{"id":85283,"date":"2019-04-15T00:00:50","date_gmt":"2019-04-17T03:19:42","guid":{"rendered":"http:\/\/www.05921.net\/index.php\/85283\/"},"modified":"2019-04-17T11:19:43","modified_gmt":"2019-04-17T03:19:43","slug":"7e3e787d71","status":"publish","type":"post","link":"https:\/\/www.05921.net\/index.php\/85283\/","title":{"rendered":"\u901f\u9012 | RNAi\u7597\u6cd5\u8fbe\u52303\u671f\u4e34\u5e8a\u7ec8\u70b9\uff0c\u5373\u5c06\u5b8c\u6210\u6eda\u52a8\u65b0\u836f\u7533\u8bf7"},"content":{"rendered":"
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\u258e\u836f\u660e<\/span><\/strong>\u5eb7\u5fb7\/\u62a5\u9053<\/span><\/strong><\/strong><\/span><\/strong><\/strong><\/span><\/strong><\/strong><\/strong><\/span><\/strong><\/strong><\/span><\/strong><\/strong><\/span><\/span><\/strong><\/strong><\/span><\/strong><\/strong><\/span><\/strong><\/strong><\/strong><\/span><\/strong><\/strong><\/span><\/strong><\/strong><\/h1>\n

\u65e5\u524d\uff0cAlnylam Pharmaceuticals\u516c\u53f8\u5ba3\u5e03\uff0c\u8be5\u516c\u53f8\u9776\u5411\u6c28\u57fa\u4e59\u9170\u4e19\u9178\u5408\u6210\u91761\uff08ALAS1\uff09\u7684RNAi\u7597\u6cd5givosiran\uff0c\u5728\u6cbb\u7597\u6025\u6027\u809d\u535f\u5549\u75c7\uff08AHP\uff09\u76843\u671f\u4e34\u5e8a\u8bd5\u9a8c\u4e2d\u8fbe\u5230\u91cd\u8981\u7ec8\u70b9\u548c\u591a\u9879\u6b21\u8981\u7ec8\u70b9<\/strong>\u3002\u8fd9\u4e00\u7ed3\u679c\u5728\u5965\u5730\u5229\u7ef4\u4e5f\u7eb3\u4e3e\u884c\u7684\u56fd\u9645\u809d\u810f\u5927\u4f1a\u4e0a\u516c\u5e03\u3002<\/span><\/p>\n


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

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AHP\u662f\u4e00\u7c7b\u7f55\u89c1\u9057\u4f20\u75c5\uff0c\u5176\u7279\u5f81\u662f\u53ef\u80fd\u5371\u53ca\u751f\u547d\u7684\u75be\u75c5\u53d1\u4f5c\u3002\u5bf9\u67d0\u4e9b\u60a3\u8005\u6765\u8bf4\uff0c\u957f\u671f\u8870\u5f31\u6027\u75c7\u72b6\u4f1a\u5bf9\u4ed6\u4eec\u7684\u65e5\u5e38\u751f\u6d3b\u548c\u751f\u6d3b\u8d28\u91cf\u4ea7\u751f\u8d1f\u9762\u5f71\u54cd\u3002AHP\u6709\u56db\u79cd\u4e9a\u578b\uff0c\u90fd\u662f\u7531\u4e8e\u57fa\u56e0\u7a81\u53d8\u5bfc\u81f4\u809d\u810f\u5185\u8840\u7ea2\u7d20\u751f\u7269\u5408\u6210\u9014\u5f84\u4e2d\u67d0\u79cd\u9176\u7684\u7f3a\u5931\u3002\u8fd9\u4e9b\u7f3a\u5931\u5bfc\u81f4\u5177\u6709\u795e\u7ecf\u6bd2\u6027\u7684\u8840\u7ea2\u7d20\u4e2d\u95f4\u4ea7\u7269\u6c28\u57fa\u4e59\u9170\u4e19\u9178\uff08ALA\uff09\u548c\u80c6\u8272\u7d20\u539f\uff08PBG\uff09\u79ef\u7d2f\u3002ALA\u88ab\u8ba4\u4e3a\u662f\u5bfc\u81f4\u75be\u75c5\u53d1\u4f5c\u548c\u53d1\u4f5c\u4e4b\u95f4\u6301\u7eed\u75c7\u72b6\u7684\u4e3b\u8981\u795e\u7ecf\u6bd2\u6027\u4e2d\u95f4\u4ea7\u7269\u3002\u76ee\u524d\uff0c\u8fd8\u6ca1\u6709\u83b7\u6279\u7597\u6cd5\u6765\u9884\u9632\u75be\u75c5\u53d1\u4f5c\u6216\u8005\u6cbb\u7597\u75be\u75c5\u7684\u6162\u6027\u75c7\u72b6\u3002<\/span><\/p>\n


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Givosiran\u662f\u4e00\u6b3e\u76ae\u4e0b\u6ce8\u5c04\u7684RNAi\u7597\u6cd5\u3002<\/span>\u6bcf\u6708\u63a5\u53d7givosiran\u6cbb\u7597\u80fd\u591f\u663e\u8457\u6301\u7eed\u964d\u4f4e\u809d\u810f\u4e2dALAS1\u7684\u6c34\u5e73\uff0c\u4ece\u800c\u964d\u4f4eALA\u548cPBG\u7684\u6c34\u5e73\u81f3\u63a5\u8fd1\u6b63\u5e38\u6c34\u5e73<\/span><\/strong>\u3002Givosiran\u5df2\u7ecf\u83b7\u5f97\u4e86\u7f8e\u56fdFDA\u6388\u4e88\u7684\u7a81\u7834\u6027\u7597\u6cd5\u8ba4\u5b9a\u548c\u6b27\u6d32EMA\u6388\u4e88\u7684PRIME\u8ba4\u5b9a\u3002<\/span><\/p>\n


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

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\u25b2Givosiran\u4f5c\u7528\u673a\u5236\uff08\u56fe\u7247\u6765\u6e90\uff1a<\/span><\/strong>Alnylam\u5b98\u7f51\uff09<\/span><\/strong><\/span><\/h1>\n


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\u5728\u540d\u4e3aENVISION\u7684\u968f\u673a\u53cc\u76f2\uff0c\u542b\u5b89\u6170\u5242\u5bf9\u7167\u76843\u671f\u4e34\u5e8a\u8bd5\u9a8c\u4e2d\uff0c94\u540dAHP\u60a3\u8005\u63a5\u53d7\u4e86givosiran\u6216\u5b89\u6170\u5242\u7684\u6cbb\u7597\u3002\u5728\u63a5\u53d7\u6cbb\u75976\u4e2a\u6708\u4e4b\u540e\uff0cgivosiran\u8fbe\u5230\u4e86\u8bd5\u9a8c\u7684\u4e3b\u8981\u7ec8\u70b9\uff0c\u4e0e\u5b89\u6170\u5242\u76f8\u6bd4\uff0c\u5c06\u60a3\u8005\u590d\u5408\u5e74\u53d1\u4f5c\u7387\u5e73\u5747\u964d\u4f4e74%<\/strong>\u3002Givosiran\u7ec4\u4e2d50%<\/strong>\u7684\u60a3\u8005\u57286\u4e2a\u6708\u7684\u8bd5\u9a8c\u8fc7\u7a0b\u4e2d\u6ca1\u6709\u51fa\u73b0\u53d1\u4f5c\uff0c\u5bf9\u7167\u7ec4\u8fd9\u4e00\u6570\u503c\u4e3a16.3%<\/strong>\u3002\u6b64\u5916\uff0cgivosiran\u8fd8\u8fbe\u52305\u9879\u6b21\u8981\u7ec8\u70b9\uff0c\u5305\u62ec\u5728\u6025\u6027\u95f4\u6b47\u6027\u535f\u5549\u75c7\uff08AIP\uff09\u60a3\u8005\u4e2d\u57283\u4e2a\u6708\u540e\u5c06\u5c3f\u6db2\u4e2dALA\u6c34\u5e73\u964d\u4f4e91%\uff1b\u57286\u4e2a\u6708\u540e\u5c06\u5c3f\u6db2\u4e2dALA\u6c34\u5e73\u964d\u4f4e83%\uff1b\u57286\u4e2a\u6708\u540e\u5c06\u5c3f\u6db2\u4e2dPBG\u964d\u4f4e73%\u3002<\/span><\/p>\n


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\u201cAHP\u60a3\u8005\u5177\u6709\u9ad8\u5ea6\u672a\u7adf\u9700\u6c42\uff0c\u8fd9\u4e9b\u7ed3\u679c\u8868\u660e\u6211\u4eec\u53ef\u80fd\u4e3aAHP\u60a3\u8005\u548c\u4ed6\u4eec\u7684\u5bb6\u4eba\u63d0\u4f9b\u4e00\u6b3e\u65b0\u7684\u6cbb\u7597\u9009\u62e9\uff0c\u201dAlnylam\u516c\u53f8\u7814\u53d1\u603b\u88c1Akshay Vaishnaw\u535a\u58eb\u8bf4\uff1a\u201cGivosiran\u80fd\u591f\u663e\u8457\u964d\u4f4e\u75be\u75c5\u53d1\u4f5c\u9891\u7387\u3002\u6211\u4eec\u575a\u4fe1\u5b83\u6709\u6f5c\u529b\u6210\u4e3a\u6cbb\u7597AHP\u60a3\u8005\u7684\u4e00\u79cd\u53d8\u9769\u6027\u7597\u6cd5\u3002\u201d<\/span><\/p>\n


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\u6839\u636e\u8fd9\u4e00\u7ed3\u679c\uff0cAlnylam\u8ba1\u5212\u5b8c\u6210givosiran\u7684\u6eda\u52a8\u65b0\u836f\u7533\u8bf7\uff08NDA\uff09\uff0c\u5e76\u4e14\u5728\u4eca\u5e74\u5e74\u4e2d\u5411EMA\u9012\u4ea4\u8425\u9500\u6388\u6743\u7533\u8bf7\uff08MAA\uff09\u3002<\/span><\/p>\n


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

[1] Alnylam Presents Positive Complete Results from ENVISION Phase 3 Study of Givosiran, an Investigational RNAi Therapeutic for the Treatment of Acute Hepatic Porphyria. Retrieved April 12, 2019, from https:<\/span>\/\/www.businesswire.com\/news\/home\/20190412005455\/en\/Alnylam-Presents-Positive-Complete-Results-ENVISION-Phase<\/span><\/span><\/p>\n


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\u672c\u6587\u6765\u81ea\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><\/p>\n


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

\u258e\u836f\u660e\u5eb7\u5fb7\/\u62a5\u9053 \u65e5\u524d\uff0cAlnylam Pharmaceuticals\u516c\u53f8\u5ba3\u5e03\uff0c\u8be5\u516c\u53f8\u9776\u5411\u6c28\u57fa\u4e59\u9170\u4e19 […]<\/p>\n","protected":false},"author":1,"featured_media":85284,"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\/85283"}],"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=85283"}],"version-history":[{"count":1,"href":"https:\/\/www.05921.net\/index.php\/wp-json\/wp\/v2\/posts\/85283\/revisions"}],"predecessor-version":[{"id":85287,"href":"https:\/\/www.05921.net\/index.php\/wp-json\/wp\/v2\/posts\/85283\/revisions\/85287"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.05921.net\/index.php\/wp-json\/wp\/v2\/media\/85284"}],"wp:attachment":[{"href":"https:\/\/www.05921.net\/index.php\/wp-json\/wp\/v2\/media?parent=85283"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.05921.net\/index.php\/wp-json\/wp\/v2\/categories?post=85283"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.05921.net\/index.php\/wp-json\/wp\/v2\/tags?post=85283"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}