{"id":567178,"date":"2025-02-24T23:56:06","date_gmt":"2025-02-25T07:56:06","guid":{"rendered":"https:\/\/nai500.com\/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\/"},"modified":"2025-02-24T23:56:44","modified_gmt":"2025-02-25T07:56:44","slug":"%e8%8f%af%e7%88%be%e8%a1%97%e8%aa%8d%e7%82%ba%e9%80%99%e5%85%a9%e9%9a%bb%e8%82%a1%e7%a5%a8%e8%a2%ab%e4%bd%8e%e4%bc%b0%e5%88%86%e5%88%a5%e6%9c%89%e6%9c%9b%e9%a3%86%e5%8d%87-66-%e5%92%8c-415","status":"publish","type":"post","link":"https:\/\/nai500.com\/zh-hant\/blog\/2025\/02\/%e8%8f%af%e7%88%be%e8%a1%97%e8%aa%8d%e7%82%ba%e9%80%99%e5%85%a9%e9%9a%bb%e8%82%a1%e7%a5%a8%e8%a2%ab%e4%bd%8e%e4%bc%b0%e5%88%86%e5%88%a5%e6%9c%89%e6%9c%9b%e9%a3%86%e5%8d%87-66-%e5%92%8c-415\/","title":{"rendered":"\u83ef\u723e\u8857\u8a8d\u70ba\u9019\u5169\u96bb\u80a1\u7968\u88ab\u4f4e\u4f30\uff0c\u5206\u5225\u6709\u671b\u98c6\u534766%\u548c415%"},"content":{"rendered":"

\u751f\u7269\u79d1\u6280\u516c\u53f8\u80a1\u50f9\u53ef\u80fd\u56e0\u51fa\u8272\u7684\u81e8\u5e8a\u6216\u76e3\u7ba1\u9032\u5c55\u800c\u5728\u77ed\u671f\u5167\u98c6\u5347\u3002\u5982\u679c\u6211\u5011\u76f8\u4fe1\u83ef\u723e\u8857\u5206\u6790\u5e2b\u7684\u8a71\uff0c\u5176\u4e2d\u4e00\u4e9b\u516c\u53f8\u5c07\u5728\u660e\u5e74\u5be6\u73fe\u9019\u4e00\u76ee\u6a19\u3002<\/p>\n

\u4ee5CRISPR Therapeutics\uff08CRSP\uff09<\/a>\u548cIntellia Therapeutics\uff08NTLA\uff09<\/a>\u70ba\u4f8b\uff0c\u9019\u662f\u5169\u5bb6\u5c08\u6ce8\u4e8e\u57fa\u56e0\u7de8\u8f2f\u7684\u751f\u7269\u79d1\u6280\u516c\u53f8\u3002\u6839\u64da\u6578\u64da\uff0c\u524d\u8005\u7684\u5e73\u5747\u76ee\u6a19\u50f9\u70ba83.60\u7f8e\u5143\uff0c\u5f8c\u8005\u7684\u76ee\u6a19\u50f9\u70ba50.80\u7f8e\u5143\u3002\u9019\u610f\u5473\u8457CRISPR Therapeutics\u6709\u7d0466%\u7684\u4e0a\u6f32\u7a7a\u9593\uff0c\u800cIntellia Therapeutics\u7684\u4e0a\u6f32\u7a7a\u9593\u9ad8\u9054415%\u3002<\/p>\n

\u904e\u53bb\u4e09\u5e74\uff0c\u9019\u5169\u5bb6\u516c\u53f8\u7684\u8868\u73fe\u660e\u986f\u843d\u5f8c\u65bc\u5e02\u5834\u3002\u5b83\u5011\u80fd\u5426\u626d\u8f49\u5c40\u9762\uff0c\u9054\u5230\u83ef\u723e\u8857\u7684\u9810\u671f\u5462\uff1f<\/p>\n

CRISPR Therapeutics<\/h2>\n

CRISPR Therapeutics\u5b8c\u6210\u4e86\u4e00\u9805\u8a31\u591a\u7d14\u57fa\u56e0\u7de8\u8f2f\u516c\u53f8\u5c1a\u672a\u5be6\u73fe\u7684\u6210\u5c31\uff1a\u7372\u5f97\u4e86\u4e00\u7a2e\u7642\u6cd5\u7684\u76e3\u7ba1\u6279\u51c6\u3002\u8a72\u516c\u53f8\u8207Vertex Pharmaceuticals\u5408\u4f5c\u958b\u767c\u7684Casgevy\uff0c\u7528\u65bc\u6cbb\u7642\u942e\u72c0\u7d30\u80de\u75c5\uff08SCD\uff09\u548c\u8f38\u8840\u4f9d\u8cf4\u6027\u03b2-\u5730\u4e2d\u6d77\u8ca7\u8840\uff08TDT\uff09\uff0c\u5df2\u5728\u7f8e\u570b\u3001\u52a0\u62ff\u5927\u3001\u82f1\u570b\u3001\u6b50\u76df\u3001\u6c99\u7279\u963f\u62c9\u4f2f\u548c\u5df4\u6797\u7372\u5f97\u6279\u51c6\u3002<\/p>\n

\u5118\u7ba1\u53d6\u5f97\u4e86\u9019\u4e00\u91cd\u5927\u6210\u5c31\uff0c\u8a72\u516c\u53f8\u7684\u80a1\u50f9\u537b\u4e00\u76f4\u5728\u4e0b\u8dcc\u3002CRISPR Therapeutics\u4ecd\u7136\u8655\u65bc\u8667\u640d\u72c0\u614b\uff0cCasgevy\u5c1a\u672a\u8ca2\u737b\u592a\u591a\u6536\u5165\u3002\u8a72\u516c\u53f8\u80fd\u5426\u5728\u672a\u4f86\u4e00\u5e74\u626d\u8f49\u5c40\u9762\uff0c\u9054\u5230\u83ef\u723e\u8857\u7684\u5e73\u5747\u76ee\u6a19\u50f9\uff1f\u9019\u53ef\u80fd\u5f97\u76ca\u65bc\u66f4\u5f37\u52c1\u7684\u8ca1\u52d9\u696d\u7e3e\u6216\u81e8\u5e8a\u9032\u5c55\u3002<\/p>\n

\u8a72\u516c\u53f8\u53ef\u80fd\u6703\u5728\u81e8\u5e8a\u9032\u5c55\u65b9\u9762\u53d6\u5f97\u4ee4\u4eba\u8208\u596e\u7684\u6210\u679c\uff0c\u5c24\u5176\u662fCTX112\uff0c\u9019\u662f\u4e00\u7a2e\u91dd\u5c0d\u67d0\u4e9b\u60e1\u6027\u816b\u7624\u7684\u6f5b\u5728\u57fa\u56e0\u7de8\u8f2f\u7642\u6cd5\u3002CRISPR Therapeutics\u5c07\u5728\u660e\u5e74\u5831\u544aCTX112\u6b63\u5728\u9032\u884c\u7684\u4e00\u4e9b\u81e8\u5e8a\u8a66\u9a57\u6578\u64da\uff0c\u4f46\u5373\u4f7f\u53d6\u5f97\u5f37\u52c1\u9032\u5c55\uff0c\u80a1\u50f9\u4e5f\u4e0d\u592a\u53ef\u80fd\u7ffb\u500d\u3002<\/p>\n

\u5118\u7ba1\u5b58\u5728\u4e00\u5b9a\u98a8\u96aa\uff0c\u5206\u6790\u5e2b\u5efa\u8b70\u5c0d\u6ce2\u52d5\u6027\u6709\u8f03\u9ad8\u627f\u53d7\u80fd\u529b\u7684\u6295\u8cc7\u8005\u8003\u616e\u5c11\u91cf\u5efa\u5009CRISPR Therapeutics\u80a1\u7968\u3002<\/p>\n

Intellia Therapeutics<\/h2>\n

Intellia Therapeutics\u76ee\u524d\u6c92\u6709\u4efb\u4f55\u85e5\u7269\u4e0a\u5e02\u3002\u5118\u7ba1\u904e\u53bb\u5e7e\u5e74\u53d6\u5f97\u4e86\u4e00\u4e9b\u81e8\u5e8a\u9032\u5c55\uff0c\u4f46\u9019\u9084\u4e0d\u8db3\u4ee5\u5438\u5f15\u6295\u8cc7\u8005\u3002\u4e0d\u904e\uff0c\u8a72\u516c\u53f8\u78ba\u5be6\u6709\u4e00\u4e9b\u6f5b\u5728\u7684\u50ac\u5316\u5291\uff0c\u83ef\u723e\u8857\u8a8d\u70ba\u9019\u4e9b\u56e0\u7d20\u53ef\u80fd\u63a8\u52d5\u80a1\u50f9\u98c6\u5347\u3002<\/p>\n

\u8a72\u516c\u53f8\u7684\u5169\u500b\u4e3b\u8981\u5019\u9078\u85e5\u7269\u662fNTLA-2002\u548cnexiguran ziclumeran\uff08\u539f\u540dNTLA-2001\uff09\uff0c\u73fe\u5728\u975e\u6b63\u5f0f\u540d\u7a31\u70ba\u201cnex-z\u201d\u3002NTLA-2002\u662f\u4e00\u7a2e\u6f5b\u5728\u7684\u6cbb\u7642\u907a\u50b3\u6027\u8840\u7ba1\u6027\u6c34\u816b\u7684\u85e5\u7269\uff0c\u9019\u662f\u4e00\u7a2e\u5c0e\u81f4\u56db\u80a2\u816b\u8139\u7684\u7f55\u898b\u75be\u75c5\u3002Nex-z\u5247\u91dd\u5c0d\u8f49\u7532\u72c0\u817a\u7d20\u86cb\u767d\uff08ATTR\uff09\u6fb1\u7c89\u6a23\u8b8a\u6027\uff0c\u9019\u662f\u4e00\u7a2e\u7531\u9ad4\u5167\u5668\u5b98\u4e2d\u86cb\u767d\u8cea\u5371\u96aa\u5806\u7a4d\u5f15\u8d77\u7684\u75be\u75c5\u3002\u9019\u5169\u500b\u5019\u9078\u85e5\u7269\u53ef\u80fd\u6703\u5728\u672a\u4f86\u4e00\u5e74\u5de6\u53f3\u767c\u4f48\u53ef\u9760\u7684\u5f8c\u671f\u81e8\u5e8a\u6578\u64da\u3002<\/p>\n

\u503c\u5f97\u4e00\u63d0\u7684\u662f\uff0cIntellia\u6b63\u5728\u8207\u518d\u751f\u5143\u88fd\u85e5\u5408\u4f5c\u958b\u767cnex-z\u3002Intellia\u4f30\u8a08\uff0c\u52302028\u5e74\uff0cnex-z\u7684\u6f5b\u5728\u5e02\u5834\u50f9\u503c\u53ef\u80fd\u9054\u5230120\u5104\u7f8e\u5143\uff0c\u800cNTLA-2002\u7684\u5e02\u5834\u50f9\u503c\u53ef\u80fd\u7d04\u70ba50\u5104\u7f8e\u5143\u3002<\/p>\n

\u5206\u6790\u5e2b\u8a8d\u70ba\uff0c\u5373\u4f7f\u8a72\u516c\u53f8\u5728nex-z\u548cNTLA-2002\u7684\u5f8c\u671f\u81e8\u5e8a\u8a66\u9a57\u4e2d\u8868\u73fe\u51fa\u8272\uff0c\u80a1\u50f9\u5f88\u96e3\u4e0a\u6f32\u8d85\u904e400%\u3002\u8003\u616e\u5230Intellia Therapeutics\u76ee\u524d\u4ecd\u6c92\u6709\u4efb\u4f55\u7522\u54c1\u4e0a\u5e02\uff0c\u5c0d\u5927\u591a\u6578\u6295\u8cc7\u8005\u4f86\u8aaa\uff0c\u8a72\u80a1\u7684\u98a8\u96aa\u4f3c\u4e4e\u592a\u9ad8\u3002<\/p>\n","protected":false},"excerpt":{"rendered":"

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