{"id":318979,"date":"2020-02-21T19:09:18","date_gmt":"2020-02-22T03:09:18","guid":{"rendered":"https:\/\/nai500.com\/?p=318979"},"modified":"2020-02-21T19:09:18","modified_gmt":"2020-02-22T03:09:18","slug":"crispr-intellia-%e5%9f%ba%e5%9b%a0%e7%bc%96%e8%be%91%e8%82%a1","status":"publish","type":"post","link":"https:\/\/nai500.com\/zh-hans\/blog\/2020\/02\/crispr-intellia-%e5%9f%ba%e5%9b%a0%e7%bc%96%e8%be%91%e8%82%a1\/","title":{"rendered":"CRISPR\u548cIntellia\uff0c\u8fd9\u4e24\u53ea\u57fa\u56e0\u7f16\u8f91\u80a1\u7684\u6295\u8d44\u6f5c\u529b\u5bf9\u6bd4"},"content":{"rendered":"

\u5728\u751f\u7269\u79d1\u6280\u80a1\u5f53\u4e2d\uff0c\u57fa\u56e0\u7f16\u8f91\u80a1\u5c24\u5176\u53d7\u5e02\u573a\u5173\u6ce8\uff0c\u5305\u62ec\u5176\u4e2d\u7684\u9f99\u5934\u80a1CRISPR Therapeutics AG\uff08NASDAQ: CRSP\uff09<\/a>\u548cIntellia Therapeutics Inc\uff08NASDAQ: NTLA\uff09<\/a>\u3002\u8fd9\u4e24\u5bb6\u516c\u53f8\u7684\u5728\u7814\u5019\u9009\u836f\u524d\u666f\u53ef\u671f\uff0c\u5f88\u591a\u751a\u81f3\u6709\u53ef\u80fd\u6210\u4e3a\u91cd\u78c5\u836f\uff0c\u540c\u65f6\u836f\u54c1\u5f00\u53d1\u8d44\u91d1\u5145\u88d5\u3002\u5bf9\u4e8e\u8ffd\u6c42\u9ad8\u98ce\u9669\u9ad8\u6536\u76ca\u7684\u6295\u8d44\u8005\u6765\u8bf4\uff0c\u8fd9\u4e24\u53ea\u4e2a\u80a1\u7684\u6295\u8d44\u6f5c\u529b\u90fd\u5f88\u7a81\u51fa\u3002<\/p>\n

\u4e0d\u8fc7\uff0c\u8fd9\u4e24\u53ea\u57fa\u56e0\u7f16\u8f91\u80a1\u4e5f\u5b58\u5728\u7740\u5dee\u5f02\uff0c\u672c\u6587\u5c06\u4ece\u4ea7\u54c1\u7ba1\u7ebf\u3001\u5546\u4e1a\u5408\u4f5c\u4f19\u4f34\u5173\u7cfb\u4ee5\u53ca\u76c8\u5229\u60c5\u51b5\u8fdb\u884c\u5206\u6790\u3002<\/p>\n

CRISPR\u7684\u4ea7\u54c1\u7ba1\u7ebf<\/h2>\n

CRISPR\u7b97\u5f97\u4e0a\u662f\u57fa\u56e0\u7f16\u8f91\u9886\u57df\u7684\u8001\u724c\u516c\u53f8\u4e86\uff0c\u516c\u53f8\u62e5\u67099\u6b3e\u5019\u9009\u836f\uff0c\u4f46\u4ec5\u67094\u6b3e\u5904\u4e8e\u4e34\u5e8a\u8bd5\u9a8c\u6216\u90bb\u8fd1\u8bd5\u9a8c\u542f\u52a8\u9636\u6bb5\u3002<\/p>\n

\u5728\u8fd9\u4e9b\u5728\u7814\u836f\u7269\u5f53\u4e2d\uff0c\u6700\u6709\u524d\u666f\u7684\u662f\u9570\u72b6\u7ec6\u80de\u75c5\uff08SCD\uff09\u548c\u8f93\u8840\u4f9d\u8d56\u6027\u03b2-\u5730\u4e2d\u6d77\u8d2b\u8840\uff08TDT\uff09\u836f\u7269CTX001\u3002\u7f8e\u56fd\u5927\u7ea6\u540e100,000\u4eba\u60a3\u6709\u9570\u72b6\u7ec6\u80de\u75c5\uff0c\u52a0\u4e0a\u76ee\u524d\u8fd9\u4e24\u79cd\u9057\u4f20\u8840\u6db2\u75c5\u5c1a\u65e0\u6cbb\u7597\u65b9\u6cd5\uff0c\u5982\u679c\u8fd9\u79cd\u57fa\u56e0\u7f16\u8f91\u836f\u7269\u88ab\u8bc1\u660e\u6709\u6548\uff0c\u5e02\u573a\u9700\u6c42\u5c06\u975e\u5e38\u9ad8\u3002<\/p>\n

CRISPR\u7684\u53e6\u59163\u6b3e\u4e34\u5e8a\u9636\u6bb5\u5019\u9009\u836f\u5206\u522b\u4e3a\u764c\u75c7\u514d\u75ab\u6cbb\u7597\u836f\u7269CTX110\u3001CTX120\u548cCTX130\uff0c\u5177\u4f53\u6765\u8bf4\u5c31\u662f\u9488\u5bf9\u665a\u671f\u764c\u75c7\u7684\u5d4c\u5408\u6297\u539f\u53d7\u4f53T\u7ec6\u80de\uff08CAR-T\uff09\u7597\u6cd5\u3002CAR-T\u7597\u6cd5\u901a\u8fc7\u4fee\u9970\u60a3\u8005\u7684\u514d\u75ab\u7ec6\u80de\uff08\u5c24\u5176\u662fT\u7ec6\u80de\uff09\u6765\u63d0\u9ad8\u5b83\u4eec\u6740\u706d\u764c\u7ec6\u80de\u7684\u6548\u7387\uff0c\u4ece\u4e8b\u5b9e\u73b0\u6cbb\u7597\u76ee\u7684\u3002<\/p>\n

\u867d\u7136\u6709\u6548\uff0c\u4f46CAR-T\u7ec6\u80de\u7597\u6cd5\u975e\u5e38\u6602\u8d35\uff0c\u5f00\u652f\u901a\u5e38\u90fd\u5728\u6570\u5341\u4e07\u751a\u81f3\u767e\u4e07\u7f8e\u5143\u7ea7\u522b\u3002CRISPR\u81ea\u5bb6\u7684CAR-T\u836f\u7269\u7684\u4ef7\u683c\u6216\u6709\u53ef\u80fd\u4f4e\u4e8e\u5176\u4ed6\u540c\u7c7b\u836f\u7269\u3002<\/p>\n

Intellia\u7684\u4ea7\u54c1\u7ba1\u7ebf<\/h2>\n

Intellia\u62e5\u67097\u6b3e\u5019\u9009\u836f\uff0c\u4f46\u5747\u5904\u4e8e\u7814\u7a76\u3001\u5019\u9009\u836f\u9009\u62e9\u6216\u4e34\u5e8a\u524d\u6d4b\u8bd5\u9636\u6bb5\u3002\u516c\u53f8\u7684\u9570\u72b6\u7ec6\u80de\u75c5\u5019\u9009\u836f\u5373\u5c06\u8fdb\u5165\u521d\u671f\u4e34\u5e8a\u8bd5\u9a8c\uff0c\u4f46\u9879\u76ee\u5c1a\u65e0\u6b63\u5f0f\u540d\u79f0\uff0c\u5728\u516c\u53f8\u5b98\u7f51\u4e0a\u4ec5\u793a\u5217\u4e3a\u201c\u9570\u72b6\u7ec6\u80de\u75c5\u201d\u836f\u7269\u3002<\/p>\n

\u6b64\u5916\uff0c\u6025\u6027\u9ad3\u7cfb\u767d\u8840\u75c5\uff08AML\uff09\u836f\u7269NTLA-5001\u7684\u4e34\u5e8a\u524d\u9879\u76ee\u4e5f\u53d6\u5f97\u8fdb\u5c55\u3002\u6025\u6027\u9ad3\u7cfb\u767d\u8840\u75c5\u60a3\u8005\u76845\u5e74\u5b58\u6d3b\u7387\u53ea\u670927.4%\uff0c\u5360\u6240\u6709\u6210\u4eba\u767d\u8840\u75c5\u75c5\u4f8b\u6570\u768432%\u3002\u7f8e\u56fd\u98df\u54c1\u836f\u54c1\u76d1\u7763\u7ba1\u7406\u5c40\uff08FDA\uff09\u5df2\u7ecf\u6279\u51c6\u591a\u6b3eAML\u836f\u7269\uff0c\u56e0\u6b64NTLA-5001\u82e5\u4e0a\u5e02\u540e\u8fd8\u5fc5\u987b\u4e0e\u5176\u4ed6\u836f\u7269\u4e89\u593a\u5e02\u573a\u4efd\u989d\u3002<\/p>\n

\u6700\u540e\uff0c\u8f6c\u7532\u72b6\u817a\u7d20\u86cb\u767d\u6dc0\u7c89\u6837\u53d8\u6027\u6cbb\u7597\u836f\u7269NTLA-2001\u76ee\u524d\u4ecd\u5904\u4e8e\u4e34\u5e8a\u524d\u5f00\u53d1\u9636\u6bb5\u3002\u8fd9\u79cd\u75be\u75c5\u7684\u53d1\u75c5\u7387\u53ea\u670910\u4e07\u5206\u4e4b\u4e00\uff0c\u4f46\u76ee\u524d\u5c1a\u65e0\u6cbb\u7597\u65b9\u6cd5\uff0c\u56e0\u6b64\u6f5c\u5728\u9700\u6c42\u9ad8\u3002<\/p>\n

\u8d22\u52a1\u72b6\u51b5<\/h2>\n

\u7531\u4e8eCRISPR\u548cIntellia\u90fd\u662f\u521d\u671f\u751f\u7269\u79d1\u6280\u516c\u53f8\uff0c\u8003\u5bdf\u5e02\u9500\u7387\uff08P\/S\uff09\u7b49\u4f20\u7edf\u8d22\u52a1\u6307\u6807\u610f\u4e49\u4e0d\u5927\uff0c\u9500\u552e\u989d\u4e5f\u4e0d\u662f\u6700\u91cd\u8981\u7684\u6307\u6807\u3002<\/p>\n

\u4e0d\u8fc7\uff0cCRISPR\u7684\u8d22\u52a1\u72b6\u51b5\u4f3c\u4e4e\u8981\u8fdc\u597d\u4e8eIntellia\u3002\u5728\u8be5\u516c\u53f8\u76842019\u5e74\u56db\u5b63\u62a5\u5f53\u4e2d\uff0cCRISPR\u516c\u544a\u5b63\u5ea6\u6536\u5165$7700\u4e07\uff08\u4e3b\u8981\u6765\u81ea\u798f\u6cf0\u5236\u836fVertex Pharmaceuticals\u7684\u5408\u4f5c\u534f\u8bae\u800c\u975e\u4ea7\u54c1\u9500\u552e\uff09\uff0c\u4f46\u8f83\u4e0a\u5e74\u540c\u671f\u7684$100,000\u4ecd\u662f\u5927\u5e45\u4e0a\u5347\u3002\u6b64\u5916\uff0c\u516c\u53f8\u73b0\u91d1\u4e5f\u4ece\u4e0a\u5e74\u7684$4.566\u4ebf\u7ffb\u4e861\u500d\u591a\u81f3$9.438\u4ebf\u3002\u6700\u540e\uff0cCRISPR\u5b9e\u73b0\u4e86\u521d\u671f\u751f\u7269\u79d1\u6280\u516c\u53f8\u5341\u5206\u7f55\u89c1\u7684\u6210\u7ee9\uff1a\u62a5\u544a\u76c8\u5229\u3002\u516c\u53f8\u62a5\u544a\u51c0\u5229\u6da6$3050\u4e07\uff0c\u800c2018\u5e74Q4\u8fd8\u662f\u4e8f\u635f$4760\u4e07\u3002<\/p>\n

\u76f8\u6bd4\u4e4b\u4e0b\uff0cIntellia\u7684\u8d22\u52a1\u72b6\u51b5\u5c31\u8981\u900a\u8272\u591a\u4e86\u3002\u8be5\u516c\u53f8\u516c\u544a\u5408\u4f5c\u6536\u5165$1060\u4e07\uff0c\u8f83\u4e0a\u5e74\u540c\u671f\u7684$740\u4e07\u589e\u957f43%\uff0c\u4f46\u4ecd\u8fdc\u4f4e\u4e8eCRISPR\u3002\u51c0\u4e8f\u635f$2460\u4e07\uff0c\u8ddf\u4e0a\u5e74\u57fa\u672c\u6301\u5e73\uff0c\u73b0\u91d1$2.958\u4ebf\u3002\u5728\u521d\u671f\u7684\u751f\u7269\u79d1\u6280\u80a1\u5f53\u4e2d\uff0c\u8fd9\u5df2\u7ecf\u662f\u76f8\u5f53\u4e0d\u9519\u4e86\u3002\u6309\u7167\u5f53\u524d\u7684\u5f00\u652f\u6c34\u5e73\uff0cIntellia\u7684\u5e93\u5b58\u73b0\u91d1\u8db3\u591f\u652f\u6301\u516c\u53f8\u81f3\u5c113\u5e74\u7684\u5f00\u9500\uff0c\u56e0\u6b64\u6295\u8d44\u8005\u65e0\u9700\u62c5\u5fc3\u8d44\u91d1\u95ee\u9898\u3002<\/p>\n","protected":false},"excerpt":{"rendered":"

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