{"id":55039,"date":"2018-09-27T01:00:00","date_gmt":"2018-09-30T02:01:07","guid":{"rendered":"http:\/\/www.05921.net\/index.php\/2018\/09\/30\/%e9%80%9f%e9%80%92-%e5%9f%ba%e5%9b%a0%e7%96%97%e6%b3%95%e8%bf%9e%e8%8e%b7%e5%96%9c%e8%ae%af%ef%bc%8cfda%e5%8f%96%e6%b6%88%e4%b8%a4%e5%ae%b6%e5%85%ac%e5%8f%b8%e4%b8%b4%e5%ba%8a%e8%af%95%e9%aa%8c\/"},"modified":"2018-09-30T10:01:08","modified_gmt":"2018-09-30T02:01:08","slug":"%e9%80%9f%e9%80%92-%e5%9f%ba%e5%9b%a0%e7%96%97%e6%b3%95%e8%bf%9e%e8%8e%b7%e5%96%9c%e8%ae%af%ef%bc%8cfda%e5%8f%96%e6%b6%88%e4%b8%a4%e5%ae%b6%e5%85%ac%e5%8f%b8%e4%b8%b4%e5%ba%8a%e8%af%95%e9%aa%8c","status":"publish","type":"post","link":"https:\/\/www.05921.net\/index.php\/55039\/","title":{"rendered":"\u901f\u9012 | \u57fa\u56e0\u7597\u6cd5\u8fde\u83b7\u559c\u8baf\uff0cFDA\u53d6\u6d88\u4e24\u5bb6\u516c\u53f8\u4e34\u5e8a\u8bd5\u9a8c\u7981\u4ee4"},"content":{"rendered":"
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\u258e\u836f\u660e<\/span><\/strong>\u5eb7\u5fb7\/\u62a5\u9053<\/span><\/strong><\/strong><\/span><\/strong><\/strong><\/span><\/strong><\/strong><\/span><\/p>\n


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\u8fd1\u65e5\uff0cFDA\u5ba3\u5e03\u53d6\u6d88\u5bf9<\/strong><\/span>Sa<\/strong>repta Therapeutics\u548cEpizyme\u516c\u53f8\u7684\u4e34\u5e8a\u8bd5\u9a8c\u7981\u4ee4\uff0c\u8fd9\u4e24\u5bb6\u516c\u53f8\u5f97\u4ee5\u7ee7\u7eed\u8fdb\u884c\u4e34\u5e8a\u8bd5\u9a8c\u3002<\/strong>Sarepta Therapeutics\u516c\u53f8\u662f\u4e00\u5bb6\u81f4\u529b\u4e8e\u5f00\u53d1\u7cbe\u51c6\u57fa\u56e0\u7597\u6cd5\uff0c\u6cbb\u7597\u7f55\u89c1\u795e\u7ecf\u808c\u8089\u75be\u75c5\u7684\u516c\u53f8\u3002\u800cEpizyme\u516c\u53f8\u662f\u4e00\u5bb6\u5f00\u53d1\u521b\u65b0\u8868\u89c2\u9057\u4f20\u5b66\u7597\u6cd5\u7684\u516c\u53f8\u3002<\/span><\/p>\n


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Sarepta\u7684\u6280\u672f\u5e73\u53f0\u662f\u5229\u7528\u5316\u5b66\u5408\u6210\u7684\u4e8c\u6c28\u57fa\u78f7\u9178\u916f\u5417\u5549\u4ee3\u5be1\u805a\u7269\uff08PMOs\uff09\u6765\u5f71\u54cdRNA\u7684\u8f6c\u8bd1\u8fc7\u7a0b\uff0c\u4ece\u800c\u6539\u53d8\u86cb\u767d\u5408\u6210\u3002PMOs\u80fd\u591f\u4e0e\u7279\u5b9aRNA\u5e8f\u5217\u76f8\u7ed3\u5408\uff0c\u4ece\u800c\u5f71\u54cd\u6838\u7cd6\u4f53\u5bf9RNA\u5e8f\u5217\u7684\u8bc6\u522b\u548c\u8f6c\u8bd1\uff0c\u4f8b\u5982\u8ba9\u6838\u7cd6\u4f53\u5728\u8f6c\u8bd1\u8fc7\u7a0b\u4e2d\u8df3\u8fc7\u4e00\u4e2a\u5916\u663e\u5b50\u3002\u5728\u675c\u5174\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u60a3\u8005\uff08DMD\uff09\u4e2d\uff0c\u7531\u4e8e\u57fa\u56e0\u7a81\u53d8\u5bfc\u81f4\u808c\u8089\u7ec6\u80de\u4e0d\u80fd\u6b63\u5e38\u751f\u6210\u6297\u808c\u840e\u7f29\u86cb\u767d\uff08dystrophin\uff09\u3002\u5f88\u591a\u57fa\u56e0\u7a81\u53d8\u5bfc\u81f4\u6297\u808c\u840e\u7f29\u86cb\u767d\u8f6c\u8bd1\u4e2d\u6b62\uff0c\u800cSarepta\u516c\u53f8\u7814\u53d1\u7684PMOs\u80fd\u591f\u8ba9\u6838\u7cd6\u4f53\u8df3\u8fc7\u53d1\u751f\u7a81\u53d8\u7684\u5916\u663e\u5b50\uff0c\u867d\u7136\u4ea7\u751f\u7684\u86cb\u767d\u53ef\u80fd\u529f\u80fd\u6709\u6240\u51cf\u5f31\uff0c\u4f46\u662f\u5b83\u4eec\u53ef\u4ee5\u4e00\u5b9a\u7a0b\u5ea6\u4e0a\u5f25\u8865\u6297\u808c\u840e\u7f29\u86cb\u767d\u7f3a\u5931\u5e26\u6765\u7684\u529f\u80fd\u7f3a\u9677\u3002<\/strong><\/span><\/p>\n


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Sarepta\u7684DMD\u5fae\u578b\u6297\u808c\u840e\u7f29\u86cb\u767d\u57fa\u56e0\u7597\u6cd5\u9879\u76ee\u6536\u5230\u7981\u4ee4\u7684\u539f\u56e0\u662fFDA\u53d1\u73b0\u7b2c\u4e09\u65b9\u63d0\u4f9b\u7ed9Sarepta\u7684\u8d28\u7c92\u4e2d\u6df7\u6742\u4e86\u5c11\u91cf\u7684DNA\u7247\u6bb5\u3002\u4e3a\u6b64\uff0cSarepta\u516c\u53f8\u4e0e\u5168\u56fd\u513f\u7ae5\u533b\u9662\uff08Nationwide Children\u2019s Hospital\uff09\u5408\u4f5c\uff0c\u5411FDA\u9012\u4ea4\u4e86\u884c\u52a8\u8ba1\u5212\uff0c\u5c06\u5bf9\u8d28\u7c92\u4f9b\u5e94\u5546\u8fdb\u884c\u5ba1\u67e5\u5e76\u4e14\u627f\u8bfa\u4f7f\u7528GMP\u6c34\u5e73\u7684\u8d28\u7c92\u751f\u4ea7\u4ee5\u540e\u7684\u4ea7\u54c1\u3002<\/span><\/p>\n


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Epizyme\u516c\u53f8\u7814\u53d1\u7684tazemetostat\u662f\u4e00\u79cd\u201cfirst-in-class\u201dEZH2\u5c0f\u5206\u5b50\u6291\u5236\u5242\u3002<\/strong>EZH2\u901a\u8fc7\u53c2\u4e0e\u7ec4\u86cb\u767d\u7532\u57fa\u5316\u8fc7\u7a0b\u53ef\u4ee5\u4ece\u8868\u89c2\u9057\u4f20\u5b66\u6c34\u5e73\u6291\u5236\u80bf\u7624\u6291\u5236\u57fa\u56e0\u7684\u8868\u8fbe\u3002\u6291\u5236EZH2\u6d3b\u6027\u53ef\u4ee5\u6fc0\u53d1\u80bf\u7624\u6291\u5236\u57fa\u56e0\u7684\u8868\u8fbe\uff0c\u4ece\u800c\u6291\u5236\u80bf\u7624\u7ec6\u80de\u751f\u957f\u3002Tazemetostat\u5728\u524d\u671f\u4e34\u5e8a\u8bd5\u9a8c\u4e2d\u5df2\u7ecf\u5728\u8840\u764c\u548c\u5177\u6709\u7279\u5b9a\u9057\u4f20\u7279\u5f81\u7684\u5b9e\u4f53\u7624\u60a3\u8005\u4e2d\u8868\u73b0\u51fa\u4e34\u5e8a\u6d3b\u6027\u3002<\/span><\/p>\n


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FDA\u5bf9tazemetostat\u7684\u4e34\u5e8a\u8bd5\u9a8c\u7981\u4ee4\u662f\u57fa\u4e8e\u5bf9\u5b83\u53ef\u80fd\u5f15\u53d1\u6b21\u7ea7\u80bf\u7624\u7684\u62c5\u5fe7\uff0c\u5176\u4e2d\u5305\u62ecT\u7ec6\u80de\u6dcb\u5df4\u6bcd\u7ec6\u80de\u6dcb\u5df4\u7624\uff08T-LBL\uff09\u3002\u5728Epizyme\u8fdb\u884c\u7684\u4e34\u5e8a\u8bd5\u9a8c\u4e2d\uff0c\u66fe\u6709\u4e00\u4f8b\u513f\u7ae5\u60a3\u8005\u51fa\u73b0\u4e86T-LBL\u3002\u4e3a\u6b64\uff0cEpizyme\u4f9d\u636e\u5df2\u53d1\u8868\u7684\u79d1\u5b66\u6587\u732e\u548c\u8be5\u516c\u53f8\u8fc4\u4eca\u4e3a\u6b62\u7684\u4e34\u5e8a\u8bd5\u9a8c\u6570\u636e\uff0c\u5411FDA\u9012\u4ea4\u4e86\u4e0etazemetostat\u53ef\u80fd\u76f8\u5173\u7684\u6b21\u7ea7\u80bf\u7624\u98ce\u9669\u7684\u5168\u9762\u7814\u7a76\u548c\u5206\u6790\u3002\u5176\u4e2d\u5305\u62ec\u8be5\u516c\u53f8\u6240\u6709\u8840\u764c\u548c\u5b9e\u4f53\u7624\u4e34\u5e8a\u8bd5\u9a8c\u7684\u7597\u6548\u548c\u5b89\u5168\u6027\u5206\u6790\uff0c\u4ee5\u53ca\u5916\u90e8\u79d1\u5b66\u548c\u533b\u5b66\u4e13\u5bb6\u5bf9\u8fd9\u4e9b\u7ed3\u679c\u7684\u5ba1\u6838\u3002<\/span><\/p>\n


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\u5728FDA\u7981\u4ee4\u89e3\u9664\u540e\uff0cSarepta\u516c\u53f8\u9884\u8ba1\u5c06\u57282018\u5e74\u5e95\u542f\u52a8\u4f7f\u7528\u5fae\u6297\u808c\u840e\u7f29\u86cb\u767d\u57fa\u56e0\u7597\u6cd5\u6cbb\u7597DMD\u60a3\u8005\u7684\u4e34\u5e8a\u8bd5\u9a8c\u3002Epizyme\u516c\u53f8\u5c06\u91cd\u65b0\u5f00\u542f\u4e0etazemetostat\u76f8\u5173\u7684\u591a\u9879\u4e34\u5e8a\u8bd5\u9a8c\uff0c\u5176\u4e2d\u5305\u62ec\u6cbb\u7597\u643a\u5e26EZH2\u6fc0\u6d3b\u6027\u7a81\u53d8\u7684\u6ee4\u6ce1\u6027\u6dcb\u5df4\u7624\u60a3\u8005\u76842\u671f\u4e34\u5e8a\u8bd5\u9a8c\u3002<\/span><\/p>\n


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\u53c2\u8003\u8d44\u6599\uff1a<\/span><\/p>\n

[1] Epizyme Announces the U.S. Food and Drug Administration Lifts Partial Clinical Hold on Tazemetostat Clinical Program. Retrieved September 24, 2018, https:\/\/epizyme.gcs-web.com\/news-releases\/news-release-details\/epizyme-announces-us-food-and-drug-administration-lifts-partial <\/span><\/p>\n

[2] Sarepta Announces Clinical Hold Lifted for its Duchenne Muscular Dystrophy Micro-dystrophin Gene Therapy Program. Retrieved September 24, 2018, from http:\/\/investorrelations.sarepta.com\/news-releases\/news-release-details\/sarepta-announces-clinical-hold-lifted-its-duchenne-muscular<\/span><\/p>\n


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\u672c\u6587\u6765\u81ea\u836f\u660e\u5eb7\u5fb7\u5fae\u4fe1\u56e2\u961f\uff0c\u6b22\u8fce\u8f6c\u53d1\u5230\u670b\u53cb\u5708\uff0c\u8c22\u7edd\u8f6c\u8f7d\u5230\u5176\u4ed6\u5e73\u53f0\uff1b\u5982\u6709\u5f00\u8bbe\u767d\u540d\u5355\u9700\u6c42\uff0c\u8bf7\u5728\u6587\u7ae0\u5e95\u90e8\u7559\u8a00\uff1b\u5982\u6709\u5176\u4ed6\u5408\u4f5c\u9700\u6c42\uff0c\u8bf7\u8054\u7cfbwuxi_media@wuxiapptec.com<\/span>
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