{"id":464032,"date":"2023-06-15T01:11:23","date_gmt":"2023-06-15T08:11:23","guid":{"rendered":"https:\/\/nai500.com\/?p=464032"},"modified":"2023-06-15T01:11:23","modified_gmt":"2023-06-15T08:11:23","slug":"%e8%bf%99%e5%8f%aa%e6%8c%a3%e6%89%8e%e4%b8%ad%e7%9a%84%e7%94%9f%e7%89%a9%e7%a7%91%e6%8a%80%e8%82%a1%e8%bf%98%e8%83%bd%e6%89%93%e7%bf%bb%e8%ba%ab%e6%88%98%e5%90%97","status":"publish","type":"post","link":"https:\/\/nai500.com\/zh-hans\/blog\/2023\/06\/%e8%bf%99%e5%8f%aa%e6%8c%a3%e6%89%8e%e4%b8%ad%e7%9a%84%e7%94%9f%e7%89%a9%e7%a7%91%e6%8a%80%e8%82%a1%e8%bf%98%e8%83%bd%e6%89%93%e7%bf%bb%e8%ba%ab%e6%88%98%e5%90%97\/","title":{"rendered":"\u80a1\u4ef7\u4e24\u5e74\u72c2\u8dcc\u8fd1\u4e09\u5206\u4e4b\u4e8c\uff0c\u8fd9\u53ea\u6323\u624e\u4e2d\u7684\u751f\u7269\u79d1\u6280\u80a1\u8fd8\u80fd\u6253\u7ffb\u8eab\u6218\u5417\uff1f"},"content":{"rendered":"<ul>\n<li>CRISPR Therapeutics\u80a1\u7968\u4ea4\u6613\u4ef7\u683c\u4e0d\u5230\u4e24\u5e74\u524d\u7684\u4e09\u5206\u4e4b\u4e00\u3002<\/li>\n<li>\u8be5\u516c\u53f8\u7684\u5148\u5bfc\u7597\u6cd5\u53ef\u80fd\u4f1a\u5728\u4eca\u5e74\u665a\u4e9b\u65f6\u5019\u6216\u660e\u5e74\u521d\u83b7\u5f97FDA\u7684\u6279\u51c6\u3002<\/li>\n<li>\u622a\u81f3\u7b2c\u4e00\u8d22\u5b63\u672b\uff0c\u8be5\u516c\u53f8\u62e5\u670918.9\u4ebf\u7f8e\u5143\u7684\u73b0\u91d1\u3002<\/li>\n<\/ul>\n<p>\u751f\u7269\u79d1\u6280\u662f\u57fa\u56e0\u7f16\u8f91\u7597\u6cd5\u9769\u547d\u7684\u4e00\u90e8\u5206\u3002\u6295\u8d44\u8005\u7684\u70ed\u60c5\u4f1a\u8d77\u4f0f\u4e0d\u5b9a\uff0c\u6709\u65f6\u4f1a\u5267\u70c8\u8d77\u4f0f\u3002\u4ee5<a href=\"https:\/\/nai500.com\/zh-hans\/%e8%82%a1%e7%a5%a8%e6%9f%a5%e8%af%a2\/?qm_symbol=CRSP\">CRISPR Therapeutics (CRSP)<\/a>\u4e3a\u4f8b\uff0c\u8be5\u516c\u53f8\u7684\u80a1\u4ef7\u7ea6\u4e3a\u6bcf\u80a161\u7f8e\u5143\uff0c\u4f4e\u4e8e2021\u5e741\u670815\u65e5\u89e6\u53ca\u7684\u9ad8\u70b9220.20\u7f8e\u5143\u3002<\/p>\n<p>\u4f46\u662f\u8fd8\u6709\u5e0c\u671b\u3002\u8be5\u516c\u53f8\u5373\u5c06\u83b7\u5f97\u7f8e\u56fd\u98df\u54c1\u836f\u54c1\u76d1\u7763\u7ba1\u7406\u5c40(FDA)\u6279\u51c6\u7684\u9996\u4e2a\u7597\u6cd5\u3002\u8be5\u7597\u6cd5exa-cel\u7531CRISPR\u548c<a href=\"https:\/\/nai500.com\/zh-hans\/%e8%82%a1%e7%a5%a8%e6%9f%a5%e8%af%a2\/?qm_symbol=VRTX\">Vertex Pharmaceuticals<\/a>\u5f00\u53d1\uff0c\u4f5c\u4e3a\u8f93\u8840\u4f9d\u8d56\u6027\u03b2\u5730\u4e2d\u6d77\u8d2b\u8840(TDT)\u548c\u9570\u72b6\u7ec6\u80de\u75c5(SCD)\u7684\u4e00\u6b21\u6027\u6cbb\u7597\u65b9\u6cd5\u3002<\/p>\n<p>\u5341\u5e74\u540e\uff0c\u5982\u679c\u8be5\u516c\u53f8\u5b9e\u73b0\u4ece\u4e34\u5e8a\u9636\u6bb5\u751f\u7269\u79d1\u6280\u516c\u53f8\u5230\u5546\u4e1a\u5316\u7684\u98de\u8dc3\uff0c\u80a1\u7968\u4ea4\u6613\u4ef7\u683c\u53ef\u80fd\u9ad8\u8fbe\u6bcf\u80a1300\u7f8e\u5143\u3002\u4ee5\u4e0b\u662f\u539f\u56e0\u3002<\/p>\n<p>Exa-cel\u57282\/3\u671f\u8bd5\u9a8c\u4e2d\u8868\u73b0\u51fa\u8272\u3002\u8be5\u7597\u6cd5\u91c7\u7528\u60a3\u8005\u81ea\u8eab\u7684\u9020\u8840\u5e72\u7ec6\u80de\uff0c\u7136\u540e\u5bf9\u5176\u8fdb\u884c\u7f16\u8f91\u4ee5\u5236\u9020\u9ad8\u6c34\u5e73\u7684\u80ce\u513f\u8840\u7ea2\u86cb\u767d\u7ea2\u7ec6\u80de\uff0c\u4ece\u6839\u672c\u4e0a\u6539\u9020\u8eab\u4f53\u4ee5\u7ed3\u675f\u9057\u4f20\u7f3a\u9677\u3002\u6839\u636e\u56fd\u5bb6\u8f6c\u5316\u79d1\u5b66\u63a8\u8fdb\u4e2d\u5fc3\u7684\u6570\u636e\uff0c\u7f8e\u56fd\u60a3\u6709SCD\u7684\u4eba\u6570\u4e0d\u5230200,000\u4eba\uff0c\u60a3\u6709\u03b2\u5730\u4e2d\u6d77\u8d2b\u8840\u7684\u4eba\u6570\u4e0d\u52305,000\u4eba\u3002<\/p>\n<p>CRISPR\u548cVertex\u5728\u4eca\u5e74\u6625\u5929\u548c6\u67088\u65e5\u63d0\u4ea4\u4e86\u7528\u4e8e\u6cbb\u7597SCD\u548cTDT\u7684exa-cel\u751f\u7269\u5236\u5242\u8bb8\u53ef\u7533\u8bf7(BLA)\uff0cFDA\u8868\u793a\u5df2\u63a5\u53d7BLA\u3002FDA\u5bf9\u8be5\u836f\u7269\u4f5c\u4e3aTDT\u7597\u6cd5\u8fdb\u884c\u4e86\u6807\u51c6\u5ba1\u67e5\u3002\u8fd9\u610f\u5473\u7740\uff0c\u6700\u65e9\u5728\u660e\u5e74\uff0cCRISPR\u53ef\u80fd\u4f1a\u83b7\u5f97\u4ea7\u54c1\u6536\u5165\uff0cCRISPR\u5c06\u83b7\u5f9740%\u7684\u5206\u6210\uff0c\u5176\u4f59\u90e8\u5206\u7531Vertex\u83b7\u5f97\u3002\u800c\u8fd9\u4ec5\u4ec5\u662f\u4e2a\u5f00\u59cb\u3002\u4e0e\u4f20\u7edf\u7597\u6cd5\u76f8\u6bd4\uff0cCRISPR\u6b63\u5728\u5f00\u53d1\u7684\u6cbb\u7597\u6027\u57fa\u56e0\u7f16\u8f91\u7597\u6cd5\u5177\u6709\u9769\u547d\u6027\u610f\u4e49\u3002\u76ee\u524d\uff0c\u5b83\u6b63\u5728\u7814\u7a76\u4fee\u590d\u6d89\u53ca\u5177\u6709\u5355\u4e00\u7a81\u53d8\u7684\u57fa\u56e0\u7684\u75be\u75c5\uff0c\u4f46\u8fd9\u4ec5\u4ec5\u662f\u4e2a\u5f00\u59cb\u3002\u5373\u4f7f\u6210\u672c\u5f88\u9ad8\uff0c\u672c\u8d28\u4e0a\u53ef\u4ee5\u6cbb\u6108\u75be\u75c5\u7684\u57fa\u56e0\u7f16\u8f91\u7597\u6cd5\u5728\u60a3\u8005\u7684\u4e00\u751f\u4e2d\u53ef\u80fd\u66f4\u5177\u6210\u672c\u6548\u76ca\uff0c\u7279\u522b\u662f\u5bf9\u4e8e\u6162\u6027\u75c5\u3002<\/p>\n<p>Exa-cel\u53ef\u80fd\u6210\u4e3aCRISPR\u7684\u91cd\u78c5\u70b8\u5f39\u7597\u6cd5\uff0c\u7ed3\u675f\u67d0\u4e9bSCD\u548cTDT\u60a3\u8005\u7ec8\u751f\u9700\u8981\u8f93\u8840\u7684\u60c5\u51b5\u3002\u4e34\u5e8a\u548c\u7ecf\u6d4e\u8bc4\u8bba\u7814\u7a76\u6240(ICER)\u6700\u8fd1\u8868\u793a\uff0c\u8fd9\u79cd\u7597\u6cd5\u6bcf\u6b21\u6cbb\u7597\u4ef7\u503c190\u4e07\u7f8e\u5143\u3002\u6309\u7167\u8fd9\u4e2a\u4ef7\u683c\uff0cCRISPR\u5f88\u5feb\u5c31\u80fd\u76c8\u5229\u3002<\/p>\n<p>\u5982\u679cexa-cel\u83b7\u5f97\u6279\u51c6\uff0c\u5b83\u5c06\u4f5c\u4e3a\u516c\u53f8\u57fa\u56e0\u7f16\u8f91\u8fc7\u7a0b\u7684\u6982\u5ff5\u8bc1\u660e\uff0c\u89e3\u9501\u516c\u53f8\u7684\u5176\u4f59\u7ba1\u7ebf\u3002\u5341\u5e74\u540e\uff0c\u8be5\u516c\u53f8\u53ef\u80fd\u4f1a\u63a8\u51fa\u51e0\u79cd\u91cd\u78c5\u7597\u6cd5\u3002<\/p>\n<p>\u7531\u4e8e\u5408\u4f5c\u6536\u5165\uff0cCRISPR\u7684\u8d22\u52a1\u72b6\u51b5\u6bd4\u5927\u591a\u6570\u8fd8\u6ca1\u6709\u4ea7\u54c1\u7684\u4e34\u5e8a\u9636\u6bb5\u751f\u7269\u79d1\u6280\u516c\u53f8\u8981\u597d\u5f97\u591a\u3002\u622a\u81f3\u7b2c\u4e00\u8d22\u5b63\uff0cCRISPR\u62e5\u670918.9\u4ebf\u7f8e\u5143\u73b0\u91d1\uff0c\u8db3\u4ee5\u4e3a\u81f3\u5c11\u4e09\u5e74\u7684\u8fd0\u8425\u63d0\u4f9b\u8d44\u91d1\u3002\u5982\u679cexa-cel\u83b7\u5f97\u6279\u51c6\uff0c\u516c\u53f8\u5c06\u80fd\u591f\u5c06\u73b0\u91d1\u7528\u4e8e\u66f4\u591a\u7814\u53d1\u3002\u6ca1\u4eba\u77e5\u9053CRISPR\u5c06\u572810\u5e74\u5185\u8d70\u5411\u4f55\u65b9\uff0c\u4f46\u4e0e\u5927\u591a\u6570\u5904\u4e8e\u4e34\u5e8a\u9636\u6bb5\u7684\u751f\u7269\u79d1\u6280\u516c\u53f8\u76f8\u6bd4\uff0c\u81f3\u5c11\u6709\u4e00\u5f20\u516c\u53f8\u53ef\u80fd\u8d70\u5411\u4f55\u65b9\u7684\u8def\u7ebf\u56fe\u3002<\/p>\n","protected":false},"excerpt":{"rendered":"<p>CRISPR Therapeutics\u80a1\u7968\u4ea4\u6613\u4ef7\u683c\u4e0d\u5230\u4e24\u5e74\u524d\u7684\u4e09\u5206\u4e4b\u4e00\u3002 \u8be5\u516c\u53f8\u7684\u5148\u5bfc\u7597\u6cd5\u53ef\u80fd\u4f1a\u5728\u4eca\u5e74\u665a\u4e9b\u65f6\u5019 [&hellip;]<\/p>\n","protected":false},"author":9,"featured_media":464036,"comment_status":"closed","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"footnotes":"","_links_to":"","_links_to_target":""},"categories":[427],"tags":[455,925,459],"class_list":["post-464032","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-market-news-zh-hans","tag-455","tag-925","tag-459","sectors-life-science-zh-hans","locations-us-zh-hans","exchange-nasdaq-zh-hans","exchange-nyse-zh-hans"],"acf":[],"_links":{"self":[{"href":"https:\/\/nai500.com\/zh-hans\/wp-json\/wp\/v2\/posts\/464032","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/nai500.com\/zh-hans\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/nai500.com\/zh-hans\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/nai500.com\/zh-hans\/wp-json\/wp\/v2\/users\/9"}],"replies":[{"embeddable":true,"href":"https:\/\/nai500.com\/zh-hans\/wp-json\/wp\/v2\/comments?post=464032"}],"version-history":[{"count":0,"href":"https:\/\/nai500.com\/zh-hans\/wp-json\/wp\/v2\/posts\/464032\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/nai500.com\/zh-hans\/wp-json\/wp\/v2\/media\/464036"}],"wp:attachment":[{"href":"https:\/\/nai500.com\/zh-hans\/wp-json\/wp\/v2\/media?parent=464032"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/nai500.com\/zh-hans\/wp-json\/wp\/v2\/categories?post=464032"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/nai500.com\/zh-hans\/wp-json\/wp\/v2\/tags?post=464032"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}