{"id":567174,"date":"2025-02-24T23:56:06","date_gmt":"2025-02-25T07:56:06","guid":{"rendered":"https:\/\/nai500.com\/?p=567174"},"modified":"2025-02-24T23:56:06","modified_gmt":"2025-02-25T07:56:06","slug":"%e5%8d%8e%e5%b0%94%e8%a1%97%e8%ae%a4%e4%b8%ba%e8%bf%99%e4%b8%a4%e5%8f%aa%e8%82%a1%e7%a5%a8%e8%a2%ab%e4%bd%8e%e4%bc%b0%e5%88%86%e5%88%ab%e6%9c%89%e6%9c%9b%e9%a3%99%e5%8d%87-66-%e5%92%8c-415","status":"publish","type":"post","link":"https:\/\/nai500.com\/zh-hans\/blog\/2025\/02\/%e5%8d%8e%e5%b0%94%e8%a1%97%e8%ae%a4%e4%b8%ba%e8%bf%99%e4%b8%a4%e5%8f%aa%e8%82%a1%e7%a5%a8%e8%a2%ab%e4%bd%8e%e4%bc%b0%e5%88%86%e5%88%ab%e6%9c%89%e6%9c%9b%e9%a3%99%e5%8d%87-66-%e5%92%8c-415\/","title":{"rendered":"\u534e\u5c14\u8857\u8ba4\u4e3a\u8fd9\u4e24\u53ea\u80a1\u7968\u88ab\u4f4e\u4f30\uff0c\u5206\u522b\u6709\u671b\u98d9\u534766%\u548c415%"},"content":{"rendered":"

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\u4ee5CRISPR Therapeutics\uff08CRSP\uff09<\/a>\u548cIntellia Therapeutics\uff08NTLA\uff09<\/a>\u4e3a\u4f8b\uff0c\u8fd9\u662f\u4e24\u5bb6\u4e13\u6ce8\u4e8e\u57fa\u56e0\u7f16\u8f91\u7684\u751f\u7269\u79d1\u6280\u516c\u53f8\u3002\u6839\u636e\u6570\u636e\uff0c\u524d\u8005\u7684\u5e73\u5747\u76ee\u6807\u4ef7\u4e3a83.60\u7f8e\u5143\uff0c\u540e\u8005\u7684\u76ee\u6807\u4ef7\u4e3a50.80\u7f8e\u5143\u3002\u8fd9\u610f\u5473\u7740CRISPR Therapeutics\u6709\u7ea666%\u7684\u4e0a\u6da8\u7a7a\u95f4\uff0c\u800cIntellia Therapeutics\u7684\u4e0a\u6da8\u7a7a\u95f4\u9ad8\u8fbe415%\u3002<\/p>\n

\u8fc7\u53bb\u4e09\u5e74\uff0c\u8fd9\u4e24\u5bb6\u516c\u53f8\u7684\u8868\u73b0\u660e\u663e\u843d\u540e\u4e8e\u5e02\u573a\u3002\u5b83\u4eec\u80fd\u5426\u626d\u8f6c\u5c40\u9762\uff0c\u8fbe\u5230\u534e\u5c14\u8857\u7684\u9884\u671f\u5462\uff1f<\/p>\n

CRISPR Therapeutics<\/h2>\n

CRISPR Therapeutics\u5b8c\u6210\u4e86\u4e00\u9879\u8bb8\u591a\u7eaf\u57fa\u56e0\u7f16\u8f91\u516c\u53f8\u5c1a\u672a\u5b9e\u73b0\u7684\u6210\u5c31\uff1a\u83b7\u5f97\u4e86\u4e00\u79cd\u7597\u6cd5\u7684\u76d1\u7ba1\u6279\u51c6\u3002\u8be5\u516c\u53f8\u4e0eVertex Pharmaceuticals\u5408\u4f5c\u5f00\u53d1\u7684Casgevy\uff0c\u7528\u4e8e\u6cbb\u7597\u9570\u72b6\u7ec6\u80de\u75c5\uff08SCD\uff09\u548c\u8f93\u8840\u4f9d\u8d56\u6027\u03b2-\u5730\u4e2d\u6d77\u8d2b\u8840\uff08TDT\uff09\uff0c\u5df2\u5728\u7f8e\u56fd\u3001\u52a0\u62ff\u5927\u3001\u82f1\u56fd\u3001\u6b27\u76df\u3001\u6c99\u7279\u963f\u62c9\u4f2f\u548c\u5df4\u6797\u83b7\u5f97\u6279\u51c6\u3002<\/p>\n

\u5c3d\u7ba1\u53d6\u5f97\u4e86\u8fd9\u4e00\u91cd\u5927\u6210\u5c31\uff0c\u8be5\u516c\u53f8\u7684\u80a1\u4ef7\u5374\u4e00\u76f4\u5728\u4e0b\u8dcc\u3002CRISPR Therapeutics\u4ecd\u7136\u5904\u4e8e\u4e8f\u635f\u72b6\u6001\uff0cCasgevy\u5c1a\u672a\u8d21\u732e\u592a\u591a\u6536\u5165\u3002\u8be5\u516c\u53f8\u80fd\u5426\u5728\u672a\u6765\u4e00\u5e74\u626d\u8f6c\u5c40\u9762\uff0c\u8fbe\u5230\u534e\u5c14\u8857\u7684\u5e73\u5747\u76ee\u6807\u4ef7\uff1f\u8fd9\u53ef\u80fd\u5f97\u76ca\u4e8e\u66f4\u5f3a\u52b2\u7684\u8d22\u52a1\u4e1a\u7ee9\u6216\u4e34\u5e8a\u8fdb\u5c55\u3002<\/p>\n

\u8be5\u516c\u53f8\u53ef\u80fd\u4f1a\u5728\u4e34\u5e8a\u8fdb\u5c55\u65b9\u9762\u53d6\u5f97\u4ee4\u4eba\u5174\u594b\u7684\u6210\u679c\uff0c\u5c24\u5176\u662fCTX112\uff0c\u8fd9\u662f\u4e00\u79cd\u9488\u5bf9\u67d0\u4e9b\u6076\u6027\u80bf\u7624\u7684\u6f5c\u5728\u57fa\u56e0\u7f16\u8f91\u7597\u6cd5\u3002CRISPR Therapeutics\u5c06\u5728\u660e\u5e74\u62a5\u544aCTX112\u6b63\u5728\u8fdb\u884c\u7684\u4e00\u4e9b\u4e34\u5e8a\u8bd5\u9a8c\u6570\u636e\uff0c\u4f46\u5373\u4f7f\u53d6\u5f97\u5f3a\u52b2\u8fdb\u5c55\uff0c\u80a1\u4ef7\u4e5f\u4e0d\u592a\u53ef\u80fd\u7ffb\u500d\u3002<\/p>\n

\u5c3d\u7ba1\u5b58\u5728\u4e00\u5b9a\u98ce\u9669\uff0c\u5206\u6790\u5e08\u5efa\u8bae\u5bf9\u6ce2\u52a8\u6027\u6709\u8f83\u9ad8\u627f\u53d7\u80fd\u529b\u7684\u6295\u8d44\u8005\u8003\u8651\u5c11\u91cf\u5efa\u4ed3CRISPR Therapeutics\u80a1\u7968\u3002<\/p>\n

Intellia Therapeutics<\/h2>\n

Intellia Therapeutics\u76ee\u524d\u6ca1\u6709\u4efb\u4f55\u836f\u7269\u4e0a\u5e02\u3002\u5c3d\u7ba1\u8fc7\u53bb\u51e0\u5e74\u53d6\u5f97\u4e86\u4e00\u4e9b\u4e34\u5e8a\u8fdb\u5c55\uff0c\u4f46\u8fd9\u8fd8\u4e0d\u8db3\u4ee5\u5438\u5f15\u6295\u8d44\u8005\u3002\u4e0d\u8fc7\uff0c\u8be5\u516c\u53f8\u786e\u5b9e\u6709\u4e00\u4e9b\u6f5c\u5728\u7684\u50ac\u5316\u5242\uff0c\u534e\u5c14\u8857\u8ba4\u4e3a\u8fd9\u4e9b\u56e0\u7d20\u53ef\u80fd\u63a8\u52a8\u80a1\u4ef7\u98d9\u5347\u3002<\/p>\n

\u8be5\u516c\u53f8\u7684\u4e24\u4e2a\u4e3b\u8981\u5019\u9009\u836f\u7269\u662fNTLA-2002\u548cnexiguran ziclumeran\uff08\u539f\u540dNTLA-2001\uff09\uff0c\u73b0\u5728\u975e\u6b63\u5f0f\u540d\u79f0\u4e3a\u201cnex-z\u201d\u3002NTLA-2002\u662f\u4e00\u79cd\u6f5c\u5728\u7684\u6cbb\u7597\u9057\u4f20\u6027\u8840\u7ba1\u6027\u6c34\u80bf\u7684\u836f\u7269\uff0c\u8fd9\u662f\u4e00\u79cd\u5bfc\u81f4\u56db\u80a2\u80bf\u80c0\u7684\u7f55\u89c1\u75be\u75c5\u3002Nex-z\u5219\u9488\u5bf9\u8f6c\u7532\u72b6\u817a\u7d20\u86cb\u767d\uff08ATTR\uff09\u6dc0\u7c89\u6837\u53d8\u6027\uff0c\u8fd9\u662f\u4e00\u79cd\u7531\u4f53\u5185\u5668\u5b98\u4e2d\u86cb\u767d\u8d28\u5371\u9669\u5806\u79ef\u5f15\u8d77\u7684\u75be\u75c5\u3002\u8fd9\u4e24\u4e2a\u5019\u9009\u836f\u7269\u53ef\u80fd\u4f1a\u5728\u672a\u6765\u4e00\u5e74\u5de6\u53f3\u53d1\u5e03\u53ef\u9760\u7684\u540e\u671f\u4e34\u5e8a\u6570\u636e\u3002<\/p>\n

\u503c\u5f97\u4e00\u63d0\u7684\u662f\uff0cIntellia\u6b63\u5728\u4e0e\u518d\u751f\u5143\u5236\u836f\u5408\u4f5c\u5f00\u53d1nex-z\u3002Intellia\u4f30\u8ba1\uff0c\u52302028\u5e74\uff0cnex-z\u7684\u6f5c\u5728\u5e02\u573a\u4ef7\u503c\u53ef\u80fd\u8fbe\u5230120\u4ebf\u7f8e\u5143\uff0c\u800cNTLA-2002\u7684\u5e02\u573a\u4ef7\u503c\u53ef\u80fd\u7ea6\u4e3a50\u4ebf\u7f8e\u5143\u3002<\/p>\n

\u5206\u6790\u5e08\u8ba4\u4e3a\uff0c\u5373\u4f7f\u8be5\u516c\u53f8\u5728nex-z\u548cNTLA-2002\u7684\u540e\u671f\u4e34\u5e8a\u8bd5\u9a8c\u4e2d\u8868\u73b0\u51fa\u8272\uff0c\u80a1\u4ef7\u5f88\u96be\u4e0a\u6da8\u8d85\u8fc7400%\u3002\u8003\u8651\u5230Intellia Therapeutics\u76ee\u524d\u4ecd\u6ca1\u6709\u4efb\u4f55\u4ea7\u54c1\u4e0a\u5e02\uff0c\u5bf9\u5927\u591a\u6570\u6295\u8d44\u8005\u6765\u8bf4\uff0c\u8be5\u80a1\u7684\u98ce\u9669\u4f3c\u4e4e\u592a\u9ad8\u3002<\/p>\n","protected":false},"excerpt":{"rendered":"

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