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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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\u4eca\u65e5\uff0cAlnylam Pharmaceuticals\u5ba3\u5e03\u5176\u9776\u5411\u6c28\u57fa\u4e59\u9170\u4e19\u9178\u5408\u6210\u91761\uff08ALAS1\uff09\u7684\u5728\u7814RNAi\u836f\u7269givosiran\uff0c\u5728\u7528\u4e8e\u6cbb\u7597\u6025\u6027\u809d\u535f\u5549\u75c7\uff08AHP\uff09\u76843\u671f\u8bd5\u9a8cENVISION\u4e2d\uff0c\u53d6\u5f97\u4e86\u79ef\u6781\u9876\u7ebf\u6570\u636e\u3002<\/strong>Alnylam\u6cbb\u7597\u6210\u4eba\u7531\u9057\u4f20\u6027\u8f6c\u7532\u72b6\u817a\u7d20\u86cb\u767d\u6dc0\u7c89\u6837\u53d8\u6027\uff08hATTR\uff09\u5f15\u8d77\u7684\u591a\u53d1\u6027\u795e\u7ecf\u75c5\uff08polyneuropathy\uff09\u7684Onpattro\uff08patisiran\uff09\uff0c\u662f<\/span>FDA\u6279\u51c6\u7684\u7b2c\u4e00\u6b3eRNAi\u836f\u7269<\/span><\/a>\u3002\u9274\u4e8egivosiran\u7684\u4f18\u5f02\u8868\u73b0\uff0c\u5176\u6709\u671b\u5f97\u5230\u52a0\u901f\u6279\u51c6\uff0c\u6210\u4e3a\u7b2c\u4e8c\u6b3e\u4e0a\u5e02RNAi\u65b0\u836f<\/strong>\u3002<\/span><\/p>\n

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\"\u901f\u9012<\/p>\n


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AHPs\u662f\u4e00\u79cd\u7f55\u89c1\u7684\u9057\u4f20\u6027\u75be\u75c5\uff0c\u5176\u7279\u5f81\u662f\u53ef\u80fd\u5371\u53ca\u751f\u547d\u7684\u53d1\u4f5c\uff0c\u5e76\u5bf9\u65e5\u5e38\u529f\u80fd\u548c\u751f\u6d3b\u8d28\u91cf\u6709\u8d1f\u9762\u5f71\u54cd\uff0c\u7279\u522b\u662f\u90a3\u4e9b\u6709\u6162\u6027\u8870\u5f31\u75c7\u72b6\u7684AHPs\u60a3\u8005\u3002AHPs\u6709\u56db\u79cd\u4e9a\u578b\uff0c\u5206\u522b\u4e3a\u6025\u6027\u95f4\u6b47\u6027\u535f\u5549\u75c7\uff08AIP\uff09\uff0c\u9057\u4f20\u6027\u7caa\u535f\u5549\u75c7\uff08HCP\uff09\uff0c\u591a\u6837\u6027\u535f\u5549\u75c7\uff08VP\uff09\u548cALAD\u7f3a\u4e4f\u6027\u535f\u5549\u75c7\uff08ADP\uff09\u3002AHPs\u7686\u4e3a\u9057\u4f20\u7f3a\u9677\u5f15\u8d77\u7684\u4e00\u79cd\u9176\u7f3a\u4e4f\u3002\u8fd9\u79cd\u5728\u809d\u810f\u4e2d\u8840\u7ea2\u7d20\u751f\u7269\u5408\u6210\u9014\u5f84\u4e2d\u7684\u9176\u7f3a\u4e4f\uff0c\u5bfc\u81f4\u5177\u6709\u795e\u7ecf\u6bd2\u6027\u7684\u8840\u7ea2\u7d20\u4e2d\u95f4\u4f53\u6c28\u57fa\u4e59\u9170\u4e19\u9178\uff08ALA\uff09\u548c\u80c6\u8272\u7d20\u539f\uff08PBG\uff09\u7684\u79ef\u7d2f\u3002\u5176\u4e2d\uff0cALA\u88ab\u8ba4\u4e3a\u662f\u4e3b\u8981\u7684\u795e\u7ecf\u6bd2\u6027\u4e2d\u95f4\u4f53\uff0c\u5bfc\u81f4\u75be\u75c5\u53d1\u4f5c\u548c\u53d1\u4f5c\u95f4\u671f\u7684\u75c7\u72b6\u6301\u7eed\u3002AHP\u7684\u5e38\u89c1\u75c7\u72b6\u5305\u62ec\u4e25\u91cd\u7684\u5f25\u6f2b\u6027\u8179\u75db\uff0c\u865a\u5f31\uff0c\u6076\u5fc3\u548c\u75b2\u52b3\u3002\u56e0\u5176\u75c7\u72b6\u901a\u5e38\u7c7b\u4f3c\u4e8e\u5176\u4ed6\u66f4\u5e38\u89c1\u7684\u75be\u75c5\uff0c\u5982\u80a0\u6613\u6fc0\u7efc\u5408\u5f81\uff0c\u9611\u5c3e\u708e\uff0c\u7ea4\u7ef4\u808c\u75db\u548c\u5b50\u5bab\u5185\u819c\u5f02\u4f4d\uff0c\u6240\u4ee5AHP\u60a3\u8005\u7ecf\u5e38\u88ab\u8bef\u8bca\u6216\u672a\u88ab\u8bca\u65ad\uff08\u5e73\u574715\u5e74\uff09\u3002\u76ee\u524d\uff0c\u8fd8\u6ca1\u6709\u83b7\u5f97\u6279\u51c6\u7528\u4e8e\u9884\u9632\u8870\u5f31\u53d1\u4f5c\u548c\u6cbb\u7597\u8be5\u75be\u75c5\u6162\u6027\u75c7\u72b6\u7684\u7597\u6cd5\u3002<\/span><\/p>\n

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\"\u901f\u9012<\/p>\n

\u25b2RNAi\u4f5c\u7528\u673a\u5236\uff08\u56fe\u7247\u6765\u6e90\uff1aAlnylam\u5b98\u7f51\uff09<\/span><\/strong>
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Givosiran\uff08ALN-AS1\uff09\u662f\u4e00\u79cd\u76ae\u4e0b\u6ce8\u5c04\u7684RNAi\u836f\u7269\uff0c\u9776\u5411ALAS1\uff0c\u7528\u4e8e\u6cbb\u7597AHPs\uff0c\u5305\u62ecAIP\uff0cVP\u548cHCP<\/span><\/strong>\u3002RNAi\u836f\u7269\u53ef\u4ee5\u9ad8\u6548\u3001\u7279\u5f02\u6027\u3001\u957f\u671f\u5730\u6c89\u9ed8\u7279\u5b9amRNA\u5e8f\u5217\u3002\u8fd9\u662f\u4e00\u9879\u8f83\u4e3a\u65b0\u9896\u7684\u6280\u672f\uff0c\u8be5\u6280\u672f\u7684\u673a\u7406\u7814\u7a76\u57282006\u5e74\u83b7\u5f97\u4e86\u8bfa\u8d1d\u5c14\u5956\u3002Givosiran\u662f\u5728Alnylam\u516c\u53f8\u72ec\u6709\u7684\u589e\u5f3a\u7a33\u5b9a\u5316\u5b66\uff08ESC\uff09-GalNAc\u6280\u672f\u5e73\u53f0\u4e0a\u5f00\u53d1\u800c\u6210\u7684\u3002\u6b64\u524d\uff0cgivosiran\u66fe\u83b7\u5f97\u6b27\u6d32\u836f\u54c1\u7ba1\u7406\u5c40\uff08EMA\uff09\u7684\u4f18\u5148\u836f\u54c1\uff08PRIME\uff09\u8d44\u683c\uff0c\u6b27\u76df\u548c\u7f8e\u56fd\u7684\u5b64\u513f\u836f\u8d44\u683c\uff0c\u4ee5\u53caFDA\u7684\u7a81\u7834\u6027\u7597\u6cd5\u8ba4\u5b9a\u3002<\/strong><\/span><\/p>\n


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ENVISION\u662f\u4e00\u9879\u968f\u673a\u3001\u53cc\u76f2\u3001\u5b89\u6170\u5242\u5bf9\u7167\u3001\u5168\u7403\u6027\u3001\u591a\u4e2d\u5fc3\u7814\u7a76\u76843\u671f\u8bd5\u9a8c\uff0c\u65e8\u5728\u8bc4\u4f30givosiran\u6cbb\u7597AHPs\u7684\u6709\u6548\u6027\u548c\u5b89\u5168\u6027\u3002\u57286\u4e2a\u6708\u7684\u6cbb\u7597\u671f\u5185\uff0c\u60a3\u8005\u4ee51:1\u7684\u6bd4\u4f8b\u968f\u673a\u5206\u7ec4\uff0c\u5206\u522b\u63a5\u53d7\u6bcf\u6708\u76ae\u4e0b\u6ce8\u5c04givosiran\uff082.5 mg\/kg\uff09\uff0c\u6216\u5b89\u6170\u5242\u3002\u8bd5\u9a8c\u7684\u4e3b\u8981\u7ec8\u70b9\u662f\u535f\u5549\u75c7\u7684\u201c\u5e74\u53d1\u4f5c\u7387\u201d\uff0c\u5373\u9700\u8981\u4f4f\u9662\u6cbb\u7597\uff0c\u7d27\u6025\u533b\u7597\u62a4\u7406\u8bbf\u95ee\uff0c\u6216\u5bb6\u4e2d\u4f7f\u7528\u6c2f\u5316\u8840\u7ea2\u7d20\u4e8b\u4ef6\u7684\u5e74\u53d1\u4f5c\u7387\u3002\u8bd5\u9a8c\u7684\u4e2d\u671f\u5206\u6790\u5305\u62ec43\u540d\u63a5\u53d7\u8bd5\u9a8c\u81f3\u5c113\u4e2a\u6708\u7684AHP\u60a3\u8005\uff0c\u5e76\u8bc4\u4f30\u4e8641\u4f8bAIP\u60a3\u8005\u5c3f\u6db2\u4e2d\u751f\u7269\u6807\u5fd7\u7269ALA\u7684\u6c34\u5e73\uff0c\u8fd9\u4e00\u9884\u5148\u786e\u5b9a\u7684\u66ff\u4ee3\u7ec8\u70b9\u53ef\u9884\u6d4b\u53ef\u80fd\u7684\u4e34\u5e8a\u6536\u76ca\u3002\u5173\u952e\u6b21\u8981\u7ec8\u70b9\u548c\u63a2\u7d22\u7ec8\u70b9\u5305\u62ecAHP\u6807\u5fd7\u6027\u75c7\u72b6\uff08\u75bc\u75db\uff0c\u6076\u5fc3\u548c\u75b2\u52b3\uff09\u7684\u51cf\u5c11\uff0c\u4ee5\u53ca\u5bf9\u751f\u6d3b\u8d28\u91cf\u7684\u5f71\u54cd\u3002<\/span><\/p>\n


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\u8bd5\u9a8c\u7ed3\u679c\u7684\u4e2d\u671f\u5206\u6790\u8868\u660e\uff0cgivosiran\u7597\u6cd5\u5728AIP\u60a3\u8005\u4e2d\uff0c\u4e0e\u5b89\u6170\u5242\u76f8\u6bd4\uff0c\u80fd\u591f\u663e\u8457\u964d\u4f4e\u5c3f\u6db2\u4e2dALA\u6c34\u5e73\uff08p<0.001\uff09\u3002<\/span><\/strong><\/span><\/p>\n

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\"\u901f\u9012<\/p>\n

\u25b2Alnylam\u7814\u53d1\u603b\u88c1Akshay Vaishnaw\u535a\u58eb\uff08\u56fe\u7247\u6765\u6e90\uff1aAlnylam\u5b98\u7f51)<\/span><\/strong>
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Alnylam\u7814\u53d1\u603b\u88c1Akshay Vaishnaw\u535a\u58eb\u8bf4\uff1a\u201cAHPs\u662f\u4e00\u79cd\u6bc1\u706d\u6027\u75be\u75c5\uff0c\u65e0\u8bba\u662f\u8111\u810a\u9ad3\u4ea4\u611f\u795e\u7ecf\u7cfb\u7edf\u7684\u53d1\u4f5c\uff0c\u8fd8\u662f\u6162\u6027\u75bc\u75db\u548c\u75b2\u52b3\uff0c\u90fd\u5bfc\u81f4\u60a3\u8005\u8870\u5f31\u3002\u4ee4Alnylam\u611f\u5230\u9ad8\u5174\u548c\u9f13\u821e\u7684\u662f\uff0cENVISION 3\u671f\u7814\u7a76\u7684\u4e2d\u671f\u5206\u6790\u8868\u660e\uff0cgivosiran\u6cbb\u7597\u4f34\u968f\u7740ALA\u7684\u7edf\u8ba1\u5b66\u663e\u8457\u964d\u4f4e\u3002ALA\u662f\u4e00\u79cd\u5408\u7406\u7684\u3001\u975e\u5e38\u53ef\u80fd\u9884\u6d4b\u4e34\u5e8a\u83b7\u76ca\u7684\u75be\u75c5\u751f\u7269\u6807\u5fd7\u7269\u3002\u57fa\u4e8e\u8fd9\u4e9bENVISION\u8bd5\u9a8c\u7684\u4e2d\u671f\u7ed3\u679c\uff0cAlnylam\u8ba1\u5212\u4e0eFDA\u4f1a\u9762\uff0c\u8ba8\u8bba\u5728\u5e74\u5e95\u524d\u63d0\u4ea4\u65b0\u836f\u7533\u8bf7\uff08NDA\uff09\u548c\u83b7\u5f97\u52a0\u901f\u6279\u51c6\u7684\u76f8\u5173\u95ee\u9898\u3002\u4e0e\u6b64\u540c\u65f6\uff0c\u7531\u4e8eENVISION\u7684\u60a3\u8005\u6ce8\u518c\u63d0\u524d\u5b8c\u6210\uff0c\u6211\u4eec\u671f\u5f85\u572819\u5e74\u521d\u516c\u5e03\u5b8c\u6574\u7814\u7a76\u7684\u9876\u7ebf\u7ed3\u679c\u3002\u5982\u679c\u5728\u6574\u4e2a\u8bd5\u9a8c\u4e2d\u80fd\u591f\u786e\u8ba4\u5176\u4e34\u5e8a\u7597\u6548\u548c\u53ef\u63a5\u53d7\u7684\u5b89\u5168\u6027\uff0c\u6211\u4eec\u76f8\u4fe1givosiran\u6709\u53ef\u80fd\u6539\u53d8AHP\u60a3\u8005\u7684\u751f\u6d3b\u3002\u201d<\/span><\/p>\n


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

[1]. Alnylam Announces Positive Topline Results from Interim Analysis of ENVISION Phase 3 Study of Givosiran in Patients with Acute Hepatic Porphyria. Retrieved September 26, 2018, from https:\/\/www.businesswire.com\/news\/home\/20180927005235\/en\/Alnylam-Announces-Positive-Topline-Results-Interim-Analysis<\/span><\/p>\n

[2]. \u91cc\u7a0b\u7891\uff01\u9996\u6b3eRNAi\u7597\u6cd5\u4eca\u65e5\u83b7\u6279. Retrieved September 26, 2018, from https:\/\/mp.weixin.qq.com\/s\/etcQd2D9zKt_zYoA5PSJBw<\/span><\/p>\n

[3]. \u809d\u75c5\u521b\u65b0\u57fa\u56e0\u7597\u6cd5\u83b7FDA\u7a81\u7834\u6027\u7597\u6cd5\u8ba4\u5b9a. Retrieved September 27, 2018, from https:\/\/mp.weixin.qq.com\/s\/zQ7T0Dtjy_WdsxKcetMRVw<\/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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