{"id":454193,"date":"2023-03-09T17:58:51","date_gmt":"2023-03-10T01:58:51","guid":{"rendered":"https:\/\/nai500.com\/blog\/2023\/03\/%e9%94%99%e5%a4%b1%e7%a6%8f%e6%b3%b0%e5%88%b6%e8%8d%af%ef%bc%9f%e9%82%a3%e5%b0%b1%e4%b8%8d%e8%a6%81%e5%86%8d%e9%94%99%e8%bf%87%e8%bf%99%e5%8f%aa%e5%9f%ba%e5%9b%a0%e7%bc%96%e8%be%91%e8%82%a1%e7%a5%a8\/"},"modified":"2023-03-09T17:59:12","modified_gmt":"2023-03-10T01:59:12","slug":"%e7%a6%8f%e6%b3%b0%e8%a3%bd%e8%97%a5-%e4%b8%8d%e8%a6%81%e5%86%8d%e9%8c%af%e9%81%8e%e9%80%99%e5%8f%aa%e5%9f%ba%e5%9b%a0%e7%b7%a8%e8%bc%af%e8%82%a1%e7%a5%a8","status":"publish","type":"post","link":"https:\/\/nai500.com\/zh-hant\/blog\/2023\/03\/%e7%a6%8f%e6%b3%b0%e8%a3%bd%e8%97%a5-%e4%b8%8d%e8%a6%81%e5%86%8d%e9%8c%af%e9%81%8e%e9%80%99%e5%8f%aa%e5%9f%ba%e5%9b%a0%e7%b7%a8%e8%bc%af%e8%82%a1%e7%a5%a8\/","title":{"rendered":"\u932f\u5931\u798f\u6cf0\u88fd\u85e5\uff1f\u90a3\u5c31\u4e0d\u8981\u518d\u932f\u904e\u9019\u53ea\u57fa\u56e0\u7de8\u8f2f\u80a1\u7968"},"content":{"rendered":"<h2>CRISPR Therapeutics\u7684\u80a1\u7968\u73fe\u5728\u9084\u4fbf\u5b9c\uff0c\u4f46\u4e0d\u6703\u6c38\u9060\u4fbf\u5b9c\u4e0b\u53bb<\/h2>\n<p>\u904e\u53bb12\u500b\u6708\uff0c\u7f8e\u80a1\u5f88\u591a\u57fa\u56e0\u7de8\u8f2f\u751f\u7269\u79d1\u6280\u80a1\u90fd\u66b4\u8dcc\uff0c\u4f46\u798f\u6cf0\u88fd\u85e5Vertex Pharmaceuticals\uff08NASDAQ:VRTX\uff09\u7684\u80a1\u50f9\u4e0a\u6f32\u8d85\u904e19%\u3002\u539f\u56e0\u4e5f\u5f88\u7c21\u55ae\uff1a\u56ca\u6027\u7e96\u7dad\u5316\uff08CF\uff09\u7642\u6cd5\u52a9\u529b\u8a72\u516c\u53f8\u5be6\u73fe\u76c8\u5229\uff0c\u540c\u6642\u6cbb\u7642\u942e\u72c0\u7d30\u80de\u75c5\uff08SCD\uff09\u548c\u8f38\u8840\u4f9d\u8cf4\u6027\u03b2\u5730\u4e2d\u6d77\u8ca7\u8840\u7684CRISPR\u57fa\u56e0\u7de8\u8f2f\u7642\u6cd5exa-cel\u6b63\u5728\u7b49\u5f85\u7f8e\u570b\u98df\u54c1\u85e5\u54c1\u76e3\u7ba1\u5c40\uff08FDA\uff09\u7684\u6279\u51c6\u3002<\/p>\n<p>\u73fe\u5728\u8ffd\u6f32\u798f\u6cf0\u88fd\u85e5\u53ef\u80fd\u5df2\u7d93\u592a\u665a\u4e86\uff0c\u4f46\u4e0d\u7528\u64d4\u5fc3\uff0c\u53e6\u4e00\u96bbCRISPR\u57fa\u56e0\u7de8\u8f2f\u516c\u53f8\uff0cexa-cel\u7684\u958b\u767c\u5408\u4f5c\u516c\u53f8<a href=\"https:\/\/nai500.com\/zh-hans\/%e8%82%a1%e7%a5%a8%e6%9f%a5%e8%af%a2\/?qm_symbol=CRSP\">CRISPR Therapeutics AG\uff08NASDAQ:CRSP\uff09<\/a>\u7684\u9577\u671f\u6295\u8cc7\u50f9\u503c\u53ef\u80fd\u66f4\u5927\u3002\u9019\u53ea\u57fa\u56e0\u7de8\u8f2f\u80a1\u7968\u7684\u50f9\u683c\u4eca\u5e74\u4e0a\u6f32\u8d85\u904e17%\uff0c\u4f46\u904e\u53bb12\u500b\u6708\u4f9d\u7136\u4e0b\u8dcc\u8d85\u904e20%\u3002<\/p>\n<p>CRISPR\u80a1\u7968\u70ba\u4ec0\u9ebc\u9069\u5408\u9577\u7dda\u6295\u8cc7\uff1f\u6709\u4ee5\u4e0b3\u500b\u7406\u7531\uff1a<\/p>\n<h2>1. exa-cel\u4e00\u65e6\u7372\u6279\uff0cCRISPR\u4e5f\u5c07\u53d7\u76ca<\/h2>\n<p>\u9019\u4e00\u57fa\u56e0\u7de8\u8f2f\u7642\u6cd5\u5982\u679c\u7372\u5f97\u6279\u51c6\uff0cCRISPR\u5c07\u7372\u5f9740%\u7684\u6536\u5165\u5206\u6210\uff0c\u9019\u53cd\u904e\u4f86\u53ef\u4ee5\u70ba\u9019\u5bb6\u81e8\u5e8a\u968e\u6bb5\u7684\u751f\u7269\u79d1\u6280\u516c\u53f8\u5176\u4ed6\u5728\u7814\u85e5\u7269\u7684\u958b\u767c\u63d0\u4f9b\u8cc7\u91d1\u652f\u6301\u3002\u53bb\u5e7412\u6708\uff0cExa-cel\u5728\u6b50\u76df\u548c\u82f1\u570b\u7684\u7533\u8acb\u7372\u5f97\u78ba\u8a8d\uff0c\u798f\u6cf0\u88fd\u85e5\u548cCRISPR\u9810\u8a08\u7f8e\u570b\u7684\u751f\u7269\u88fd\u5291\u8a31\u53ef\u7533\u8acb\uff08BLA\uff09\u5c07\u5728\u9019\u500b\u5b63\u5ea6\u5b8c\u6210\u3002<\/p>\n<p>\u6211\u5011\u4e0d\u80fd\u4fdd\u8b49FDA\u4e00\u5b9a\u6703\u6279\u51c6exa-cel\uff0c\u4f46\u8a72\u57fa\u56e0\u7de8\u8f2f\u7642\u6cd5\u7684\u4e09\u671f\u8a66\u9a57\u2014\u2014\u6cbb\u7642SCD\u7684CLIMB-121\u7814\u7a76\u4ee5\u53ca\u6cbb\u7642TDT\u7684CLIMB-111\u7814\u7a76\u2014\u2014\u7684\u7d50\u679c\u90fd\u975e\u5e38\u4e0d\u932f\u3002\u5b98\u65b9\u6578\u64da\u986f\u793a\uff0c\u7f8e\u570bTDT\u548cSCD\u7684\u60a3\u8005\u4eba\u6578\u5206\u5225\u4e0d\u52305000\u4eba\u548c\u5927\u7d0410\u842c\u4eba\uff0c\u4f46exa-cel\u7684\u5b9a\u50f9\u6216\u5c0d\u6a19Bluebird bio Inc\uff08NASDAQ:BLUE\uff09\u7684TDT\u57fa\u56e0\u7642\u6cd5Zynteglo\uff08$280\u842c\uff09\uff0c\u96d6\u7136\u6602\u8cb4\u4f46\u6709\u7372\u5229\u6f5b\u529b\uff0c\u56e0\u6b64\u4fdd\u96aa\u516c\u53f8\u53ef\u80fd\u9858\u610f\u627f\u4fdd\u3002<\/p>\n<h2>2. 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