{"id":454191,"date":"2023-03-09T17:58:51","date_gmt":"2023-03-10T01:58:51","guid":{"rendered":"https:\/\/nai500.com\/?p=454191"},"modified":"2023-03-09T17:58:51","modified_gmt":"2023-03-10T01:58:51","slug":"%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","status":"publish","type":"post","link":"https:\/\/nai500.com\/zh-hans\/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\/","title":{"rendered":"\u9519\u5931\u798f\u6cf0\u5236\u836f\uff1f\u90a3\u5c31\u4e0d\u8981\u518d\u9519\u8fc7\u8fd9\u53ea\u57fa\u56e0\u7f16\u8f91\u80a1\u7968"},"content":{"rendered":"<h2>CRISPR Therapeutics\u7684\u80a1\u7968\u73b0\u5728\u8fd8\u4fbf\u5b9c\uff0c\u4f46\u4e0d\u4f1a\u6c38\u8fdc\u4fbf\u5b9c\u4e0b\u53bb<\/h2>\n<p>\u8fc7\u53bb12\u4e2a\u6708\uff0c\u7f8e\u80a1\u5f88\u591a\u57fa\u56e0\u7f16\u8f91\u751f\u7269\u79d1\u6280\u80a1\u90fd\u66b4\u8dcc\uff0c\u4f46\u798f\u6cf0\u5236\u836fVertex Pharmaceuticals\uff08NASDAQ:VRTX\uff09\u7684\u80a1\u4ef7\u4e0a\u6da8\u8d85\u8fc719%\u3002\u539f\u56e0\u4e5f\u5f88\u7b80\u5355\uff1a\u56ca\u6027\u7ea4\u7ef4\u5316\uff08CF\uff09\u7597\u6cd5\u52a9\u529b\u8be5\u516c\u53f8\u5b9e\u73b0\u76c8\u5229\uff0c\u540c\u65f6\u6cbb\u7597\u9570\u72b6\u7ec6\u80de\u75c5\uff08SCD\uff09\u548c\u8f93\u8840\u4f9d\u8d56\u6027\u03b2\u5730\u4e2d\u6d77\u8d2b\u8840\u7684CRISPR\u57fa\u56e0\u7f16\u8f91\u7597\u6cd5exa-cel\u6b63\u5728\u7b49\u5f85\u7f8e\u56fd\u98df\u54c1\u836f\u54c1\u76d1\u7ba1\u5c40\uff08FDA\uff09\u7684\u6279\u51c6\u3002<\/p>\n<p>\u73b0\u5728\u8ffd\u6da8\u798f\u6cf0\u5236\u836f\u53ef\u80fd\u5df2\u7ecf\u592a\u665a\u4e86\uff0c\u4f46\u4e0d\u7528\u62c5\u5fc3\uff0c\u53e6\u4e00\u53eaCRISPR\u57fa\u56e0\u7f16\u8f91\u516c\u53f8\uff0cexa-cel\u7684\u5f00\u53d1\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\u957f\u671f\u6295\u8d44\u4ef7\u503c\u53ef\u80fd\u66f4\u5927\u3002\u8fd9\u53ea\u57fa\u56e0\u7f16\u8f91\u80a1\u7968\u7684\u4ef7\u683c\u4eca\u5e74\u4e0a\u6da8\u8d85\u8fc717%\uff0c\u4f46\u8fc7\u53bb12\u4e2a\u6708\u4f9d\u7136\u4e0b\u8dcc\u8d85\u8fc720%\u3002<\/p>\n<p>CRISPR\u80a1\u7968\u4e3a\u4ec0\u4e48\u9002\u5408\u957f\u7ebf\u6295\u8d44\uff1f\u6709\u4ee5\u4e0b3\u4e2a\u7406\u7531\uff1a<\/p>\n<h2>1. exa-cel\u4e00\u65e6\u83b7\u6279\uff0cCRISPR\u4e5f\u5c06\u53d7\u76ca<\/h2>\n<p>\u8fd9\u4e00\u57fa\u56e0\u7f16\u8f91\u7597\u6cd5\u5982\u679c\u83b7\u5f97\u6279\u51c6\uff0cCRISPR\u5c06\u83b7\u5f9740%\u7684\u6536\u5165\u5206\u6210\uff0c\u8fd9\u53cd\u8fc7\u6765\u53ef\u4ee5\u4e3a\u8fd9\u5bb6\u4e34\u5e8a\u9636\u6bb5\u7684\u751f\u7269\u79d1\u6280\u516c\u53f8\u5176\u4ed6\u5728\u7814\u836f\u7269\u7684\u5f00\u53d1\u63d0\u4f9b\u8d44\u91d1\u652f\u6301\u3002\u53bb\u5e7412\u6708\uff0cExa-cel\u5728\u6b27\u76df\u548c\u82f1\u56fd\u7684\u7533\u8bf7\u83b7\u5f97\u786e\u8ba4\uff0c\u798f\u6cf0\u5236\u836f\u548cCRISPR\u9884\u8ba1\u7f8e\u56fd\u7684\u751f\u7269\u5236\u5242\u8bb8\u53ef\u7533\u8bf7\uff08BLA\uff09\u5c06\u5728\u8fd9\u4e2a\u5b63\u5ea6\u5b8c\u6210\u3002<\/p>\n<p>\u6211\u4eec\u4e0d\u80fd\u4fdd\u8bc1FDA\u4e00\u5b9a\u4f1a\u6279\u51c6exa-cel\uff0c\u4f46\u8be5\u57fa\u56e0\u7f16\u8f91\u7597\u6cd5\u7684\u4e09\u671f\u8bd5\u9a8c\u2014\u2014\u6cbb\u7597SCD\u7684CLIMB-121\u7814\u7a76\u4ee5\u53ca\u6cbb\u7597TDT\u7684CLIMB-111\u7814\u7a76\u2014\u2014\u7684\u7ed3\u679c\u90fd\u975e\u5e38\u4e0d\u9519\u3002\u5b98\u65b9\u6570\u636e\u663e\u793a\uff0c\u7f8e\u56fdTDT\u548cSCD\u7684\u60a3\u8005\u4eba\u6570\u5206\u522b\u4e0d\u52305000\u4eba\u548c\u5927\u7ea610\u4e07\u4eba\uff0c\u4f46exa-cel\u7684\u5b9a\u4ef7\u6216\u5bf9\u6807Bluebird bio Inc\uff08NASDAQ:BLUE\uff09\u7684TDT\u57fa\u56e0\u7597\u6cd5Zynteglo\uff08$280\u4e07\uff09\uff0c\u867d\u7136\u6602\u8d35\u4f46\u6709\u83b7\u5229\u6f5c\u529b\uff0c\u56e0\u6b64\u4fdd\u9669\u516c\u53f8\u53ef\u80fd\u613f\u610f\u627f\u4fdd\u3002<\/p>\n<h2>2. 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