{"id":49331,"date":"2024-11-17T23:56:00","date_gmt":"2024-11-17T15:56:00","guid":{"rendered":"https:\/\/www.granitefirm.com\/blog\/?p=49331"},"modified":"2025-11-07T11:11:41","modified_gmt":"2025-11-07T03:11:41","slug":"%e5%9f%ba%e5%9b%a0%e7%b7%a8%e8%bc%af%e7%99%82%e6%b3%95%e5%89%b5%e5%a7%8b%e8%80%85crispr%e5%a6%82%e4%bd%95%e8%b3%ba%e9%8c%a2%ef%bc%9f","status":"publish","type":"post","link":"https:\/\/www.granitefirm.com\/blog\/blog\/2024\/11\/17\/%e5%9f%ba%e5%9b%a0%e7%b7%a8%e8%bc%af%e7%99%82%e6%b3%95%e5%89%b5%e5%a7%8b%e8%80%85crispr%e5%a6%82%e4%bd%95%e8%b3%ba%e9%8c%a2%ef%bc%9f\/","title":{"rendered":"\u57fa\u56e0\u7de8\u8f2f\u7642\u6cd5\u5275\u59cb\u8005CRISPR\u5982\u4f55\u8cfa\u9322\uff1f"},"content":{"rendered":"\n<p>\u300a<a href=\"https:\/\/www.granitefirm.com\/blog\/blog\/2024\/11\/02\/%e5%9f%ba%e5%9b%a0%e7%b7%a8%e8%bc%afcasgevy%e8%a2%ab%e6%89%b9%e5%87%86\/\">\u5168\u7403\u9996\u500b\u57fa\u56e0\u7de8\u8f2f\u7642\u6cd5Casgevy\u7d42\u65bc\u88ab\u6279\u51c6\u4e0a\u5e02<\/a>\u300b\u662f\u672c\u6587\u7684\u9644\u5c6c\u8cbc\u6587\uff0c\u5efa\u8b70\u60a8\u4e00\u4f75\u95b1\u8b80\u3002<\/p>\n\n\n\n<div id=\"ez-toc-container\" class=\"ez-toc-v2_0_84 counter-hierarchy ez-toc-counter ez-toc-custom ez-toc-container-direction\">\n<div class=\"ez-toc-title-container\">\n<p class=\"ez-toc-title\" style=\"cursor:inherit\">Table of Contents<\/p>\n<span class=\"ez-toc-title-toggle\"><a href=\"#\" class=\"ez-toc-pull-right ez-toc-btn ez-toc-btn-xs ez-toc-btn-default ez-toc-toggle\" aria-label=\"\u986f\u793a\/\u96b1\u85cf\u5167\u5bb9\u76ee\u9304\"><span class=\"ez-toc-js-icon-con\"><span class=\"\"><span class=\"eztoc-hide\" style=\"display:none;\">Toggle<\/span><span class=\"ez-toc-icon-toggle-span\"><svg style=\"fill: #ffffff;color:#ffffff\" xmlns=\"http:\/\/www.w3.org\/2000\/svg\" class=\"list-377408\" width=\"20px\" height=\"20px\" viewBox=\"0 0 24 24\" fill=\"none\"><path d=\"M6 6H4v2h2V6zm14 0H8v2h12V6zM4 11h2v2H4v-2zm16 0H8v2h12v-2zM4 16h2v2H4v-2zm16 0H8v2h12v-2z\" fill=\"currentColor\"><\/path><\/svg><svg style=\"fill: #ffffff;color:#ffffff\" class=\"arrow-unsorted-368013\" xmlns=\"http:\/\/www.w3.org\/2000\/svg\" width=\"10px\" height=\"10px\" viewBox=\"0 0 24 24\" version=\"1.2\" baseProfile=\"tiny\"><path d=\"M18.2 9.3l-6.2-6.3-6.2 6.3c-.2.2-.3.4-.3.7s.1.5.3.7c.2.2.4.3.7.3h11c.3 0 .5-.1.7-.3.2-.2.3-.5.3-.7s-.1-.5-.3-.7zM5.8 14.7l6.2 6.3 6.2-6.3c.2-.2.3-.5.3-.7s-.1-.5-.3-.7c-.2-.2-.4-.3-.7-.3h-11c-.3 0-.5.1-.7.3-.2.2-.3.5-.3.7s.1.5.3.7z\"\/><\/svg><\/span><\/span><\/span><\/a><\/span><\/div>\n<nav><ul class='ez-toc-list ez-toc-list-level-1 ' ><li class='ez-toc-page-1 ez-toc-heading-level-2'><a class=\"ez-toc-link ez-toc-heading-1\" href=\"https:\/\/www.granitefirm.com\/blog\/blog\/2024\/11\/17\/%e5%9f%ba%e5%9b%a0%e7%b7%a8%e8%bc%af%e7%99%82%e6%b3%95%e5%89%b5%e5%a7%8b%e8%80%85crispr%e5%a6%82%e4%bd%95%e8%b3%ba%e9%8c%a2%ef%bc%9f\/#%E5%9F%BA%E5%9B%A0%E7%B7%A8%E8%BC%AF%E7%9A%84%E6%8B%93%E8%8D%92%E8%80%85\" >\u57fa\u56e0\u7de8\u8f2f\u7684\u62d3\u8352\u8005<\/a><\/li><li class='ez-toc-page-1 ez-toc-heading-level-2'><a class=\"ez-toc-link ez-toc-heading-2\" href=\"https:\/\/www.granitefirm.com\/blog\/blog\/2024\/11\/17\/%e5%9f%ba%e5%9b%a0%e7%b7%a8%e8%bc%af%e7%99%82%e6%b3%95%e5%89%b5%e5%a7%8b%e8%80%85crispr%e5%a6%82%e4%bd%95%e8%b3%ba%e9%8c%a2%ef%bc%9f\/#%E8%AB%BE%E8%B2%9D%E7%88%BE%E5%8C%96%E5%AD%B8%E7%8D%8E%E5%BE%97%E4%B8%BB\" >\u8afe\u8c9d\u723e\u5316\u5b78\u734e\u5f97\u4e3b<\/a><\/li><li class='ez-toc-page-1 ez-toc-heading-level-2'><a class=\"ez-toc-link ez-toc-heading-3\" href=\"https:\/\/www.granitefirm.com\/blog\/blog\/2024\/11\/17\/%e5%9f%ba%e5%9b%a0%e7%b7%a8%e8%bc%af%e7%99%82%e6%b3%95%e5%89%b5%e5%a7%8b%e8%80%85crispr%e5%a6%82%e4%bd%95%e8%b3%ba%e9%8c%a2%ef%bc%9f\/#%E4%B8%BB%E8%A6%81%E7%94%A2%E5%93%81\" >\u4e3b\u8981\u7522\u54c1<\/a><\/li><li class='ez-toc-page-1 ez-toc-heading-level-2'><a class=\"ez-toc-link ez-toc-heading-4\" href=\"https:\/\/www.granitefirm.com\/blog\/blog\/2024\/11\/17\/%e5%9f%ba%e5%9b%a0%e7%b7%a8%e8%bc%af%e7%99%82%e6%b3%95%e5%89%b5%e5%a7%8b%e8%80%85crispr%e5%a6%82%e4%bd%95%e8%b3%ba%e9%8c%a2%ef%bc%9f\/#%E5%92%8C%E7%A6%8F%E6%B3%B0%E5%90%88%E4%BD%9C\" >\u548c\u798f\u6cf0\u5408\u4f5c<\/a><\/li><li class='ez-toc-page-1 ez-toc-heading-level-2'><a class=\"ez-toc-link ez-toc-heading-5\" href=\"https:\/\/www.granitefirm.com\/blog\/blog\/2024\/11\/17\/%e5%9f%ba%e5%9b%a0%e7%b7%a8%e8%bc%af%e7%99%82%e6%b3%95%e5%89%b5%e5%a7%8b%e8%80%85crispr%e5%a6%82%e4%bd%95%e8%b3%ba%e9%8c%a2%ef%bc%9f\/#%E4%B8%BB%E8%A6%81%E7%AB%B6%E7%88%AD%E5%B0%8D%E6%89%8B\" >\u4e3b\u8981\u7af6\u722d\u5c0d\u624b<\/a><ul class='ez-toc-list-level-3' ><li class='ez-toc-heading-level-3'><a class=\"ez-toc-link ez-toc-heading-6\" href=\"https:\/\/www.granitefirm.com\/blog\/blog\/2024\/11\/17\/%e5%9f%ba%e5%9b%a0%e7%b7%a8%e8%bc%af%e7%99%82%e6%b3%95%e5%89%b5%e5%a7%8b%e8%80%85crispr%e5%a6%82%e4%bd%95%e8%b3%ba%e9%8c%a2%ef%bc%9f\/#%E8%97%8D%E9%B3%A5%E7%94%9F%E6%8A%80%EF%BC%88Bluebird_Bio%EF%BC%89\" >\u85cd\u9ce5\u751f\u6280\uff08Bluebird Bio\uff09<\/a><\/li><\/ul><\/li><li class='ez-toc-page-1 ez-toc-heading-level-2'><a class=\"ez-toc-link ez-toc-heading-7\" href=\"https:\/\/www.granitefirm.com\/blog\/blog\/2024\/11\/17\/%e5%9f%ba%e5%9b%a0%e7%b7%a8%e8%bc%af%e7%99%82%e6%b3%95%e5%89%b5%e5%a7%8b%e8%80%85crispr%e5%a6%82%e4%bd%95%e8%b3%ba%e9%8c%a2%ef%bc%9f\/#%E8%BF%91%E6%9C%9F%E7%87%9F%E9%81%8B%E8%A1%A8%E7%8F%BE\" >\u8fd1\u671f\u71df\u904b\u8868\u73fe<\/a><\/li><li class='ez-toc-page-1 ez-toc-heading-level-2'><a class=\"ez-toc-link ez-toc-heading-8\" href=\"https:\/\/www.granitefirm.com\/blog\/blog\/2024\/11\/17\/%e5%9f%ba%e5%9b%a0%e7%b7%a8%e8%bc%af%e7%99%82%e6%b3%95%e5%89%b5%e5%a7%8b%e8%80%85crispr%e5%a6%82%e4%bd%95%e8%b3%ba%e9%8c%a2%ef%bc%9f\/#%E4%BB%8D%E7%84%B6%E6%B2%92%E6%9C%89%E7%9B%88%E5%88%A9\" >\u4ecd\u7136\u6c92\u6709\u76c8\u5229<\/a><\/li><li class='ez-toc-page-1 ez-toc-heading-level-2'><a class=\"ez-toc-link ez-toc-heading-9\" href=\"https:\/\/www.granitefirm.com\/blog\/blog\/2024\/11\/17\/%e5%9f%ba%e5%9b%a0%e7%b7%a8%e8%bc%af%e7%99%82%e6%b3%95%e5%89%b5%e5%a7%8b%e8%80%85crispr%e5%a6%82%e4%bd%95%e8%b3%ba%e9%8c%a2%ef%bc%9f\/#%E8%B3%87%E6%9C%AC%E5%B8%82%E5%A0%B4%E7%9A%84%E8%A1%A8%E7%8F%BE\" >\u8cc7\u672c\u5e02\u5834\u7684\u8868\u73fe<\/a><\/li><li class='ez-toc-page-1 ez-toc-heading-level-2'><a class=\"ez-toc-link ez-toc-heading-10\" href=\"https:\/\/www.granitefirm.com\/blog\/blog\/2024\/11\/17\/%e5%9f%ba%e5%9b%a0%e7%b7%a8%e8%bc%af%e7%99%82%e6%b3%95%e5%89%b5%e5%a7%8b%e8%80%85crispr%e5%a6%82%e4%bd%95%e8%b3%ba%e9%8c%a2%ef%bc%9f\/#%E7%9B%B8%E9%97%9C%E6%96%87%E7%AB%A0\" >\u76f8\u95dc\u6587\u7ae0<\/a><\/li><\/ul><\/nav><\/div>\n<h2 class=\"wp-block-heading\"><span class=\"ez-toc-section\" id=\"%E5%9F%BA%E5%9B%A0%E7%B7%A8%E8%BC%AF%E7%9A%84%E6%8B%93%E8%8D%92%E8%80%85\"><\/span>\u57fa\u56e0\u7de8\u8f2f\u7684\u62d3\u8352\u8005<span class=\"ez-toc-section-end\"><\/span><\/h2>\n\n\n\n<p><a href=\"https:\/\/en.wikipedia.org\/wiki\/CRISPR_Therapeutics\" target=\"_blank\" rel=\"noreferrer noopener\">CRISPR Therapeutics\uff08\u7f8e\u80a1\u4ee3\u78bc\uff1aCRSP\uff09<\/a>\u662f\u4e00\u5bb6\u7e3d\u90e8\u4f4d\u65bc\u745e\u58eb\u7684\u751f\u6280\u516c\u53f8\u3002\u5b83\u662f\u6700\u65e9\u6210\u7acb\u7684\u5229\u7528CRISPR\u57fa\u56e0\u7de8\u8f2f\u5e73\u53f0\u958b\u767c\u6cbb\u7642\u5404\u7a2e\u7f55\u898b\u548c\u5e38\u898b\u75be\u75c5\u85e5\u7269\u7684\u516c\u53f8\u4e4b\u4e00\u3002<\/p>\n\n\n\n<h2 class=\"wp-block-heading\"><span class=\"ez-toc-section\" id=\"%E8%AB%BE%E8%B2%9D%E7%88%BE%E5%8C%96%E5%AD%B8%E7%8D%8E%E5%BE%97%E4%B8%BB\"><\/span>\u8afe\u8c9d\u723e\u5316\u5b78\u734e\u5f97\u4e3b<span class=\"ez-toc-section-end\"><\/span><\/h2>\n\n\n\n<p>CRISPR \u7531Emmanuelle Charpentier \u3001Shaun Foy \u548c Rodger Novak\u65bc 2013 \u5e74\u5275\u7acb\u3002Charpentier \u5f8c\u4f86\u8207Jennifer Doudna\u5206\u4eab\u4e862020 \u5e74\u8afe\u8c9d\u723e\u5316\u5b78\u734e\u3002\u4f5c\u70ba\u5de5\u4f5c\u5c0f\u7d44\u7684\u4e00\u54e1\uff0c\u5979\u63d0\u4f9b\u4e86\u7b2c\u4e00\u4efd\u6709\u95dcCRISPR \u57fa\u56e0\u7de8\u8f2f\u7684\u958b\u767c\u548c\u4f7f\u7528\u7684\u79d1\u5b78\u6587\u737b\u3002\u9019\u4f7f\u5f97 DNA \u80fd\u5920\u88ab\u5c08\u9580\u4fee\u6539\u548c\u7de8\u8f2f\uff0c\u5f9e\u800c\u53ef\u4ee5\u7528\u4f86\u6539\u5584\u75be\u75c5\u3002CRISPR \u6b63\u61c9\u7528\u6b64\u6280\u8853\u5e73\u53f0\u4f86\u7814\u7a76\u3001\u958b\u767c\u548c\u5546\u696d\u5316\u6cbb\u7642\u5404\u7a2e\u75be\u75c5\u7684\u85e5\u7269\uff0c\u5305\u62ec\u942e\u72c0\u7d30\u80de\u75be\u75c5\u3001\u03b2\u5730\u4e2d\u6d77\u8ca7\u8840\u3001\u5404\u7a2e\u764c\u75c7\u3001\u7b2c\u4e00\u578b\u7cd6\u5c3f\u75c5\u548c\u5fc3\u8840\u7ba1\u75be\u75c5\u3002<\/p>\n\n\n\n<h2 class=\"wp-block-heading\"><span class=\"ez-toc-section\" id=\"%E4%B8%BB%E8%A6%81%E7%94%A2%E5%93%81\"><\/span>\u4e3b\u8981\u7522\u54c1<span class=\"ez-toc-section-end\"><\/span><\/h2>\n\n\n\n<p>CRISPR Therapeutics \u6b63\u5728\u958b\u767c\u591a\u7a2e\u6cbb\u7642\u8840\u6db2\u75be\u75c5\u3001\u764c\u75c7\u3001\u7cd6\u5c3f\u75c5\u548c\u5176\u4ed6\u56b4\u91cd\u75be\u75c5\u7684\u85e5\u7269\u3002<\/p>\n\n\n\n<p>2024 \u5e74\uff0c\u9664\u4e86\u6b63\u5728\u9032\u884c\u7684\u764c\u75c7\u548c\u81ea\u9ad4\u514d\u75ab\u75be\u75c5\u8a66\u9a57\u4e4b\u5916\uff0cCRISPR \u4ecd\u6709\u671b\u555f\u52d5 CTX112 \u7684\u81e8\u5e8a\u8a66\u9a57\uff0cCTX112 \u662f\u4e00\u7a2e\u6cbb\u7642\u7cfb\u7d71\u6027\u7d05\u6591\u72fc\u7621\u7684\u57fa\u56e0\u7de8\u8f2f\u7642\u6cd5\u3002<\/p>\n\n\n\n<h2 class=\"wp-block-heading\"><span class=\"ez-toc-section\" id=\"%E5%92%8C%E7%A6%8F%E6%B3%B0%E5%90%88%E4%BD%9C\"><\/span>\u548c\u798f\u6cf0\u5408\u4f5c<span class=\"ez-toc-section-end\"><\/span><\/h2>\n\n\n\n<p>\u7531\u65bcCRISPR\u662f\u65b0\u8208\u7684\u5c0f\u751f\u6280\u696d\u8005\uff0c\u70ba\u4e86\u751f\u5b58\u548c\u696d\u52d9\u64f4\u5f35\uff0c\u548c\u5176\u5b83\u751f\u6280\u65b0\u5275\u696d\u8005\u6c92\u6709\u4e0d\u540c\uff0c\u5fc5\u9700\u5148\u627e\u4e00\u5bb6\u570b\u969b\u578b\u5927\u85e5\u5ee0\u5408\u4f5c\uff0c\u624d\u80fd\u5b8c\u6210\u516c\u53f8\u7684\u5546\u696d\u76ee\u7684\u3002 <\/p>\n\n\n\n<p>\u56e0\u6b64\uff0c\u798f\u6cf0\u88fd\u85e5\u548c CRISPR \u65bc 2015 \u5e74\u9054\u6210\u7b56\u7565\u6027\u7814\u7a76\u5408\u4f5c\uff0c\u91cd\u9ede\u662f\u5229\u7528 CRISPR\/Cas9 \u767c\u73fe\u548c\u958b\u767c\u91dd\u5c0d\u4eba\u985e\u75be\u75c5\u6f5b\u5728\u907a\u50b3\u539f\u56e0\u7684\u6f5b\u5728\u65b0\u7642\u6cd5\u3002<\/p>\n\n\n\n<h2 class=\"wp-block-heading\"><span class=\"ez-toc-section\" id=\"%E4%B8%BB%E8%A6%81%E7%AB%B6%E7%88%AD%E5%B0%8D%E6%89%8B\"><\/span>\u4e3b\u8981\u7af6\u722d\u5c0d\u624b<span class=\"ez-toc-section-end\"><\/span><\/h2>\n\n\n\n<h3 class=\"wp-block-heading\"><span class=\"ez-toc-section\" id=\"%E8%97%8D%E9%B3%A5%E7%94%9F%E6%8A%80%EF%BC%88Bluebird_Bio%EF%BC%89\"><\/span>\u85cd\u9ce5\u751f\u6280\uff08Bluebird Bio\uff09<span class=\"ez-toc-section-end\"><\/span><\/h3>\n\n\n\n<p>2023\u5e74 12 \u6708\uff0cFDA \u6279\u51c6\u4e86\u85cd\u9ce5\u751f\u6280\uff08\u7f8e\u80a1\u4ee3\u78bc\uff1aBLUE\uff09\u7684 Lyfgenia \u6cbb\u7642\u942e\u72c0\u7d30\u80de\u75be\u75c5\uff0c\u4ee5\u53ca\u7531 Vertex Pharmaceuticals (VRTX) \u548c CRISPR Therapeutics (CRSP) \u958b\u767c\u7684\u91dd\u5c0d\u8a72\u75be\u75c5\u7684\u7af6\u722d\u5c0d\u624b\u57fa\u56e0\u7642\u6cd5\u3002<\/p>\n\n\n\n<p>\u5b83\u5728\u5e02\u9762\u4e0a\u6709\u92b7\u552e TDT \u548c SCD \u7684\u57fa\u56e0\u7de8\u8f2f\u6cbb\u7642\u65b9\u6cd5\u3002\u4f46\u6709\u4e09\u500b\u539f\u56e0\u986f\u793a\u5b83\u7684\u5a01\u8105\u6027\u61c9\u8a72\u4e0d\u5927\uff1a<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u56e0\u70ba\u9996\u5148\u85cd\u9ce5\u751f\u6280\u50c5\u5728\u7f8e\u570b\u71df\u904b\u3002<\/li>\n\n\n\n<li>\u5176\u6b21\uff0c\u798f\u6cf0\u662f\u4e00\u5bb6\u6bd4\u85cd\u9ce5\u751f\u6280\u6216 CRISPR \u898f\u6a21\u5927\u5f97\u591a\u7684\u751f\u7269\u6280\u8853\u516c\u53f8\uff0c\u4e26\u4e14\u64c1\u6709\u8207\u7b2c\u4e09\u65b9\u4ed8\u6b3e\u4eba\u8ac7\u5224\u5229\u6f64\u8c50\u539a\u4ea4\u6613\u7684\u8a18\u9304\u3002 <\/li>\n\n\n\n<li>\u7b2c\u4e09\uff0c\u85cd\u9ce5\u751f\u6280\u7684 SCD \u6cbb\u7642\u85e5\u7269 Lyfgenia \u5e36\u6709\u91dd\u5c0d\u8840\u764c\u7684\u9ed1\u6846\u8b66\u544a\u3002 \u6700\u5f8c\uff0cLyfgenia \u7684\u6210\u672c\u70ba 310 \u842c\u7f8e\u5143\uff0c\u800c Casgevy \u7684\u6210\u672c\u70ba 220 \u842c\u7f8e\u5143\u3002<\/li>\n<\/ul>\n\n\n\n<h2 class=\"wp-block-heading\"><span class=\"ez-toc-section\" id=\"%E8%BF%91%E6%9C%9F%E7%87%9F%E9%81%8B%E8%A1%A8%E7%8F%BE\"><\/span>\u8fd1\u671f\u71df\u904b\u8868\u73fe<span class=\"ez-toc-section-end\"><\/span><\/h2>\n\n\n\n<p>\u5df2\u516c\u4f48\u76842024\u7b2c\u4e00\u5b63\u300c\u7372\u5229\u300d\u7d50\u679c\u770b\u8d77\u4f86\u66f4\u50cf\u662f\u8667\u640d\uff0c\u4f7f\u8a72\u516c\u53f8\u6536\u76e4\u4e0b\u8dcc 8.5%\u3002 \u7531\u65bc\u7f3a\u4e4f\u50cf 2023 \u5e74\u7b2c\u4e00\u5b63\u90a3\u6a23\u7684\u91cd\u5927\u91cc\u7a0b\u7891\u4ed8\u6b3e\uff0cCRISPR \u7684\u71df\u6536\u5e74\u6e1b 99.5%\uff0c\u8a72\u516c\u53f8\u5831\u544a\u6bcf\u80a1\u8667\u640d 1.43 \u7f8e\u5143\uff0c\u662f\u53bb\u5e74\u540c\u671f\u8667\u640d\u7684\u5169\u500d\u591a\u3002<\/p>\n\n\n\n<h2 class=\"wp-block-heading\"><span class=\"ez-toc-section\" id=\"%E4%BB%8D%E7%84%B6%E6%B2%92%E6%9C%89%E7%9B%88%E5%88%A9\"><\/span>\u4ecd\u7136\u6c92\u6709\u76c8\u5229<span class=\"ez-toc-section-end\"><\/span><\/h2>\n\n\n\n<p>2024 \u5e74\u7b2c\u4e00\u5b63\u5831\u544a\u4e2d\u7684\u5b63\u5ea6\u81ea\u7531\u73fe\u91d1\u6d41\u50c5\u70ba 1.665 \u5104\u7f8e\u5143\u3002 <\/p>\n\n\n\n<p>\u4f46\u597d\u6d88\u606f\u662fCRISPR \u81f3\u5c11\u9084\u6709\u4e09\u5e74\u7684\u6642\u9593\u624d\u53ef\u80fd\u9700\u8981\u984d\u5916\u7684\u73fe\u91d1\u3002 \u9019\u662f\u56e0\u70ba\u8a72\u516c\u53f8\u8207 Vertex Pharmaceuticals \u5408\u4f5c\u958b\u767c\u7684\u6cbb\u7642\u942e\u72c0\u7d30\u80de\u6027\u8ca7\u8840\u7684 Casgevy \u7642\u6cd5\u5df2\u7372\u5f97 FDA \u6279\u51c6\u3002\u5b83\u53ef\u4ee5\u4f9d\u9760 CRISPR \u548c Vertex \u4e4b\u9593 40-60 \u7684\u92b7\u552e\u6536\u5165\u5206\u6210\uff0c\u6d3b\u4e0b\u4f86\u662f\u4e0d\u6210\u554f\u984c\u3002<\/p>\n\n\n\n<h2 class=\"wp-block-heading\"><span class=\"ez-toc-section\" id=\"%E8%B3%87%E6%9C%AC%E5%B8%82%E5%A0%B4%E7%9A%84%E8%A1%A8%E7%8F%BE\"><\/span>\u8cc7\u672c\u5e02\u5834\u7684\u8868\u73fe<span class=\"ez-toc-section-end\"><\/span><\/h2>\n\n\n\n<p>CRISPR\u7684\u5408\u4f5c\u4f19\u4f34\u798f\u6cf0\u88fd\u85e5\u7684\u80a1\u50f9\u57282023\u5e74\u7684\u4e00\u5e74\u5167\u4e0a\u6f32\u4e86\u7d04 42.26%\uff1b\u800c\u540c\u671f\u9593CRISPR Therapeutics\u5247\u4e0a\u6f32\u4e8652.68%\u3002<\/p>\n\n\n\n<figure class=\"wp-block-image size-full\"><img loading=\"lazy\" decoding=\"async\" width=\"120\" height=\"131\" src=\"https:\/\/www.granitefirm.com\/blog\/wp-content\/uploads\/2024\/05\/CRISPR-Custom.png\" alt=\"CRISPR\" class=\"wp-image-49369\"\/><figcaption class=\"wp-element-caption\">credit: Wiki<\/figcaption><\/figure>\n\n\n\n<p><em><strong>\u672c\u4eba\u70ba\u539f\u6587\u4f5c\u8005\uff0c\u672c\u6587\u7cbe\u83ef\u7248\u539f\u520a\u767b\u5728Smart\u667a\u5bcc\u6708<\/strong><\/em><strong><em>\u520b2024\u5e7410\u6708\u865f<\/em><\/strong><\/p>\n\n\n\n<h2 class=\"wp-block-heading\"><span class=\"ez-toc-section\" id=\"%E7%9B%B8%E9%97%9C%E6%96%87%E7%AB%A0\"><\/span>\u76f8\u95dc\u6587\u7ae0<span class=\"ez-toc-section-end\"><\/span><\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u300a<a href=\"https:\/\/www.granitefirm.com\/blog\/blog\/2025\/11\/06\/%e7%be%8e%e5%9c%8b%e6%9c%80%e5%a4%a7%e7%9a%84%e9%86%ab%e7%99%82%e9%99%a2%e6%89%80hca-healthcare\/\">\u7f8e\u570b\u6700\u5927\u7684\u91ab\u7642\u9662\u6240HCA Healthcare\u5982\u4f55\u8cfa\u9322\uff1f<\/a>\u300b<\/li>\n\n\n\n<li>\u300a<a href=\"https:\/\/www.granitefirm.com\/blog\/blog\/2025\/10\/18\/%e4%b8%ad%e5%9c%8b%e7%9a%84%e7%94%9f%e6%8a%80%e8%a3%bd%e8%97%a5%e6%a5%ad%e9%96%8b%e5%a7%8b%e5%b1%95%e7%8f%be%e5%ae%83%e7%9a%84%e6%b7%b1%e5%ba%a6%e6%b1%82%e7%b4%a2%e6%99%82%e5%88%bb\/\">\u4e2d\u570b\u7684\u751f\u6280\u88fd\u85e5\u696d\u958b\u59cb\u5c55\u73fe\u5b83\u7684\u6df1\u5ea6\u6c42\u7d22\u6642\u523b<\/a>\u300b<\/li>\n\n\n\n<li>\u300a<a href=\"https:\/\/www.granitefirm.com\/blog\/blog\/2025\/09\/16\/%e5%ba%b7%e6%96%b9%e8%82%ba%e7%99%8c%e8%97%a5%e6%96%b0%e8%97%a5%e7%99%82%e6%95%88%e5%a4%a7%e5%8b%9d%e5%85%a8%e7%90%83%e8%97%a5%e6%a6%9c%e9%8a%b7%e9%87%8f%e5%86%a0%e8%bb%8d\/\">\u5eb7\u65b9\uff08Akeso&nbsp;\uff09\u80ba\u764c\u85e5\u65b0\u85e5\u7642\u6548\u5927\u52dd\u5168\u7403\u85e5\u699c\u92b7\u91cf\u51a0\u8ecd\uff0c\u9707\u64bc\u7684\u751f\u6280\u696dDeepSeek\u4e8b\u4ef6<\/a>\u300b<\/li>\n\n\n\n<li>\u300a<a href=\"https:\/\/www.granitefirm.com\/blog\/blog\/2023\/11\/15\/%e7%a6%ae%e4%be%86%e7%82%ba%e4%bd%95%e8%83%bd%e6%88%90%e7%82%ba%e6%96%b0%e4%b8%80%e4%bb%a3%e7%9a%84%e5%85%a8%e7%90%83%e8%a3%bd%e8%97%a5%e5%b8%82%e5%80%bc%e7%8e%8b%ef%bc%9f\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u79ae\u4f86\uff08Eli Lilly\uff09\u70ba\u4f55\u80fd\u6210\u70ba\u65b0\u4e00\u4ee3\u7684\u5168\u7403\u88fd\u85e5\u5e02\u503c\u738b\uff1f<\/a>\u300b<\/li>\n\n\n\n<li>\u300a<a href=\"https:\/\/www.granitefirm.com\/blog\/blog\/2024\/11\/29\/%e7%a6%8f%e6%b3%b0%ef%bc%88vertex%ef%bc%89%e7%b0%a1%e4%bb%8b\/\">\u751f\u6280\u65b0\u661f\u798f\u6cf0\uff08Vertex\uff09\u5982\u4f55\u8cfa\u9322\uff1f<\/a>\u300b<\/li>\n\n\n\n<li>\u300a<a href=\"https:\/\/www.granitefirm.com\/blog\/blog\/2024\/11\/17\/%E5%9F%BA%E5%9B%A0%E7%B7%A8%E8%BC%AF%E7%99%82%E6%B3%95%E5%89%B5%E5%A7%8B%E8%80%85crispr%E5%A6%82%E4%BD%95%E8%B3%BA%E9%8C%A2%EF%BC%9F\/\">\u57fa\u56e0\u7de8\u8f2f\u7642\u6cd5\u5275\u59cb\u8005CRISPR\u5982\u4f55\u8cfa\u9322\uff1f<\/a>\u300b<\/li>\n\n\n\n<li>\u300a<a href=\"https:\/\/www.granitefirm.com\/blog\/blog\/2024\/11\/02\/%e5%9f%ba%e5%9b%a0%e7%b7%a8%e8%bc%afcasgevy%e8%a2%ab%e6%89%b9%e5%87%86\/\">\u5168\u7403\u9996\u500b\u57fa\u56e0\u7de8\u8f2f\u7642\u6cd5Casgevy\u7d42\u65bc\u88ab\u6279\u51c6\u4e0a\u5e02<\/a>\u300b<\/li>\n\n\n\n<li>\u300a<a href=\"https:\/\/www.granitefirm.com\/blog\/blog\/2024\/06\/28\/%e6%8a%97%e7%97%85%e6%af%92%e8%97%a5%e7%8e%8b%e5%90%89%e5%88%a9%e5%be%b7%e9%96%8b%e7%99%bc%e5%87%ba%e4%b8%96%e4%b8%8a%e9%a6%96%e6%94%af%e6%84%9b%e6%bb%8b%e7%96%ab%e8%8b%97\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u6297\u75c5\u6bd2\u85e5\u738b\u5409\u5229\u5fb7\uff08Gilead\uff09\u958b\u767c\u51fa\u4e16\u4e0a\u9996\u652f\u611b\u6ecb\u75ab\u82d7<\/a>\u300b<\/li>\n\n\n\n<li>\u300a<a href=\"https:\/\/www.granitefirm.com\/blog\/blog\/2024\/06\/02\/%E9%9D%9E%E9%85%92%E7%B2%BE%E6%80%A7%E8%84%82%E8%82%AA%E8%82%9D%E7%82%8Emash\/#google_vignette\" target=\"_blank\" rel=\"noreferrer noopener\">FDA\u9996\u6b21\u6279\u51c6\u975e\u9152\u7cbe\u6027\u8102\u80aa\u809d\u708e(MASH)\u6cbb\u7642\u85e5\u7269<\/a>\u300b<\/li>\n\n\n\n<li>\u300a<a href=\"https:\/\/www.granitefirm.com\/blog\/blog\/2023\/12\/07\/%E9%98%BF%E8%8C%B2%E6%B5%B7%E9%BB%98%E7%97%87%EF%BC%8C%E5%94%AF%E4%B8%80%E5%B0%9A%E5%BE%85%E6%94%BB%E5%85%8B%E7%9A%84%E4%B8%BB%E8%A6%81%E7%96%BE%E7%97%85%E7%9A%84%E7%9B%AE%E5%89%8D%E9%80%B2%E5%B1%95\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u963f\u8332\u6d77\u9ed8\u75c7\uff0c\u552f\u4e00\u5c1a\u5f85\u653b\u514b\u7684\u4e3b\u8981\u75be\u75c5\u7684\u76ee\u524d\u9032\u5c55\u548c\u76f8\u95dc\u4f01\u696d<\/a>\u300b<\/li>\n\n\n\n<li>\u300a<a href=\"https:\/\/www.granitefirm.com\/blog\/blog\/2023\/08\/09\/%e4%bc%b0%e5%80%bc%e9%a9%9a%e4%ba%ba%e7%9a%84%e5%a4%a7%e8%97%a5%e5%bb%a0%e7%a6%ae%e4%be%86\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u6700\u65b0\u5e02\u503c\u5168\u7403\u7b2c\u4e00\u4f30\u503c\u9a5a\u4eba\u7684\u5927\u85e5\u5ee0\u79ae\u4f86\uff08Eli Lilly\uff09<\/a>\u300b<\/li>\n\n\n\n<li>\u300a<a href=\"https:\/\/www.granitefirm.com\/blog\/blog\/2023\/03\/30\/%e8%ab%be%e5%92%8c%e8%ab%be%e5%be%b7%e5%8f%b8%e7%be%8e%e6%a0%bc%e9%ad%af%e8%82%bd%e5%92%8c%e8%85%b8%e6%b3%8c%e7%b4%a0%e7%9a%84%e6%b8%9b%e9%87%8d%e6%95%88%e6%9e%9c\/\">\u8afe\u548c\u8afe\u5fb7\uff08Novo Nordisk\uff09\u7cd6\u5c3f\u75c5\u7684\u65b0\u85e5Semaglutide\uff08\u53f8\u7f8e\u683c\u9b6f\u80bd\uff09\u8afe\u548c\u6cf0 Ozempic\uff0cWegovy\u548cMounjaro\u88ab\u767c\u73fe\u5177\u6709\u9a5a\u4eba\u7684\u6e1b\u91cd\u6548\u679c<\/a>\u300b<\/li>\n\n\n\n<li>\u300a<a href=\"https:\/\/www.granitefirm.com\/blog\/blog\/2023\/08\/07\/%e6%b8%9b%e9%87%8d%e8%97%8d%e6%b5%b7%e6%98%af%e8%97%a5%e5%93%81%e5%b8%82%e5%a0%b4%e6%9c%aa%e4%be%86%e7%9a%84%e9%87%91%e9%9b%9e%e6%af%8d\/\">\u6e1b\u91cd\u85cd\u6d77\u662f\u85e5\u54c1\u5e02\u5834\u672a\u4f86\u7684\u91d1\u96de\u6bcd<\/a>\u300b<\/li>\n\n\n\n<li>\u300a<a href=\"https:\/\/www.granitefirm.com\/blog\/blog\/2023\/01\/16\/%e6%88%90%e5%8a%9f%e7%9a%84%e4%bc%81%e6%a5%ad%e4%b8%8d%e6%98%af%e6%8a%80%e8%a1%93%e7%99%bc%e6%98%8e%e8%80%85\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u6280\u8853\u767c\u660e\u8005\u5f88\u5c11\u6210\u529f\uff0c\u6539\u826f\u5275\u65b0\u8005\u53cd\u800c\u6210\u529f\u7372\u5229<\/a>\u300b<\/li>\n\n\n\n<li>\u300a<a href=\"https:\/\/www.granitefirm.com\/blog\/blog\/2022\/10\/17\/%e5%85%a8%e7%be%8e%e6%9c%80%e5%a4%a7%e7%9a%84%e5%81%a5%e4%bf%9d%e9%9b%86%e5%9c%98%e8%81%af%e5%90%88%e5%81%a5%e5%ba%b7%e4%bf%9d%e9%9a%aa\/\">\u5168\u7403\u6700\u5927\u7684\u5065\u4fdd\u5546\u806f\u5408\u5065\u5eb7\u4fdd\u96aa\uff0c\u9053\u74ca\u7684\u512a\u7b49\u751f<\/a>\u300b<\/li>\n\n\n\n<li>\u300a<a href=\"https:\/\/www.granitefirm.com\/blog\/blog\/2023\/11\/06\/%E6%9B%BE%E7%B6%93%E7%9A%84%E5%85%A8%E7%90%83%E5%A4%A7%E8%97%A5%E5%BB%A0%E9%BB%98%E6%B2%99%E6%9D%B1%EF%BC%88Merck%EF%BC%89\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u66fe\u7d93\u7684\u5168\u7403\u5927\u85e5\u5ee0\u9ed8\u6c99\u6771\uff08Merck\uff09<\/a>\u300b<\/li>\n\n\n\n<li>\u300a<a href=\"https:\/\/www.granitefirm.com\/blog\/blog\/2022\/07\/02\/%E5%85%A8%E7%90%83%E6%9C%80%E9%87%8D%E8%A6%81%E7%9A%84%E9%86%AB%E7%99%82%E5%99%A8%E6%9D%90%E5%92%8C%E7%87%9F%E9%A4%8A%E9%A3%9F%E5%93%81%E5%95%86%E4%BA%9E%E5%9F%B9\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u5168\u7403\u6700\u91cd\u8981\u7684\u91ab\u7642\u5668\u6750\u548c\u71df\u990a\u98df\u54c1\u5546\u4e9e\u57f9\uff08Abbott\uff09\u662f\u5982\u4f55\u8cfa\u9322\u7684\uff1f<\/a>\u300b<\/li>\n\n\n\n<li>\u300a<a href=\"https:\/\/www.granitefirm.com\/blog\/blog\/2022\/06\/24\/%e9%bb%98%e5%85%8b\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u7a4f\u5b9a\u7684\u9053\u74ca\u88fd\u85e5\u6210\u4efd\u80a1\u9ed8\u6c99\u6771\uff08Merck\uff09\u5982\u4f55\u8cfa\u9322\uff1f<\/a>\u300b<\/li>\n\n\n\n<li>\u300a<a href=\"https:\/\/www.granitefirm.com\/blog\/blog\/2022\/11\/29\/%e7%be%8e%e8%82%a1%e4%b8%bb%e8%a6%81%e8%97%a5%e5%bb%a0%e5%88%86%e6%8b%86%e5%8f%b2\/\">\u7f8e\u80a1\u4e3b\u8981\u85e5\u5ee0\u5206\u62c6\u53f2\uff0c\u548c\u6301\u6709\u5176\u80a1\u7968\u7684\u597d\u8655<\/a>\u300b<\/li>\n\n\n\n<li>\u300a<a href=\"https:\/\/www.granitefirm.com\/blog\/blog\/2022\/11\/18\/%e8%99%95%e6%96%b9%e8%97%a5%e9%a0%98%e5%b0%8e%e5%95%86%e8%89%be%e4%bc%af%e7%b6%ad\/\">\u4e0b\u4e00\u500b\u6392\u540d\u7b2c\u4e00\u7684\u88fd\u85e5\u696d\u9818\u8896\u827e\u4f2f\u7dad\uff08AbbVie\uff09<\/a>\u300b<\/li>\n\n\n\n<li>\u300a<a href=\"https:\/\/www.granitefirm.com\/blog\/blog\/2023\/05\/17\/%e7%be%85%e6%b0%8f%e6%8a%97%e7%99%8c%e8%97%a5%e7%a5%9e\/\">\u7f85\u6c0f\u88fd\u85e5\uff08Roche\uff09\uff0c\u6297\u764c\u85e5\u795e<\/a>\u300b<\/li>\n\n\n\n<li>\u300a<a href=\"https:\/\/www.granitefirm.com\/blog\/?s=%E5%85%A8%E7%90%83%E7%AC%AC%E4%B8%80%E5%A4%A7%E8%97%A5%E5%BB%A0%E8%BC%9D\" target=\"_blank\" rel=\"noreferrer noopener\">\u5168\u7403\u7b2c\u4e00\u5927\u85e5\u5ee0\u8f1d\u745e\uff08Pfizer<\/a>\uff09\u300b<\/li>\n\n\n\n<li><span style=\"font-size: revert;\">\u300a<a href=\"https:\/\/www.granitefirm.com\/blog\/blog\/2022\/09\/21\/%e5%ac%8c%e7%94%9fj%e6%98%af%e5%a6%82%e4%bd%95%e8%b3%ba%e9%8c%a2%e7%9a%84\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u64c1\u67093A\u50b5\u4fe1\u8a55\u7b49\u7684\u5b0c\u751f\uff08Johnson &amp; Johnson\uff09\u662f\u5982\u4f55\u8cfa\u9322\u7684\uff1f<\/a>\u300b<\/span><\/li>\n<\/ul>\n\n\n\n<p><em><strong>\u91cd\u8981\u8072\u660e<\/strong><\/em><\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li><em>\u672c\u7ad9\u5167\u5bb9\u70ba\u4f5c\u8005\u500b\u4eba\u610f\u898b\uff0c\u50c5\u4f9b\u53c3\u8003\uff0c\u672c\u4eba\u4e0d\u5c0d\u6587\u7ae0\u5167\u5bb9\u3001\u8cc7\u6599\u4e4b\u6b63\u78ba\u6027\u3001\u770b\u6cd5\u3001\u8207\u5373\u6642\u6027\u8ca0\u4efb\u4f55\u8cac\u4efb\uff0c\u8b80\u8005\u8acb\u52d9\u5fc5\u81ea\u884c\u5224\u65b7\u3002<\/em><\/li>\n\n\n\n<li><em>\u5c0d\u65bc\u8b80\u8005\u76f4\u63a5\u6216\u9593\u63a5\u4f9d\u8cf4\u4e26\u53c3\u8003\u672c\u7ad9\u8cc7\u8a0a\u5f8c\uff0c\u63a1\u53d6\u4efb\u4f55\u6295\u8cc7\u884c\u70ba\u6240\u5c0e\u81f4\u4e4b\u76f4\u63a5\u6216\u9593\u63a5\u640d\u5931\uff0c\u6216\u56e0\u6b64\u7522\u751f\u4e4b\u4e00\u5207\u8cac\u4efb\uff0c\u672c\u4eba\u5747\u4e0d\u8ca0\u4efb\u4f55\u640d\u5bb3\u8ce0\u511f\u53ca\u5176\u4ed6\u6cd5\u5f8b\u4e0a\u4e4b\u8cac\u4efb\u3002<\/em><\/li>\n<\/ul>\n","protected":false},"excerpt":{"rendered":"<p>CRISPR\u662f\u4e00\u5bb6\u7e3d\u90e8\u4f4d\u65bc\u745e\u58eb\u7684\u751f\u6280\u516c\u53f8\u3002Emmanuelle Charpentier \u3001Shaun Foy \u548c Rodger Novak\u65bc 2013 \u5e74\u5275\u7acb\u3002\u5b83\u662f\u6700\u65e9\u5229\u7528CRISPR\u57fa\u56e0\u7de8\u8f2f\u4e26\u7372\u5f97\u9996\u6279\u7684\u751f\u6280\u516c\u53f8\u3002<\/p>\n","protected":false},"author":1,"featured_media":49369,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[338,270,339],"tags":[1390,1411,1351],"class_list":["post-49331","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-biotech","category-new-technology","category-pharma","tag-blue","tag-csrp","tag-vrtx"],"_links":{"self":[{"href":"https:\/\/www.granitefirm.com\/blog\/wp-json\/wp\/v2\/posts\/49331","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.granitefirm.com\/blog\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.granitefirm.com\/blog\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.granitefirm.com\/blog\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/www.granitefirm.com\/blog\/wp-json\/wp\/v2\/comments?post=49331"}],"version-history":[{"count":3,"href":"https:\/\/www.granitefirm.com\/blog\/wp-json\/wp\/v2\/posts\/49331\/revisions"}],"predecessor-version":[{"id":74886,"href":"https:\/\/www.granitefirm.com\/blog\/wp-json\/wp\/v2\/posts\/49331\/revisions\/74886"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.granitefirm.com\/blog\/wp-json\/wp\/v2\/media\/49369"}],"wp:attachment":[{"href":"https:\/\/www.granitefirm.com\/blog\/wp-json\/wp\/v2\/media?parent=49331"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.granitefirm.com\/blog\/wp-json\/wp\/v2\/categories?post=49331"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.granitefirm.com\/blog\/wp-json\/wp\/v2\/tags?post=49331"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}