{"id":8437,"date":"2018-06-12T01:00:00","date_gmt":"2018-07-27T09:32:21","guid":{"rendered":"http:\/\/www.05921.net\/index.php\/2018\/07\/27\/2018%e5%b9%b4%e5%85%a8%e7%90%83%e5%9f%ba%e5%9b%a0%e6%b2%bb%e7%96%97%e9%a2%86%e5%9f%9f%e8%bf%9b%e5%b1%95%ef%bc%88bd%e4%b8%8b%e7%af%87%ef%bc%89\/"},"modified":"2018-07-27T17:32:22","modified_gmt":"2018-07-27T09:32:22","slug":"2018%e5%b9%b4%e5%85%a8%e7%90%83%e5%9f%ba%e5%9b%a0%e6%b2%bb%e7%96%97%e9%a2%86%e5%9f%9f%e8%bf%9b%e5%b1%95%ef%bc%88bd%e4%b8%8b%e7%af%87%ef%bc%89","status":"publish","type":"post","link":"https:\/\/www.05921.net\/index.php\/8437\/","title":{"rendered":"2018\u5e74\u5168\u7403\u57fa\u56e0\u6cbb\u7597\u9886\u57df\u8fdb\u5c55\uff08BD\u4e0b\u7bc7\uff09"},"content":{"rendered":"<div class=\"bpp-post-content\">\n<p style=\"text-align: justify;\"><span style=\"font-size: 15px;\"><\/span><\/p>\n<p style=\"max-width: 100%;min-height: 1em;\"><span style=\"max-width: 100%;font-size: 15px;box-sizing: border-box !important;word-wrap: break-word !important;\"><strong style=\"max-width: 100%;box-sizing: border-box !important;word-wrap: break-word !important;\"><span style=\"max-width: 100%;color: rgb(0, 82, 255);box-sizing: border-box !important;word-wrap: break-word !important;\">\u4f5c\u8005 | \u5403\u74dc\u7fa4\u4f17<\/span><\/strong><\/span><\/p>\n<p style=\"max-width: 100%;min-height: 1em;\"><span style=\"max-width: 100%;font-size: 15px;box-sizing: border-box !important;word-wrap: break-word !important;\"><strong style=\"max-width: 100%;box-sizing: border-box !important;word-wrap: break-word !important;\"><span style=\"max-width: 100%;color: rgb(0, 82, 255);box-sizing: border-box !important;word-wrap: break-word !important;\">\u7f16\u8f91 | \u7ec6\u80de\u623f\u95f4<\/span><\/strong><\/span><\/p>\n<p style=\"text-align: justify;\"><span style=\"font-size: 15px;\"><br  \/><\/span><\/p>\n<p style=\"text-align: justify;\"><span style=\"font-size: 15px;\">\u5728\u4e0a\u4e00\u7bc7\u4e2d<\/span><span style=\"font-size: 15px;\">\uff08<\/span><a href=\"https:\/\/mp.weixin.qq.com\/s?__biz=MzAxMDc4OTIxMw==&amp;mid=2663359258&amp;idx=1&amp;sn=68da70e5b9068ad07fb4ecd824d9924b&amp;scene=21#wechat_redirect\" target=\"_blank\"><span style=\"font-size: 15px;\">2018\u5e74\u5168\u7403\u57fa\u56e0\u6cbb\u7597\u9886\u57df\u8fdb\u5c55\u2014BD\u4e0a\u7bc7<\/span><\/a><span style=\"font-size: 16px;\">\uff09<\/span><span style=\"font-size: 15px;\">\uff0c\u5c0f\u7f16\u56de\u987e\u4e862018\u5e74\u4ee5\u6765\u5168\u7403\u57fa\u56e0\u6cbb\u7597\u9886\u57df\u7684\u6536\u8d2d\u3001\u878d\u8d44\u53ca\u653f\u5e9c\u8d44\u52a9\u6848\u4f8b\u3002\u63a5\u4e0b\u6765\uff0c\u5c0f\u7f16\u5c06\u8fdb\u4e00\u6b65\u5e26\u9886\u5927\u5bb6\u4e86\u89e3\u8fd9\u6bb5\u65f6\u95f4\u4ee5\u6765\uff0c\u8be5\u9886\u57df\u7684\u5408\u4f5c\u5f00\u53d1\u6848\u4f8b\u3002<\/span><\/p>\n<p style=\"text-align: justify;\"><span style=\"font-size: 15px;\"><\/span><br  \/><\/p>\n<p style=\"text-align: justify;\"><span style=\"font-size: 15px;\">\u5728\u5c0f\u7f16\u770b\u6765\uff0c\u533b\u836f\u9886\u57df\u7684BD\u5408\u4f5c\u901a\u5e38\u6709<strong>\u4e09\u79cd\u6a21\u5f0f<\/strong>\uff0c<strong>\u7b2c\u4e00\u79cd\u662f\u4e59\u65b9\u51fa\u94b1\u8d44\u52a9\u7532\u65b9\u7814\u53d1<\/strong>\uff0c\u4e59\u65b9\u5bf9\u7532\u65b9\u7684\u7814\u53d1\u6210\u679c\u6709\u4f18\u5148\u83b7\u5f97\u6743\uff0c\u8fd9\u79cd\u6a21\u5f0f\u6700\u4f20\u7edf\u3002<strong>\u7b2c\u4e8c\u79cd\u662f\u7532\u4e59\u53cc\u65b9\u5f3a\u5f3a\u8054\u5408<\/strong>\uff0c\u53d1\u6325\u5404\u81ea\u6280\u672f\u5e73\u53f0\u7684\u4f18\u52bf\uff0c\u8fd9\u79cd\u6a21\u5f0f\u76ee\u524d\u8d8a\u6765\u8d8a\u666e\u904d\uff0c\u96be\u70b9\u662f\u5982\u4f55\u5f00\u8bda\u5e03\u516c\uff0c\u5b9e\u73b01+1&gt;2\u7684\u5408\u4f5c\u6548\u679c\u3002<strong>\u6700\u540e\u4e00\u79cd\u662f\u4e59\u65b9\u51fa\u94b1\u4e70\u4e0b\u7532\u65b9\u7684\u9879\u76ee<\/strong>\uff0c\u5e76\u7531\u4e59\u65b9\u7ee7\u7eed\u7814\u53d1\u3002\u8fd9\u79cd\u6a21\u5f0f\u4e0d\u592a\u5e38\u89c1\uff0c\u7b97\u662f\u6536\u8d2d\u7684\u6f14\u8fdb\u7248\u672c\u3002\u5c3d\u7ba1\u4ee3\u4ef7\u6bd4\u6536\u8d2d\u8981\u5c0f\u4e00\u4e9b\uff0c\u4f46\u5982\u4f55\u5b9e\u73b0\u53cc\u65b9\u6280\u672f\u56e2\u961f\u7684\u65e0\u7f1d\u5bf9\u63a5\uff0c\u4fdd\u8bc1\u9879\u76ee\u6309\u671f\u5b8c\u6210\uff0c\u96be\u5ea6\u4e0d\u5c0f\u3002\u57fa\u56e0\u6cbb\u7597\u9886\u57df\u7684BD\u5408\u4f5c\u4e5f\u5448\u73b0\u8fd9\u79cd\u5206\u5e03\u7279\u70b9\uff0c\u4e0b\u9762\u5c0f\u7f16\u4e00\u4e00\u7ed9\u5927\u5bb6\u9053\u6765\u3002<\/span><\/p>\n<p style=\"text-align: justify;\"><span style=\"font-size: 15px;\"><br  \/><\/span><\/p>\n<p style=\"text-align: justify;\"><span style=\"color: rgb(0, 209, 0);\"><strong><span style=\"font-size: 15px;\">\u4f20\u7edf\u6a21\u5f0f<\/span><\/strong><\/span><\/p>\n<p style=\"text-align: justify;\"><span style=\"font-size: 15px;\">2018\u5e74\u4ee5\u6765\uff0c\u6b66\u7530\u5236\u836f\u5728BD\u65b9\u9762\u5f88\u6d3b\u8dc3\u30021\u6708\u4efd\uff0c\u6b66\u7530\u65a5\u8d445.2\u4ebf\u6b27\u5143\u6536\u8d2d\u6bd4\u5229\u65f6\u7ec6\u80de\u836f\u7269\u7814\u53d1\u516c\u53f8TiGenix\uff0c\u540e\u8005\u7684Alofisel 2\u4e2a\u6708\u540e\u83b7\u6279\u3002\u8fd1\u65e5\uff0c\u6b66\u7530\u66f4\u662f\u4ee5622\u4ebf\u7f8e\u5143\u7684\u5929\u4ef7\u6536\u8d2dShire\uff0c\u8fd9\u7b14\u4ea4\u6613\u5e94\u8be5\u662f2018\u5e74\u5ea6\u5236\u836f\u9886\u57df\u6700\u5927\u7684\u4ea4\u6613\u3002\u5728\u57fa\u56e0\u6cbb\u7597\u9886\u57df\uff0c2\u6708\u4efd\uff0c\u6b66\u7530\u4e0e\u603b\u90e8\u4f4d\u4e8e\u7f8e\u56fd\u9ebb\u7701\u5251\u6865\u5e02\u7684Wave Life Sciences\u8fbe\u6210\u5408\u4f5c\u534f\u8bae\uff0c\u5c06\u5171\u540c\u5f00\u53d1\u57fa\u4e8eRNA\u7684\u836f\u7269\uff0c\u9488\u5bf9\u591a\u79cd\u795e\u7ecf\u75be\u75c5\u3002\u57fa\u4e8e\u8fd9\u9879\u534f\u8bae\uff0cWave\u5c06\u4ece\u6b66\u7530\u83b7\u5f971.1\u4ebf\u7f8e\u5143\u7684\u73b0\u91d1\uff1b\u6b66\u7530\u8fd8\u5c06\u4ee5\u6bcf\u80a154.70\u7f8e\u5143\u7684\u4ef7\u683c\u8d2d\u4e706000\u4e07\u7f8e\u5143\u7684Wave\u80a1\u7968\uff0c\u5e76\u5728\u672a\u6765\u56db\u5e74\u5185\u4e3aWave\u63d0\u4f9b\u81f3\u5c116000\u4e07\u7f8e\u5143\u7684\u7814\u7a76\u8d44\u91d1\uff1b\u6b66\u7530\u53ef\u4ee5\u5728Wave\u7684\u8111\u90e8\u75be\u75c5\u5728\u7814\u9879\u76ee\u4e2d\u83b7\u5f97\u90e8\u5206\u6743\u5229\u3002Wave\u6253\u52a8\u6b66\u7530\u4e4b\u5904\uff0c\u5728\u4e8e\u5176\u5728RNA\u836f\u7269\u7814\u53d1\u65b9\u9762\u8003\u8651\u5230\u4e86\u4ea7\u54c1\u7684\u624b\u6027\u3002<\/span><\/p>\n<p style=\"text-align: center;\"><img class=\"\"      style=\"\" src=\"http:\/\/www.05921.net\/wp-content\/uploads\/2018\/07\/beepress3-1532683942.jpg\" alt=\"2018\u5e74\u5168\u7403\u57fa\u56e0\u6cbb\u7597\u9886\u57df\u8fdb\u5c55\uff08BD\u4e0b\u7bc7\uff09\" title=\"2018\u5e74\u5168\u7403\u57fa\u56e0\u6cbb\u7597\u9886\u57df\u8fdb\u5c55\uff08BD\u4e0b\u7bc7\uff09\"  \/><\/p>\n<p style=\"text-align: justify;\"><span style=\"font-size: 15px;\"><br  \/><\/span><\/p>\n<p style=\"text-align: justify;\"><span style=\"font-size: 15px;\">1\u6708\u521d\uff0cSangamo Therapeutics\u5ba3\u5e03\u4e0e\u8f89\u745e\u5408\u4f5c\u7814\u53d1\u4e00\u79cd\u4f7f\u7528\u950c\u6307\u86cb\u767d\u8f6c\u5f55\u56e0\u5b50\uff08ZFP-TF\uff09\u7684\u57fa\u56e0\u7597\u6cd5\u6cbb\u7597\u808c\u840e\u7f29\u4fa7\u7d22\u786c\u5316\u75c7\uff08ALS\uff09\u4ee5\u53ca\u4e0eC9ORF72\u57fa\u56e0\u7a81\u53d8\u76f8\u5173\u7684\u989d\u989e\u53f6\u53d8\u6027\uff08FTLD\uff09\u3002<\/span><\/p>\n<p style=\"text-align: justify;\"><span style=\"font-size: 15px;\"><br  \/><\/span><\/p>\n<p style=\"text-align: justify;\"><span style=\"font-size: 15px;\">\u6839\u636e\u5408\u4f5c\u534f\u8bae\uff0c\u8f89\u745e\u5c06\u652f\u4ed8Sangamo\u516c\u53f81200\u4e07\u7f8e\u5143\u7684\u9884\u4ed8\u6b3e\u3002\u53e6\u5916\uff0cSangamo\u8fd8\u6709\u8d44\u683c\u83b7\u5f971.5\u4ebf\u7f8e\u5143\u7684\u7814\u53d1\u548c\u5546\u4e1a\u91cc\u7a0b\u7891\u4ed8\u6b3e\u3002Sangamo\u516c\u53f8\u5c06\u8d1f\u8d23ZFP-TF\u5019\u9009\u4ea7\u54c1\u7684\u7814\u53d1\u3002<\/span><\/p>\n<p style=\"text-align: center;\"><img class=\"\"      style=\"\" src=\"http:\/\/www.05921.net\/wp-content\/uploads\/2018\/07\/beepress2-1532683942.jpg\" alt=\"2018\u5e74\u5168\u7403\u57fa\u56e0\u6cbb\u7597\u9886\u57df\u8fdb\u5c55\uff08BD\u4e0b\u7bc7\uff09\" title=\"2018\u5e74\u5168\u7403\u57fa\u56e0\u6cbb\u7597\u9886\u57df\u8fdb\u5c55\uff08BD\u4e0b\u7bc7\uff09\"  \/><\/p>\n<p style=\"text-align: justify;\"><span style=\"font-size: 15px;\"><br  \/><\/span><\/p>\n<p style=\"text-align: justify;\"><span style=\"font-size: 15px;\">\u76f8\u6bd4\u524d\u4e24\u9879\u5408\u4f5c\uff0c\u7b2c3\u4e2a\u5408\u4f5c\u7684\u89c4\u6a21\u8981\u5c0f\u4e00\u4e9b\u30022\u6708\u4efd\uff0cOxford BioMedica\uff08OXB\uff09\u5ba3\u5e03\u4e0eBioverativ\u516c\u53f8\u8fbe\u6210\u4e00\u9879\u91cd\u8981\u7684\u65b0\u5408\u4f5c\u548c\u8bb8\u53ef\u534f\u8bae\uff0c\u5f00\u53d1\u548c\u751f\u4ea7\u7528\u4e8e\u6cbb\u7597\u8840\u53cb\u75c5\u7684\u6162\u75c5\u6bd2\u8f7d\u4f53\u3002\u8be5\u534f\u8bae\u5305\u62ec\u8bb8\u53efBioverativ\u4f7f\u7528OXB\u4e13\u6709\u7684LentiVector Enabled\u6280\u672f\u548c\u5176\u5de5\u4e1a\u89c4\u6a21\u7684\u5236\u9020\u6280\u672f\u3002\u6839\u636e\u534f\u8bae\u6761\u6b3e\uff0cOXB\u516c\u53f8\u5c06\u4eceBioverativ\u83b7\u5f97500\u4e07\u7f8e\u5143\u7684\u9884\u4ed8\u6b3e\uff0c\u4ee5\u53ca1\u4ebf\u7f8e\u5143\u7684\u591a\u9879\u91cc\u7a0b\u7891\u4ed8\u6b3e\u3002Bioverativ\u8fd8\u5c06\u8d44\u52a9Oxford BioMedica\u7684\u6162\u75c5\u6bd2\u8f7d\u4f53\u8840\u53cb\u75c5\u4ea7\u54c1\u7684\u5de5\u827a\u5f00\u53d1\u548c\u6269\u5927\u751f\u4ea7\u3002\u8be5\u534f\u8bae\u8fd8\u5141\u8bb8\u53cc\u65b9\u5728OXB\u516c\u53f8\u751f\u4ea7GMP\u6807\u51c6\u7684\u8840\u53cb\u75c5\u4ea7\u54c1\u8fbe\u6210\u4e34\u5e8a\u4f9b\u5e94\u534f\u8bae\u3002<\/span><\/p>\n<p style=\"text-align: center;\"><img class=\"\"      style=\"\" src=\"http:\/\/www.05921.net\/wp-content\/uploads\/2018\/07\/beepress2-1532683942-1.jpg\" alt=\"2018\u5e74\u5168\u7403\u57fa\u56e0\u6cbb\u7597\u9886\u57df\u8fdb\u5c55\uff08BD\u4e0b\u7bc7\uff09\" title=\"2018\u5e74\u5168\u7403\u57fa\u56e0\u6cbb\u7597\u9886\u57df\u8fdb\u5c55\uff08BD\u4e0b\u7bc7\uff09\"  \/><\/p>\n<p style=\"text-align: justify;\"><span style=\"font-size: 15px;\"><br  \/><\/span><\/p>\n<p style=\"text-align: justify;\"><span style=\"font-size: 15px;\">\u6700\u540e\u4e00\u9879\u4ea4\u6613\u6709\u70b9\u53e6\u7c7b\u30023\u6708\u4efd\uff0cIonis Pharmaceuticals\u4e0e\u5176\u5b50\u516c\u53f8Akcea Therapeutics\u5ba3\u5e03\uff0cIonis\u5168\u7403\u72ec\u5bb6\u6388\u6743\u8bb8\u53efAkcea\u8fdb\u884cInotersen\u548cAKCEA-TTR-LRx\uff08\u524dIONIS-TTR-LRx\uff09\u4e24\u9879\u836f\u7269\u7684\u5f00\u53d1\u548c\u63a8\u5e7f\uff0c\u8be5\u534f\u8bae\u603b\u503c\u8fbe17\u4ebf\u7f8e\u5143\u3002\u65b0\u7ec4\u5408\u7684Akcea\u56e2\u961f\u6b63\u51c6\u5907\u5728\u7f8e\u56fd\u548c\u6b27\u76df\u4e0a\u5e02Inotersen\uff0c\u8be5\u836f\u7269\u9884\u8ba1\u4e8e2018\u5e74\u4e2d\u6279\u51c6\uff0c\u7528\u4ee5\u6cbb\u7597\u9057\u4f20\u6027\u7532\u72b6\u817a\u7d20\u8fd0\u8f7d\u86cb\u767d\u6dc0\u7c89\u6837\u53d8\u6027\uff08hATTR\uff09\u3002\u6839\u636e\u534f\u8bae\uff0cIonis\u5c06\u6536\u5230Akcea\u76841.5\u4ebf\u7f8e\u5143\u9884\u4ed8\u6b3e\uff1b\u7f8e\u56fd\u548c\u6b27\u76df\u76d1\u7ba1\u673a\u6784\u7684\u6279\u51c6\u5c06\u63a8\u52a8Ionis\u6536\u52305000\u4e07\u7f8e\u5143\u548c4000\u4e07\u7f8e\u5143\u7684\u91cc\u7a0b\u7891\u4ed8\u6b3e\u3002Akcea\u53ef\u4ee5\u9009\u62e9\u4f7f\u7528\u80a1\u7968\u800c\u4e0d\u662f\u73b0\u91d1\u652f\u4ed8\u9884\u4ed8\u6b3e\u548c\u76f8\u5173\u8d39\u7528\u3002\u6b64\u5916\uff0c\u8be5\u534f\u8bae\u8fd8\u6709\u53e6\u591613\u4ebf\u7f8e\u5143\u7684\u91cc\u7a0b\u7891\u6b3e\u9879\u3002<\/span><\/p>\n<p style=\"text-align: center;\"><img class=\"\"      style=\"\" src=\"http:\/\/www.05921.net\/wp-content\/uploads\/2018\/07\/beepress5-1532683942.jpg\" alt=\"2018\u5e74\u5168\u7403\u57fa\u56e0\u6cbb\u7597\u9886\u57df\u8fdb\u5c55\uff08BD\u4e0b\u7bc7\uff09\" title=\"2018\u5e74\u5168\u7403\u57fa\u56e0\u6cbb\u7597\u9886\u57df\u8fdb\u5c55\uff08BD\u4e0b\u7bc7\uff09\"  \/><\/p>\n<p style=\"text-align: justify;\"><span style=\"font-size: 15px;\"><br  \/><\/span><\/p>\n<p style=\"text-align: justify;\"><strong><span style=\"font-size: 15px;color: rgb(0, 209, 0);\">\u5408\u4f5c\u5f00\u53d1<\/span><\/strong><\/p>\n<p style=\"text-align: justify;\"><span style=\"font-size: 15px;\">1\u6708\u5e95\uff0cAdverum Biotechnologies\uff08Adverum\uff09\u5ba3\u5e03\u6269\u5927\u4e0eEditas Medicine\uff08Editas\uff09\u7684\u5408\u4f5c\u534f\u8bae\u3002\u4e24\u5bb6\u516c\u53f8\u66fe\u4e8e2016\u5e748\u6708\u516c\u5e03\u4e86\u5408\u4f5c\u5173\u7cfb\uff0c\u534f\u529b\u5f00\u53d1\u57fa\u56e0\u7ec4\u7f16\u8f91\u7c7b\u836f\u7269\u4ee5\u6cbb\u7597\u4e94\u79cd\u9057\u4f20\u6027\u7684\u89c6\u7f51\u819c\u75be\u75c5\u3002\u6839\u636e\u6269\u5927\u534f\u8bae\u7684\u6761\u6b3e\uff0cAdverum\u548cEditas\u5c06\u7814\u7a76\u671f\u9650\u5ef6\u957f\u81f32018\u5e74\u7b2c\u4e09\u5b63\u5ea6\uff1b\u4ece\u73b0\u5728\u81f32020\u5e748\u6708\uff0cEditas\u4fdd\u7559\u4e86\u4e00\u7cfb\u5217\u53ef\u884c\u6743\u529b\u3002Adverum\u4e0eEditas\u7684\u5408\u4f5c\u5185\u5bb9\u4e3b\u8981\u96c6\u4e2d\u5728\u5229\u7528\u524d\u8005\u7684\u4e0b\u4e00\u4ee3AAV\u8f7d\u4f53\u6765\u4f20\u9012Editas\u57fa\u4e8eCRISPR\u7684\u57fa\u56e0\u7ec4\u7f16\u8f91\u6280\u672f\uff0c\u4ece\u800c\u5f00\u53d1\u65b0\u7684\u6cbb\u7597\u65b9\u6cd5\u3002<\/span><\/p>\n<p style=\"text-align: center;\"><img class=\"\"      style=\"width: 431px;height: 92px;\" src=\"http:\/\/www.05921.net\/wp-content\/uploads\/2018\/07\/beepress10-1532683942.jpg\" alt=\"2018\u5e74\u5168\u7403\u57fa\u56e0\u6cbb\u7597\u9886\u57df\u8fdb\u5c55\uff08BD\u4e0b\u7bc7\uff09\" title=\"2018\u5e74\u5168\u7403\u57fa\u56e0\u6cbb\u7597\u9886\u57df\u8fdb\u5c55\uff08BD\u4e0b\u7bc7\uff09\"  \/><\/p>\n<p style=\"text-align: justify;\"><span style=\"font-size: 15px;\"><br  \/><\/span><\/p>\n<p style=\"text-align: justify;\"><span style=\"font-size: 15px;\">2\u670821\u65e5\uff0c\u827e\u4f2f\u7ef4\uff08AbbVie\uff09\u516c\u53f8\u5ba3\u5e03\u4e0eVoyager Therapeutics\u8fbe\u6210\u6218\u7565\u5408\u4f5c\uff0c\u5c06\u524d\u8005\u7684\u5355\u514b\u9686\u6297\u4f53\u6280\u672f\u5e73\u53f0\u4e0e\u5168\u7403\u4e34\u5e8a\u5f00\u53d1\u80fd\u529b\uff0c\u4ee5\u53ca\u540e\u8005\u7684\u817a\u76f8\u5173\u75c5\u6bd2\uff08AAV\uff09\u8f7d\u4f53\u6280\u672f\u76f8\u7ed3\u5408\uff0c\u5171\u540c\u5f00\u53d1\u76f4\u63a5\u9488\u5bf9Tau\u86cb\u767d\u7684\u6297\u4f53\uff0c\u4ee5\u6cbb\u7597\u963f\u5179\u6d77\u9ed8\u75c5\u7b49\u795e\u7ecf\u9000\u884c\u6027\u75be\u75c5\u3002\u827e\u4f2f\u7ef4\u4e0eVoyager\u671f\u671b\u80fd\u4f7f\u7528\u76ee\u524d\u65b0\u5174\u7684\u57fa\u56e0\u7597\u6cd5\uff0c\u5229\u7528AAV\u9012\u9001\u6280\u672f\uff0c\u5c06\u7f16\u7801\u6297tau\u86cb\u767d\u6297\u4f53\u7684\u9057\u4f20\u4fe1\u606f\u4f20\u8f93\u8fdb\u5927\u8111\uff0c\u5728\u5927\u8111\u4e2d\u751f\u6210\u6297\u4f53\uff0c\u4e00\u52b3\u6c38\u9038\u5730\u51cf\u5c11Tau\u86cb\u767d\u3002\u65e0\u72ec\u6709\u5076\uff0c1\u6708\u4efd\uff0c\u5f3a\u751f\u548cJanssen Pharmaceuticals Inc.\uff08JPI\uff09\u4e0e\u5bbe\u5915\u6cd5\u5c3c\u4e9a\u5927\u5b66\u7684\u57fa\u56e0\u7597\u6cd5\u9879\u76ee\u7ec4\u7b7e\u7f72\u4e86\u72ec\u5bb6\u5408\u4f5c\u7814\u7a76\u534f\u8bae\u3002\u8be5\u5408\u4f5c\u5c06\u540c\u65f6\u5229\u7528\u7531\u5bbe\u5915\u6cd5\u5c3c\u4e9a\u5927\u5b66\u7814\u53d1\u7684\u817a\u76f8\u5173\u75c5\u6bd2\uff08AAV\uff09\u8f7d\u4f53\u548cJPI\u7814\u53d1\u7684\u9776\u5411\u963f\u5179\u6d77\u9ed8\u75c5\u7684\u6297\u4f53\uff0c\u5f00\u5c55\u7814\u7a76\u3002\u8fd1\u5e74\u6765\uff0c\u5404\u9879\u963f\u5179\u6d77\u9ed8\u75c5\u7684\u836f\u7269\u7814\u53d1\u9879\u76ee\u7eb7\u7eb7\u6298\u621f\uff0c\u5e0c\u671b\u57fa\u56e0\u6cbb\u7597\u548c\u6297\u4f53\u6280\u672f\u7684\u7ed3\u5408\u80fd\u7ed9\u8fd9\u4e2a\u9886\u57df\u5e26\u6765\u4e00\u4e1d\u66d9\u5149\u3002<\/span><\/p>\n<p style=\"text-align: justify;\"><span style=\"font-size: 15px;\"><br  \/><\/span><\/p>\n<p style=\"text-align: justify;\"><span style=\"font-size: 15px;\">2\u670822\u65e5\uff0c\u836f\u660e\u5eb7\u5fb7\u96c6\u56e2\u4f01\u4e1a\u5408\u5168\u836f\u4e1a\u5ba3\u5e03\u4e0eRegulus Therapeutics\u8fbe\u6210\u5408\u4f5c\u534f\u8bae\uff0c\u5171\u540c\u5f00\u5c55\u5be1\u6838\u82f7\u9178\u9886\u57df\u7684\u7814\u53d1\u4ee5\u53ca\u4e2d\u578b\u89c4\u6a21\u7684non-GMP\/GMP\u751f\u4ea7\u3002Regulus\u662f\u4e00\u5bb6\u751f\u7269\u5236\u836f\u516c\u53f8\uff0c\u81f4\u529b\u4e8e\u5f00\u53d1\u4ee5microRNA\u4e3a\u9776\u6807\u7684\u521b\u65b0\u836f\u7269\u3002\u836f\u660e\u5eb7\u5fb7\u4f5c\u4e3a\u56fd\u5185\u533b\u836f\u7814\u53d1CRO\u4e2d\u7684\u72ec\u89d2\u517d\uff0c\u4ea7\u4e1a\u94fe\u5e03\u5c40\u4e4b\u5e7f\uff0c\u4ee4\u4eba\u5370\u8c61\u6df1\u523b\u3002<\/span><\/p>\n<p style=\"text-align: center;\"><img class=\"\"      style=\"width: 288px;height: 146px;\" src=\"http:\/\/www.05921.net\/wp-content\/uploads\/2018\/07\/beepress9-1532683942.png\" alt=\"2018\u5e74\u5168\u7403\u57fa\u56e0\u6cbb\u7597\u9886\u57df\u8fdb\u5c55\uff08BD\u4e0b\u7bc7\uff09\" title=\"2018\u5e74\u5168\u7403\u57fa\u56e0\u6cbb\u7597\u9886\u57df\u8fdb\u5c55\uff08BD\u4e0b\u7bc7\uff09\"  \/><\/p>\n<p style=\"text-align: justify;\"><span style=\"font-size: 15px;\"><br  \/><\/span><\/p>\n<p style=\"text-align: justify;\"><span style=\"font-size: 15px;\">\u65f6\u95f4\u6765\u52303\u6708\uff0c\u518d\u751f\u5143\uff08Regeneron\uff09\u4e0eAlnylam\u5ba3\u5e03\u8fbe\u6210\u5408\u4f5c\uff0c\u4ee5\u5f00\u53d1\u7528\u4e8e\u6cbb\u7597\u975e\u9152\u7cbe\u6027\u8102\u80aa\u6027\u809d\u708e\uff08NASH\uff09\u548c\u5176\u5b83\u76f8\u5173\u75be\u75c5\u7684RNAi\u7597\u6cd5\u3002\u57fa\u4e8e\u8fd9\u9879\u7814\u53d1\u5408\u4f5c\uff0c\u518d\u751f\u5143\u5c06\u8d1f\u8d23\u5bf9\u809d\u7ec6\u80de\u8868\u8fbe\u3001\u9057\u4f20\u5b66\u9a8c\u8bc1\u7684HSD17B13\u9776\u70b9\u8fdb\u884c\u7814\u7a76\uff0cAlnylam\u5c06\u5229\u7528\u5176RNAi\u6cbb\u7597\u5e73\u53f0\u6765\u8bc6\u522b\u9488\u5bf9\u8be5\u9776\u70b9\u7684\u5316\u5408\u7269\u3002\u518d\u751f\u5143\u548cAlnylam\u6253\u7b97\u5728\u672a\u6765\u8fdb\u884c\u5355\u72ec\u7684\u4e94\u4e94\u5206\u5408\u4f5c\uff08fifty-fifty collaboration\uff09\uff0c\u4ee5\u8fdb\u4e00\u6b65\u7814\u53d1\u548c\u63a8\u5e7f\u7531\u6b64\u6b21\u5408\u4f5c\u4ea7\u751f\u7684\u4efb\u4f55\u6cbb\u7597\u5019\u9009\u4ea7\u54c1\u3002<\/span><\/p>\n<p style=\"text-align: center;\"><img class=\"\"      style=\"width: 516px;height: 80px;\" src=\"http:\/\/www.05921.net\/wp-content\/uploads\/2018\/07\/beepress7-1532683942.png\" alt=\"2018\u5e74\u5168\u7403\u57fa\u56e0\u6cbb\u7597\u9886\u57df\u8fdb\u5c55\uff08BD\u4e0b\u7bc7\uff09\" title=\"2018\u5e74\u5168\u7403\u57fa\u56e0\u6cbb\u7597\u9886\u57df\u8fdb\u5c55\uff08BD\u4e0b\u7bc7\uff09\"  \/><\/p>\n<p style=\"text-align: justify;\"><span style=\"font-size: 15px;\"><br  \/><\/span><\/p>\n<p style=\"text-align: justify;\"><strong><span style=\"font-size: 15px;color: rgb(0, 209, 0);\">\u76f4\u63a5\u4e70\u65ad<\/span><\/strong><\/p>\n<p style=\"text-align: justify;\"><span style=\"font-size: 15px;\">\u540c\u6837\u662fNASH\uff0c\u963f\u65af\u5229\u5eb7\u91c7\u7528\u53e6\u4e00\u79cd\u5408\u4f5c\u65b9\u5f0f\u30024\u670810\u65e5\uff0c\u4f4d\u4e8e\u52a0\u5dde\u5723\u5730\u4e9a\u54e5\u7684Ionis Pharmaceuticals\u516c\u53f8\u5ba3\u5e03\u8be5\u516c\u53f8\u6388\u4e88\u963f\u65af\u5229\u5eb7\u8fdb\u4e00\u6b65\u5f00\u53d1IONIS-AZ6-2.5-LRx\uff08\u6cbb\u7597NASH\uff09\u7684\u72ec\u5bb6\u6388\u6743\u3002Ionis\u516c\u53f8\u5c06\u4ece\u963f\u65af\u5229\u5eb7\u83b7\u5f973000\u4e07\u7f8e\u5143\u7684\u4ed8\u6b3e\uff0c\u5e76\u4e14\u6839\u636e\u836f\u7269\u5f00\u53d1\u8fc7\u7a0b\u4e2d\u7684\u91cc\u7a0b\u7891\u53ef\u80fd\u83b7\u5f97\u989d\u5916\u9ad8\u8fbe3\u4ebf\u7f8e\u5143\u7684\u4ed8\u6b3e\u3002\u963f\u65af\u5229\u5eb7\u5c06\u8d1f\u8d23\u8fd9\u4e00\u836f\u7269\u968f\u540e\u7684\u6240\u6709\u5f00\u53d1\u548c\u63a8\u5e7f\u8fc7\u7a0b\u3002Ionis\u516c\u53f8\u5f00\u53d1\u7684IONIS-AZ6-2.5-LRx\u662f\u6291\u5236\u4e00\u4e2a\u672a\u62ab\u9732\u7684NASH\u9776\u70b9\u7684\u53cd\u4e49RNA\u7597\u6cd5\u3002\u8fd9\u4e00\u7597\u6cd5\u5e94\u7528\u4e86Ionis\u516c\u53f8\u6700\u65b0\u76842.5\u4ee3\u5316\u5b66\u4fee\u9970\u6280\u672f\u548c\u914d\u4f53\u590d\u5408\u53cd\u4e49\u6280\u672f\uff0c\u4e0e2\u4ee3\u76f8\u6bd4\u836f\u6548\u53ef\u4ee5\u63d0\u9ad810\u500d\u3002<\/span><\/p>\n<p style=\"text-align: justify;\"><span style=\"font-size: 15px;\"><br  \/><\/span><\/p>\n<p style=\"text-align: justify;\"><strong><span style=\"font-size: 15px;color: rgb(0, 209, 0);\">\u5c0f\u7ed3<\/span><\/strong><\/p>\n<p style=\"text-align: justify;\"><span style=\"font-size: 15px;\">\u81ea\u53bb\u5e74FDA\u6279\u51c6\u4e24\u4e2aCAR-T\u836f\u7269\u4ee5\u6765\uff0c\u7279\u522b\u662f\u5e74\u5e95\u524d\u6279\u51c6\u7f8e\u56fd\u9996\u4e2a\u57fa\u56e0\u6cbb\u7597\u836f\u7269\uff0c\u8d44\u91d1\u5f00\u59cb\u5927\u89c4\u6a21\u8fdb\u5165\u57fa\u56e0\u6cbb\u7597\u9886\u57df\uff0c\u5176\u4e2d\u4e0d\u4e4f\u4f20\u7edf\u5236\u836f\u5de8\u5934\u3002\u7ecf\u8fc7\u8fd130\u5e74\u7684\u53d1\u5c55\uff0c<strong>\u57fa\u56e0\u6cbb\u7597<\/strong>\u6709\u671b\u9010\u6b65\u6210\u4e3a\u5e38\u89c4\u6cbb\u7597\u624b\u6bb5\uff0c\u7279\u522b\u662f\u5404\u79cd\u9057\u4f20\u6027\u75be\u75c5\u3002\u57fa\u56e0\u6cbb\u7597\u836f\u7269\u7531\u4e8e\u75c5\u4eba\u6570\u76ee\u6709\u9650\uff0c\u7edd\u5927\u90e8\u5206\u90fd\u662f\u7f55\u89c1\u75c5\uff0c\u9020\u6210\u836f\u7269\u5b9a\u4ef7\u8fc7\u9ad8\u3002\u6b27\u76df\u6279\u51c6\u7684\u4e24\u4e2a\u57fa\u56e0\u6cbb\u7597\u836f\u7269\u548cSpark\u521a\u521a\u83b7\u6279\u7684\u57fa\u56e0\u6cbb\u7597\u836f\u7269\uff0c\u5b9a\u4ef7\u90fd\u662f\u51e0\u5341\u4e07\u7f8e\u5143\uff0c\u5bf9\u533b\u4fdd\u4f53\u7cfb\u5e26\u6765\u5f88\u5927\u652f\u4ed8\u538b\u529b\u3002\u7136\u800c\uff0c<strong>\u5c0f\u7f16\u8ba4\u4e3a<\/strong>\u79d1\u6280\u8fdb\u6b65\u7684\u521d\u8877\u662f\u4e3a\u4e86\u9020\u798f\u4eba\u7c7b\uff0c\u5b9a\u4ef7\u8fc7\u9ad8\u7684\u95ee\u9898\u968f\u7740\u6280\u672f\u6210\u719f\u548c\u5e94\u7528\u63a8\u5e7f\uff0c\u6709\u671b\u8fdb\u4e00\u6b65\u964d\u4f4e\u3002\u591a\u4e00\u79cd\u6cbb\u7597\u624b\u6bb5\u5bf9\u4e8e\u9971\u53d7\u75c5\u75db\u6298\u78e8\u7684\u60a3\u8005\u6765\u8bf4\uff0c\u65e0\u7591\u662f\u5929\u5927\u7684\u798f\u97f3\u3002\u8fd9\u4e5f\u5e94\u8be5\u662f\u6bcf\u4e00\u4f4d\u533b\u836f\u884c\u4e1a\u4ece\u4e1a\u4eba\u5458\u7684\u521d\u8877\u3002\u4e0d\u5fd8\u521d\u5fc3\uff0c\u653e\u5f97\u59cb\u7ec8\uff01<\/span><\/p>\n<p 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border-box !important;word-wrap: break-word !important;\"><strong style=\"max-width: 100%;color: rgb(0, 112, 192);line-height: 19.6px;font-family: inherit;font-size: 1em;text-decoration: inherit;box-sizing: border-box !important;word-wrap: break-word !important;\">\u5c0f\u7f16\u56e2\u961f\u73b0\u670921\u4f4d\u6210\u5458\uff1a<\/strong><span style=\"max-width: 100%;color: rgb(0, 0, 0);\"><\/span><\/span><\/p>\n<p style=\"max-width: 100%;min-height: 1em;text-align: center;line-height: 19.6px;font-size: 1em;box-sizing: border-box !important;word-wrap: break-word !important;\"><span style=\"max-width: 100%;font-size: 14px;box-sizing: border-box !important;word-wrap: break-word !important;\"><span style=\"max-width: 100%;color: rgb(0, 0, 0);\">Armstrong\u3001\u533b\u836f\u5c40\u5916\u4eba\u3001Fairy\u3001<\/span><span style=\"max-width: 100%;color: rgb(0, 0, 0);line-height: 19.6px;font-family: inherit;text-decoration: inherit;\">Jone\u3001<\/span><span style=\"max-width: 100%;line-height: 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class='keep-source'>\n<p>\u59cb\u53d1\u4e8e\u5fae\u4fe1\u516c\u4f17\u53f7\uff1a                         \u751f\u7269\u5236\u836f\u5c0f\u7f16                      <\/p>\n<\/blockquote>\n<\/div>\n","protected":false},"excerpt":{"rendered":"<p>\u4f5c\u8005 | \u5403\u74dc\u7fa4\u4f17 \u7f16\u8f91 | \u7ec6\u80de\u623f\u95f4 \u5728\u4e0a\u4e00\u7bc7\u4e2d\uff082018\u5e74\u5168\u7403\u57fa\u56e0\u6cbb\u7597\u9886\u57df\u8fdb\u5c55\u2014BD\u4e0a\u7bc7\uff09\uff0c\u5c0f\u7f16\u56de\u987e\u4e8620 [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":8438,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":[],"categories":[2],"tags":[],"_links":{"self":[{"href":"https:\/\/www.05921.net\/index.php\/wp-json\/wp\/v2\/posts\/8437"}],"collection":[{"href":"https:\/\/www.05921.net\/index.php\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.05921.net\/index.php\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.05921.net\/index.php\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/www.05921.net\/index.php\/wp-json\/wp\/v2\/comments?post=8437"}],"version-history":[{"count":1,"href":"https:\/\/www.05921.net\/index.php\/wp-json\/wp\/v2\/posts\/8437\/revisions"}],"predecessor-version":[{"id":8447,"href":"https:\/\/www.05921.net\/index.php\/wp-json\/wp\/v2\/posts\/8437\/revisions\/8447"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.05921.net\/index.php\/wp-json\/wp\/v2\/media\/8438"}],"wp:attachment":[{"href":"https:\/\/www.05921.net\/index.php\/wp-json\/wp\/v2\/media?parent=8437"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.05921.net\/index.php\/wp-json\/wp\/v2\/categories?post=8437"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.05921.net\/index.php\/wp-json\/wp\/v2\/tags?post=8437"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}