{"id":26175,"date":"2025-05-20T04:00:00","date_gmt":"2025-05-19T20:00:00","guid":{"rendered":"https:\/\/www.franchise.com.hk\/%e9%9d%96%e5%9b%a0%e8%97%a5%e6%a5%ad%e8%88%87crispr-therapeutics%e5%ae%a3%e4%bd%88%e5%90%88%e4%bd%9c%ef%bc%8c%e5%85%b1%e5%90%8c%e6%8e%a8%e9%80%b2sirna%e5%89%b5%e6%96%b0%e7%99%82%e6%b3%95%e9%96%8b\/26175"},"modified":"2025-05-20T04:00:00","modified_gmt":"2025-05-19T20:00:00","slug":"%e9%9d%96%e5%9b%a0%e8%97%a5%e6%a5%ad%e8%88%87crispr-therapeutics%e5%ae%a3%e4%bd%88%e5%90%88%e4%bd%9c%ef%bc%8c%e5%85%b1%e5%90%8c%e6%8e%a8%e9%80%b2sirna%e5%89%b5%e6%96%b0%e7%99%82%e6%b3%95%e9%96%8b","status":"publish","type":"post","link":"https:\/\/www.franchise.com.hk\/en\/%e9%9d%96%e5%9b%a0%e8%97%a5%e6%a5%ad%e8%88%87crispr-therapeutics%e5%ae%a3%e4%bd%88%e5%90%88%e4%bd%9c%ef%bc%8c%e5%85%b1%e5%90%8c%e6%8e%a8%e9%80%b2sirna%e5%89%b5%e6%96%b0%e7%99%82%e6%b3%95%e9%96%8b\/26175","title":{"rendered":"\u9756\u56e0\u85e5\u696d\u8207CRISPR Therapeutics\u5ba3\u4f48\u5408\u4f5c\uff0c\u5171\u540c\u63a8\u9032siRNA\u5275\u65b0\u7642\u6cd5\u958b\u767c"},"content":{"rendered":"

-\u8a72\u5408\u4f5c\u5c07\u96d9\u65b9\u5728\u7814\u767c\u8207\u7522\u696d\u5316\u65b9\u9762\u7684\u4e92\u88dc\u80fd\u529b\u76f8\u7d50\u5408\uff0c\u5171\u540c\u958b\u767c\u4e26\u5546\u696d\u5316\u4e0b\u4e00\u4ee3\u9577\u6548Factor XI\uff08FXI\uff09\u9776\u5411\u5c0f\u5e72\u64feRNA\uff08siRNA\uff09\u7642\u6cd5SRSD107\uff0c\u7528\u65bc\u6cbb\u7642\u8840\u6813\u53ca\u8840\u6813\u6813\u585e\u6027\u75be\u75c5\u3002<\/p>\n

-\u6839\u64da\u5354\u8b70\u689d\u6b3e\uff0c\u9756\u56e0\u85e5\u696d\u5c07\u7372\u5f97CRISPR Therapeutics\uff08\u7d0d\u65af\u9054\u514b\u80a1\u7968\u4ee3\u78bc\uff1aCRSP\uff09\u652f\u4ed8\u76849500\u842c\u7f8e\u5143\u73fe\u91d1\u53ca\u73fe\u91d1\u7b49\u50f9\u7269\u4f5c\u70ba\u9996\u4ed8\u6b3e\uff0c\u4e26\u6709\u8cc7\u683c\u7372\u5f97\u8d858\u5104\u7f8e\u5143\u7684\u9810\u4ed8\u6b3e\u548c\u91cc\u7a0b\u7891\u4ed8\u6b3e\u3002\u96d9\u65b9\u5c07\u4ee550-50\u7684\u6210\u672c\u548c\u5229\u6f64\u5206\u6524\u6a5f\u5236\u5171\u540c\u958b\u767cSRSD107\u3002\u6b64\u5916\uff0c\u5354\u8b70\u9084\u6388\u4e88CRISPR Therapeutics\u5728\u672a\u4f86\u7368\u5bb6\u5f15\u9032\u6700\u591a\u5169\u500b\u984d\u5916siRNA\u9805\u76ee\u7684\u512a\u5148\u6388\u6b0a\u6b0a\u5229\u3002<\/p>\n

\u8056\u5730\u4e9e\u54e5\u3001\u4e0a\u6d77\u3001\u745e\u58eb\u695a\u683c\u548c\u6ce2\u58eb\u9813<\/span>2025\u5e745\u670820\u65e5<\/span> \/\u7f8e\u901a\u793e\/ — \u9756\u56e0\u85e5\u696d\u662f\u4e00\u5bb6\u8655\u65bc\u81e8\u5e8a\u968e\u6bb5\u3001\u5c08\u6ce8\u65bc\u958b\u767c\u9762\u5411\u5168\u7403\u5e02\u5834\u5275\u65b0\u5c0f\u5e72\u64feRNA\uff08siRNA\uff09\u7642\u6cd5\u7684\u751f\u7269\u6280\u8853\u516c\u53f8\uff1bCRISPR Therapeutics\uff08\u7d0d\u65af\u9054\u514b\u80a1\u7968\u4ee3\u78bc\uff1aCRSP\uff09\u662f\u4e00\u5bb6\u5c08\u6ce8\u65bc\u70ba\u56b4\u91cd\u75be\u75c5\u958b\u767c\u8b8a\u9769\u6027\u57fa\u56e0\u7642\u6cd5\u7684\u751f\u7269\u88fd\u85e5\u516c\u53f8\u3002\u4eca\u65e5\uff0c\u96d9\u65b9\u5ba3\u4f48\u9054\u6210\u6230\u7565\u5408\u4f5c\u5925\u4f34\u95dc\u4fc2\uff0c\u5c07\u651c\u624b\u63a8\u9032siRNA\u7642\u6cd5\u7684\u806f\u5408\u958b\u767c\u8207\u5546\u696d\u5316\u9032\u7a0b\u3002<\/p>\n

\u9756\u56e0\u85e5\u696d\u57f7\u884c\u8463\u4e8b\u9996\u5e2d\u57f7\u884c\u5b98\u5180\u7fa4\u5347\u535a\u58eb\u8868\u793a\uff1a\u300c\u6211\u5011\u975e\u5e38\u9ad8\u8208\u8207CRISPR\u9054\u6210\u6b64\u6b21\u5408\u4f5c\u3002CRISPR Therapeutics\u662f\u57fa\u56e0\u7642\u6cd5\u958b\u767c\u9818\u57df\u516c\u8a8d\u7684\u5168\u7403\u9818\u5c0e\u8005\u3002\u8840\u6813\u6027\u75be\u75c5\u5b58\u5728\u5927\u91cf\u672a\u88ab\u6eff\u8db3\u7684\u91ab\u7642\u9700\u6c42\uff0cSRSD107\u5c55\u793a\u7684\u4ee4\u4eba\u9f13\u821e\u76841\u671f\u81e8\u5e8a\u6578\u64da\uff0c\u9032\u4e00\u6b65\u9a57\u8b49\u4e86\u5176\u4f5c\u70ba\u9776\u5411\u51dd\u8840\u56e0\u5b50XI\uff08FXI\uff09\u540c\u985e\u6700\u4f73\u7642\u6cd5\u7684\u6f5b\u529b\u3002\u5180\u535a\u58eb\u88dc\u5145\u9053\uff0c \u300c\u9756\u56e0\u85e5\u696d\u81f4\u529b\u65bc\u70ba\u5168\u7403\u60a3\u8005\u63d0\u4f9b\u5275\u65b0\u6cbb\u7642\u65b9\u6848\u3002\u6b64\u6b21\u5408\u4f5c\u5c07\u5145\u5206\u767c\u63ee\u96d9\u65b9\u512a\u52e2\uff0c\u651c\u624b\u52a0\u901f\u65b0\u4e00\u4ee3siRNA\u7642\u6cd5\u7684\u5168\u7403\u958b\u767c\u8207\u81e8\u5e8a\u8f49\u5316\u3002\u300d<\/p>\n

CRISPR Therapeutics\u8463\u4e8b\u9577\u517c\u9996\u5e2d\u57f7\u884c\u5b98Samarth Kulkarni\u535a\u58eb\u8868\u793a\uff1a\u300c\u6211\u5011\u5f88\u9ad8\u8208\u8207\u9756\u56e0\u85e5\u696d\u5efa\u7acb\u5408\u4f5c\u95dc\u4fc2\uff0c\u4e26\u5728\u8fd1\u671f\u516c\u4f48\u7684CTX310\u9805\u76ee\uff08\u9776\u5411ANGPTL3\uff09\u4ee4\u4eba\u632f\u596e\u7684\u9802\u7dda\u6578\u64da\u57fa\u790e\u4e0a\uff0c\u9032\u4e00\u6b65\u62d3\u5c55\u6211\u5011\u5728\u5fc3\u8840\u7ba1\u75be\u75c5\u9818\u57df\u7684\u7ba1\u7dda\u4f48\u5c40\u3002\u300d\u51dd\u8840\u56e0\u5b50XI\u662f\u4e00\u500b\u5275\u65b0\u4e14\u6975\u5177\u5438\u5f15\u529b\u7684\u9776\u9ede\uff0c\u5728\u6cbb\u7642\u5f71\u97ff\u5168\u7403\u6578\u4ee5\u767e\u842c\u8a08\u60a3\u8005\u7684\u8840\u6813\u6027\u75be\u75c5\u65b9\u9762\u5c55\u73fe\u51fa\u5de8\u5927\u6f5b\u529b\u3002\u4ed6\u9032\u4e00\u6b65\u6307\u51fa\uff1a\u300cSRSD107\u6709\u671b\u6210\u70ba\u540c\u985e\u6700\u4f73\u7642\u6cd5\uff0c\u6191\u501f\u66f4\u4f4e\u7684\u7d66\u85e5\u983b\u7387\u548c\u66f4\u512a\u7684\u81e8\u5e8a\u7372\u76ca\uff0c\u70ba\u81e8\u5e8a\u6cbb\u7642\u5e36\u4f86\u91cd\u5927\u7a81\u7834\u3002\u9756\u56e0\u85e5\u696d\u5728siRNA\u5e73\u53f0\u65b9\u9762\u7684\u524d\u6cbf\u6280\u8853\u8207\u6211\u5011\u73fe\u6709\u7684\u7814\u767c\u80fd\u529b\u9ad8\u5ea6\u4e92\u88dc\uff0c\u9032\u4e00\u6b65\u8c50\u5bcc\u4e86\u6211\u5011\u7684\u6cbb\u7642\u6280\u8853\u5e73\u53f0\uff0c\u52a9\u529b\u6211\u5011\u958b\u767c\u66f4\u5ee3\u6cdb\u7684\u8b8a\u9769\u6027\u57fa\u56e0\u7642\u6cd5\u3002\u300d<\/p>\n

\u300c\u7531\u65bc\u5fc3\u8840\u7ba1\u75be\u75c5\u3001\u60e1\u6027\u816b\u7624\u3001\u548c\u9ad8\u51dd\u72c0\u614b\u7b49\u57fa\u790e\u75be\u75c5\u7684\u5b58\u5728\uff0c\u5927\u91cf\u60a3\u8005\u9762\u81e8\u6f5b\u5728\u7684\u81f4\u6b7b\u6027\u8840\u6813\u6813\u585e\u6027\u4e8b\u4ef6\u98a8\u96aa\u3002\u5176\u4e2d\u76f8\u7576\u4e00\u90e8\u5206\u60a3\u8005\u7531\u65bc\u64d4\u6182\u51fa\u8840\u98a8\u96aa\u6216\u7528\u85e5\u4f9d\u5f9e\u6027\u554f\u984c\uff0c\u672a\u80fd\u7372\u5f97\u6cbb\u7642\u6216\u5145\u5206\u6cbb\u7642\u3002\u300dTIMI\u7814\u7a76\u7d44\u8cc7\u6df1\u7814\u7a76\u54e1\u3001\u5e03\u88e1\u683c\u59c6\u5a66\u79d1\u91ab\u9662\u5fc3\u81df\u75c5\u79d1\u4e3b\u4efb\u3001\u54c8\u4f5b\u91ab\u5b78\u9662\u526f\u6559\u6388Christian T. Ruff\u91ab\u5b78\u535a\u58eb\u8868\u793a\uff0c\u300cSRSD107\u6709\u671b\u6210\u70ba\u4e00\u6b3e\u5177\u6709\u5dee\u7570\u5316\u512a\u52e2\u7684\u7642\u6cd5\uff0c\u5176\u7279\u9ede\u5305\u62ec\u51fa\u8840\u98a8\u96aa\u66f4\u4f4e\u3001\u7d66\u85e5\u983b\u7387\u66f4\u4f4e\uff0c\u60a3\u8005\u4f9d\u5f9e\u6027\u66f4\u9ad8\u3001\u7121\u814e\u81df\u6e05\u9664\u6216\u85e5\u7269\u76f8\u4e92\u4f5c\u7528\u64d4\u6182\u3001\u4e26\u4e14\u5177\u5099\u66f4\u4f73\u7684\u53ef\u9006\u6027\uff0c\u5f9e\u800c\u9032\u4e00\u6b65\u964d\u4f4e\u51fa\u8840\u98a8\u96aa\u7684\u53ef\u80fd\u6027\u3002\u9019\u4e9b\u512a\u52e2\u4f7f\u5176\u6709\u671b\u5728\u73fe\u6709\u7642\u6cd5\u53ca\u9776\u5411FXI\u5176\u5b83\u7642\u6cd5\u4e2d\u812b\u7a4e\u800c\u51fa\u3002\u300d<\/p>\n

SRSD107\u662f\u4e00\u6b3e\u65b0\u4e00\u4ee3\u7684\u9577\u6548siRNA\u7642\u6cd5\uff0c\u65e8\u5728\u9078\u64c7\u6027\u6291\u5236\u51dd\u8840\u56e0\u5b50XI\uff08FXI\uff09\u3002FXI\u9776\u9ede\u5728\u75c5\u7406\u6027\u8840\u6813\u5f62\u6210\u4e2d\u8d77\u95dc\u9375\u4f5c\u7528\uff0c\u4f46\u5c0d\u6b63\u5e38\u6b62\u8840\u529f\u80fd\u7684\u5f71\u97ff\u8f03\u5c0f\u3002\u901a\u904e\u9776\u5411FXI\uff0cSRSD107\u6709\u671b\u5728\u964d\u4f4e\u8840\u6813\u4e8b\u4ef6\u767c\u751f\u7684\u540c\u6642\uff0c\u986f\u8457\u6e1b\u5c11\u51fa\u8840\u98a8\u96aa\uff0c\u5c55\u73fe\u51fa\u6709\u5225\u8207\u51dd\u8840\u56e0\u5b50X\u6d3b\u6027\uff08FXa\uff09\u6291\u5236\u5291\u7684\u6cbb\u7642\u512a\u52e2\u3002SRSD107\u7684\u6f5b\u5728\u9069\u61c9\u75c7\u5ee3\u6cdb\uff0c\u5305\u62ec\u623f\u986b\u3001\u975c\u8108\u8840\u6813\u6813\u585e\u75c7\uff08VTE\uff09\u3001\u816b\u7624\u76f8\u95dc\u6027\u8840\u6813\u3001\u63a5\u53d7\u8840\u6db2\u900f\u6790\u7684\u7d42\u672b\u671f\u814e\u75c5\u60a3\u8005\uff0c\u4ee5\u53ca\u56e0\u51fa\u8840\u98a8\u96aa\u9650\u5236\u73fe\u6709\u6cbb\u7642\u624b\u6bb5\u7684\u91cd\u5927\u9aa8\u79d1\u624b\u8853\u4eba\u7fa4\u3002<\/p>\n

\u76ee\u524d\uff0cSRSD107\u5df2\u5b8c\u6210\u5169\u98051\u671f\u81e8\u5e8a\u8a66\u9a57\uff0c\u7d50\u679c\u986f\u793a\u55ae\u6b21\u7d66\u85e5\u5b89\u5168\u6027\u4e14\u8010\u53d7\u6027\u826f\u597d\u3002\u76f8\u95dc\u7814\u7a76\u6210\u679c\u5df2\u57282025\u5e74\u7f8e\u570b\u5fc3\u81df\u75c5\u5b78\u6703\uff08ACC\uff09\u5e74\u6703\u548c2024\u5e74\u7f8e\u570b\u8840\u6db2\u5b78\u6703(ASH)\u5e74\u6703\u4e0a\u767c\u4f48\u3002SRSD107\u76842\u671f\u81e8\u5e8a\u8a66\u9a57\u6b63\u5728\u555f\u52d5\uff0c\u65e8\u5728\u8a55\u4f30\u5176\u5728\u819d\u95dc\u7bc0\u7f6e\u63db\u8853\u60a3\u8005\u4e2d\u9810\u9632\u975c\u8108\u8840\u6813\u6813\u585e\u75c7\uff08VTE\uff09\u7684\u5b89\u5168\u6027\u548c\u6709\u6548\u6027\u3002\u8a72\u7814\u7a76\u5c07\u70ba\u78ba\u8a8d\u5176\u6297\u51dd\u81e8\u5e8a\u7372\u76ca\u53ca\u5f8c\u7e8c\u95dc\u9375\u6027\u8a66\u9a57\u7684\u5291\u91cf\u9078\u64c7\u63d0\u4f9b\u81e8\u5e8a\u79d1\u5b78\u4f9d\u64da\u3002<\/p>\n

\u96d9\u65b9\u5c07\u4ee550:50\u7684\u6210\u672c\u8207\u5229\u6f64\u5206\u6210\u6a21\u5f0f\uff0c\u5171\u540c\u63a8\u9032SRSD107\u7684\u958b\u767c\u3002\u6839\u64da\u5354\u8b70\uff0cCRISPR\u5c07\u8ca0\u8cac\u8a72\u7522\u54c1\u5728\u7f8e\u570b\u7684\u5546\u696d\u5316\uff0c\u9756\u56e0\u5247\u8ca0\u8cac\u5927\u4e2d\u83ef\u5340\u5e02\u5834\u3002<\/p>\n

\u6b64\u5916\uff0cCRISPR\u64c1\u6709\u63d0\u540d\u6700\u591a\u5169\u500bsiRNA\u9776\u9ede\u7684\u6b0a\u5229\uff0c\u4e26\u53ef\u9078\u64c7\u4e3b\u5c0e\u5f8c\u7e8c\u7684\u81e8\u5e8a\u958b\u767c\u8207\u5546\u696d\u5316\u3002\u9756\u56e0\u5c07\u6709\u8cc7\u683c\u7372\u5f97\u8207\u9805\u76ee\u9032\u5c55\u639b\u9264\u7684\u91cc\u7a0b\u7891\u4ed8\u6b3e\u3001\u9644\u5e36\u689d\u4ef6\u7684\u6fc0\u52f5\u4ed8\u6b3e\uff0c\u4ee5\u53ca\u6309\u92b7\u552e\u984d\u5206\u7d1a\u8a08\u7b97\u7684\u7248\u7a05\u6536\u5165\u3002<\/p>\n

\u95dc\u65bc\u8840\u7ba1\u6813\u585e<\/p>\n

\u8840\u6813\u5f62\u6210\u662f\u8840\u7ba1\u5167\u9650\u5236\u8840\u6db2\u6d41\u52d5\u7684\u8840\u51dd\u584a\uff0c\u53ef\u767c\u751f\u5728\u52d5\u8108\u6216\u975c\u8108\u5faa\u74b0\u4e2d\uff0c\u662f\u5927\u591a\u6578\u5fc3\u808c\u6897\u6b7b\u3001\u7f3a\u8840\u6027\u8166\u5352\u4e2d\u548c\u975c\u8108\u8840\u6813\u6813\u585e\u75c7\uff08VTE\uff09\u7684\u5171\u540c\u75c5\u7406\u57fa\u790e\u3002\u5168\u7403\u6709\u56db\u5206\u4e4b\u4e00\u7684\u4eba\u6b7b\u65bc\u8840\u6813\u6813\u585e\u5f15\u767c\u7684\u75be\u75c5\u3002[1]<\/p>\n

\u95dc\u65bcSRSD107\u6ce8\u5c04\u6db2<\/p>\n

SRSD107\u6ce8\u5c04\u6db2\u662f\u4e00\u6b3e\u9756\u56e0\u85e5\u696d\u64c1\u6709\u81ea\u4e3b\u77e5\u8b58\u7522\u6b0a\u7684\u96d9\u93c8\u5c0f\u5e72\u64fe\u6838\u9178\uff08siRNA\uff09\u85e5\u7269\u3002\u901a\u904e\u7279\u7570\u6027\u809d\u9776\u5411\u4eba\u51dd\u8840\u56e0\u5b50XI\uff08FXI \uff09mRNA\uff0c\u6291\u5236FXI\u7684\u86cb\u767d\u8868\u9054\uff0c\u963b\u65b7\u5167\u6e90\u6027\u51dd\u8840\u9014\u5f91\u7684\u6fc0\u6d3b\uff0c\u5f9e\u800c\u9054\u5230\u6297\u51dd\u8840\u4f5c\u7528\u3002\u81e8\u5e8a\u524d\u8a66\u9a57\u6578\u64da\u986f\u793a\uff0c\u55ae\u6b21\u76ae\u4e0b\u6ce8\u5c04SRSD107\uff0c\u53ef\u964d\u4f4e\u5916\u5468\u8840FXI\u6fc3\u5ea6\u8fd1100%\uff0c\u4e14\u6301\u7e8c\u6642\u9593\u53ef\u9577\u9054\u534a\u5e74\uff0c\u540c\u6642\u672a\u898b\u51fa\u8840\u3002SRSD107\u517c\u5177\u5f37\u6548\u6301\u4e45\u4f5c\u7528\u548c\u826f\u597d\u7684\u5b89\u5168\u6027\uff0c\u6709\u671b\u6210\u70ba\u6f5b\u5728\u7684\u540c\u985e\u9996\u5275\uff08First-in Class\uff09\u548c\u540c\u985e\u6700\u4f73\uff08Best-in-Class\uff09\u65b0\u4e00\u4ee3\u66f4\u5b89\u5168\u7684\u6297\u51dd\u85e5\u7269\u3002<\/p>\n

\u95dc\u65bc\u9756\u56e0\u85e5\u696d<\/p>\n

\u9756\u56e0\u85e5\u696d\u00a0(Sirius Therapeutics) \u662f\u4e00\u5bb6\u81e8\u5e8a\u968e\u6bb5\u7684\u751f\u7269\u6280\u8853\u516c\u53f8\uff0c\u4ee5\u4eba\u985e\u5065\u5eb7\u798f\u7949\u70ba\u4f7f\u547d\uff0c\u81f4\u529b\u958b\u767c\u985b\u8986\u6162\u75c5\u9632\u6cbb\u7684siRNA\u65b0\u7642\u6cd5\u7684\u9818\u8ecd\u8005\u3002\u76ee\u524d\u5df2\u9032\u5165\u81e8\u5e8a\u958b\u767c\u968e\u6bb5\u7684\u7522\u54c1\u5305\u62ec\u7528\u65bc\u6cbb\u7642\u8840\u6813\u6813\u585e\u6027\u75be\u75c5\u7684SRSD107\uff0c\u9ad8\u8102\u86cb\u767d(a)\u7684SRSD216\uff0c\u548c\u8840\u8102\u7570\u5e38\u7684SRSD101\u3002<\/p>\n

\u6210\u7acb\u65bc2021\u5e74\uff0c\u7531\u570b\u969b\u5353\u8d8a\u7684\u7ba1\u7406\u5718\u968a\u548c\u5168\u7403\u77e5\u540d\u91ab\u7642\u5065\u5eb7\u6295\u8cc7\u6a5f\u69cb\u5b75\u5316\u3002\u516c\u53f8\u63a1\u53d6\u570b\u969b\u5316\u7684\u6230\u7565\u5b9a\u4f4d\uff0c\u532f\u805a\u4e2d\u7f8e\u5169\u5730\u5728\u5c0f\u6838\u9178\u7642\u6cd5\u9818\u57df\u7684\u4eba\u624d\u548c\u8cc7\u6e90\u7684\u512a\u52e2\uff0c\u5efa\u7acb\u4e86\u4ee5\u7f8e\u570b\u70ba\u6e90\u982d\u5275\u65b0\u767c\u73fe\u4e2d\u5fc3\uff0c\u4e2d\u570b\u70ba\u5168\u7403\u8f49\u5316\u91ab\u5b78\u4e2d\u5fc3\u7684\u4f48\u5c40\u3002\u5229\u7528\u5176\u5c0f\u6838\u9178\u85e5\u7269\u7814\u767c\u6838\u5fc3\u6280\u8853\u5e73\u53f0\uff0c\u958b\u767c\u4e86\u591a\u500b\u5177\u6709\u540c\u985e\u9996\u5275\u6216\u540c\u985e\u6700\u4f73\u6f5b\u8cea\u7684\u5dee\u7570\u5316\u5168\u7403\u7af6\u722d\u512a\u52e2\u7684\u7522\u54c1\u7ba1\u7dda\u3002\u516c\u53f8\u5df2\u7d2f\u8a08\u878d\u8cc7\u8fd11.5\u5104\u7f8e\u5143\u3002\u6b32\u77ad\u89e3\u66f4\u591a\u4fe1\u606f\uff0c\u8acb\u8a2a\u554fwww.siriusrna.com<\/a><\/p>\n

\u95dc\u65bcCRISPR Therapeutics<\/p>\n

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\u5f15\u7528\u6587\u737b\uff1a<\/p>\n

1. Lozano R, Naghavi M, Foreman K, et al. Global and regional mortality from 235 causes of death for 20 age groups in 1990 and 2010: a systematic analysis for the Global Burden of Disease Study 2010. Lancet. 2012;380, 2095-1128.<\/p>\n

\u5a92\u9ad4\u806f\u7e6b\u4eba\uff1a <\/p>\n

+86 21 61207756
info@siriusrna.com<\/a><\/p>\n

\u00a0<\/p>","protected":false},"excerpt":{"rendered":"

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\u95dc\u65bcCRISPR Therapeutics \u81ea\u5341\u591a\u5e74\u524d\u6210\u7acb\u4ee5\u4f86\uff0cCRISPR Therapeutics\u5df2\u5f9e\u4e00\u5bb6\u81f4\u529b\u65bc\u63a8\u9032\u57fa\u56e0\u7de8\u8f2f\u8a08\u5283\u7684\u7814\u7a76\u578b\u516c\u53f8\u767c\u5c55\u6210\u70ba\u4e00\u5bb6\u884c\u696d\u9818\u5c0e\u8005\uff0c\u4e26\u6176\u795d\u4e86\u5168\u7403\u9996\u500bCRISPR\u57fa\u56e0\u7642\u6cd5\u7372\u6279\u7684\u6b77\u53f2\u6027\u6642\u523b\u3002\u8a72\u516c\u53f8\u64c1\u6709\u591a\u5143\u5316\u7684\u7522\u54c1\u5019\u9078\u7d44\u5408\uff0c\u6db5\u84cb\u5ee3\u6cdb\u7684\u75be\u75c5\u9818\u57df\uff0c\u5305\u62ec\u8840\u7d05\u86cb\u767d\u75c5\u3001\u816b\u7624\u5b78\u3001\u518d\u751f\u91ab\u5b78\u3001\u5fc3\u8840\u7ba1\u75be\u75c5\u3001\u81ea\u8eab\u514d\u75ab\u75be\u75c5\u548c\u7f55\u898b\u75c5\u30022018\u5e74\uff0cCRISPR 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[exa-cel]\uff09\u5728\u591a\u500b\u570b\u5bb6\u7372\u6279\uff0c\u7528\u65bc\u6cbb\u7642\u7b26\u5408\u689d\u4ef6\u7684\u942e\u72c0\u7d30\u80de\u75c5\u6216\u8f38\u8840\u4f9d\u8cf4\u6027\u03b2\u5730\u4e2d\u6d77\u8ca7\u8840\u60a3\u8005\u3002\u8afe\u8c9d\u723e\u734e\u5f97\u4e3b\u7684CRISPR\u6280\u8853\u5fb9\u5e95\u6539\u8b8a\u4e86\u751f\u7269\u91ab\u5b78\u7814\u7a76\uff0c\u4ee3\u8868\u4e86\u4e00\u7a2e\u7d93\u904e\u81e8\u5e8a\u9a57\u8b49\u7684\u5f37\u5927\u65b9\u6cd5\uff0c\u5177\u6709\u6f5b\u529b\u5275\u9020\u4e00\u985e\u5168\u65b0\u7684\u3001\u53ef\u80fd\u5177\u6709\u8b8a\u9769\u6027\u7684\u85e5\u7269\u3002\u70ba\u4e86\u52a0\u901f\u548c\u64f4\u5927\u5176\u52aa\u529b\uff0cCRISPR Therapeutics\u8207Vertex Pharmaceuticals\u7b49\u9818\u5148\u516c\u53f8\u5efa\u7acb\u4e86\u6230\u7565\u5408\u4f5c\u5925\u4f34\u95dc\u4fc2\u3002CRISPR Therapeutics AG\u7e3d\u90e8\u4f4d\u65bc\u745e\u58eb\u695a\u683c\uff0c\u5176\u5168\u8cc7\u5b50\u516c\u53f8CRISPR Therapeutics, Inc.\u4f4d\u65bc\u7f8e\u570b\uff0c\u7814\u767c\u696d\u52d9\u5247\u8a2d\u5728\u7f8e\u570b\u99ac\u85a9\u8af8\u585e\u5dde\u6ce2\u58eb\u9813\u548c\u52a0\u5229\u798f\u5c3c\u4e9e\u5dde\u820a\u91d1\u5c71\u3002\u6b32\u77ad\u89e3\u66f4\u591a\u4fe1\u606f\uff0c\u8acb\u8a2a\u554fwww.crisprtx.com\u3002 \u5f15\u7528\u6587\u737b\uff1a 1. Lozano R, Naghavi M, Foreman K, et al. Global and regional mortality from 235 causes of death for 20 age groups in 1990 and 2010: a systematic analysis for the Global Burden of Disease Study 2010. Lancet. 2012;380, 2095-1128. \u5a92\u9ad4\u806f\u7e6b\u4eba\uff1a +86 21 61207756info@siriusrna.com \u00a0<\/p>\n","protected":false},"author":0,"featured_media":0,"comment_status":"","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"_generate-full-width-content":"","spay_email":"","footnotes":""},"categories":[1],"tags":[],"yoast_head":"\n\u9756\u56e0\u85e5\u696d\u8207CRISPR Therapeutics\u5ba3\u4f48\u5408\u4f5c\uff0c\u5171\u540c\u63a8\u9032siRNA\u5275\u65b0\u7642\u6cd5\u958b\u767c - \u9999\u6e2f\u7279\u8a31\u7d93\u71df<\/title>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/www.franchise.com.hk\/en\/%e9%9d%96%e5%9b%a0%e8%97%a5%e6%a5%ad%e8%88%87crispr-therapeutics%e5%ae%a3%e4%bd%88%e5%90%88%e4%bd%9c%ef%bc%8c%e5%85%b1%e5%90%8c%e6%8e%a8%e9%80%b2sirna%e5%89%b5%e6%96%b0%e7%99%82%e6%b3%95%e9%96%8b\/26175\" \/>\n<meta property=\"og:locale\" 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\uff09mRNA\uff0c\u6291\u5236FXI\u7684\u86cb\u767d\u8868\u9054\uff0c\u963b\u65b7\u5167\u6e90\u6027\u51dd\u8840\u9014\u5f91\u7684\u6fc0\u6d3b\uff0c\u5f9e\u800c\u9054\u5230\u6297\u51dd\u8840\u4f5c\u7528\u3002\u81e8\u5e8a\u524d\u8a66\u9a57\u6578\u64da\u986f\u793a\uff0c\u55ae\u6b21\u76ae\u4e0b\u6ce8\u5c04SRSD107\uff0c\u53ef\u964d\u4f4e\u5916\u5468\u8840FXI\u6fc3\u5ea6\u8fd1100%\uff0c\u4e14\u6301\u7e8c\u6642\u9593\u53ef\u9577\u9054\u534a\u5e74\uff0c\u540c\u6642\u672a\u898b\u51fa\u8840\u3002SRSD107\u517c\u5177\u5f37\u6548\u6301\u4e45\u4f5c\u7528\u548c\u826f\u597d\u7684\u5b89\u5168\u6027\uff0c\u6709\u671b\u6210\u70ba\u6f5b\u5728\u7684\u540c\u985e\u9996\u5275\uff08First-in Class\uff09\u548c\u540c\u985e\u6700\u4f73\uff08Best-in-Class\uff09\u65b0\u4e00\u4ee3\u66f4\u5b89\u5168\u7684\u6297\u51dd\u85e5\u7269\u3002 \u95dc\u65bc\u9756\u56e0\u85e5\u696d \u9756\u56e0\u85e5\u696d\u00a0(Sirius Therapeutics) \u662f\u4e00\u5bb6\u81e8\u5e8a\u968e\u6bb5\u7684\u751f\u7269\u6280\u8853\u516c\u53f8\uff0c\u4ee5\u4eba\u985e\u5065\u5eb7\u798f\u7949\u70ba\u4f7f\u547d\uff0c\u81f4\u529b\u958b\u767c\u985b\u8986\u6162\u75c5\u9632\u6cbb\u7684siRNA\u65b0\u7642\u6cd5\u7684\u9818\u8ecd\u8005\u3002\u76ee\u524d\u5df2\u9032\u5165\u81e8\u5e8a\u958b\u767c\u968e\u6bb5\u7684\u7522\u54c1\u5305\u62ec\u7528\u65bc\u6cbb\u7642\u8840\u6813\u6813\u585e\u6027\u75be\u75c5\u7684SRSD107\uff0c\u9ad8\u8102\u86cb\u767d(a)\u7684SRSD216\uff0c\u548c\u8840\u8102\u7570\u5e38\u7684SRSD101\u3002 \u6210\u7acb\u65bc2021\u5e74\uff0c\u7531\u570b\u969b\u5353\u8d8a\u7684\u7ba1\u7406\u5718\u968a\u548c\u5168\u7403\u77e5\u540d\u91ab\u7642\u5065\u5eb7\u6295\u8cc7\u6a5f\u69cb\u5b75\u5316\u3002\u516c\u53f8\u63a1\u53d6\u570b\u969b\u5316\u7684\u6230\u7565\u5b9a\u4f4d\uff0c\u532f\u805a\u4e2d\u7f8e\u5169\u5730\u5728\u5c0f\u6838\u9178\u7642\u6cd5\u9818\u57df\u7684\u4eba\u624d\u548c\u8cc7\u6e90\u7684\u512a\u52e2\uff0c\u5efa\u7acb\u4e86\u4ee5\u7f8e\u570b\u70ba\u6e90\u982d\u5275\u65b0\u767c\u73fe\u4e2d\u5fc3\uff0c\u4e2d\u570b\u70ba\u5168\u7403\u8f49\u5316\u91ab\u5b78\u4e2d\u5fc3\u7684\u4f48\u5c40\u3002\u5229\u7528\u5176\u5c0f\u6838\u9178\u85e5\u7269\u7814\u767c\u6838\u5fc3\u6280\u8853\u5e73\u53f0\uff0c\u958b\u767c\u4e86\u591a\u500b\u5177\u6709\u540c\u985e\u9996\u5275\u6216\u540c\u985e\u6700\u4f73\u6f5b\u8cea\u7684\u5dee\u7570\u5316\u5168\u7403\u7af6\u722d\u512a\u52e2\u7684\u7522\u54c1\u7ba1\u7dda\u3002\u516c\u53f8\u5df2\u7d2f\u8a08\u878d\u8cc7\u8fd11.5\u5104\u7f8e\u5143\u3002\u6b32\u77ad\u89e3\u66f4\u591a\u4fe1\u606f\uff0c\u8acb\u8a2a\u554fwww.siriusrna.com \u95dc\u65bcCRISPR Therapeutics \u81ea\u5341\u591a\u5e74\u524d\u6210\u7acb\u4ee5\u4f86\uff0cCRISPR Therapeutics\u5df2\u5f9e\u4e00\u5bb6\u81f4\u529b\u65bc\u63a8\u9032\u57fa\u56e0\u7de8\u8f2f\u8a08\u5283\u7684\u7814\u7a76\u578b\u516c\u53f8\u767c\u5c55\u6210\u70ba\u4e00\u5bb6\u884c\u696d\u9818\u5c0e\u8005\uff0c\u4e26\u6176\u795d\u4e86\u5168\u7403\u9996\u500bCRISPR\u57fa\u56e0\u7642\u6cd5\u7372\u6279\u7684\u6b77\u53f2\u6027\u6642\u523b\u3002\u8a72\u516c\u53f8\u64c1\u6709\u591a\u5143\u5316\u7684\u7522\u54c1\u5019\u9078\u7d44\u5408\uff0c\u6db5\u84cb\u5ee3\u6cdb\u7684\u75be\u75c5\u9818\u57df\uff0c\u5305\u62ec\u8840\u7d05\u86cb\u767d\u75c5\u3001\u816b\u7624\u5b78\u3001\u518d\u751f\u91ab\u5b78\u3001\u5fc3\u8840\u7ba1\u75be\u75c5\u3001\u81ea\u8eab\u514d\u75ab\u75be\u75c5\u548c\u7f55\u898b\u75c5\u30022018\u5e74\uff0cCRISPR Therapeutics\u5c07\u9996\u500bCRISPR\/Cas9\u57fa\u56e0\u7de8\u8f2f\u7642\u6cd5\u63a8\u9032\u81e8\u5e8a\uff0c\u7528\u65bc\u7814\u7a76\u942e\u72c0\u7d30\u80de\u75c5\u548c\u8f38\u8840\u4f9d\u8cf4\u6027\u03b2\u5730\u4e2d\u6d77\u8ca7\u8840\u7684\u6cbb\u7642\u3002\u81ea2023\u5e74\u5e95\u8d77\uff0cCASGEVY\u00ae\uff08exagamglogene autotemcel [exa-cel]\uff09\u5728\u591a\u500b\u570b\u5bb6\u7372\u6279\uff0c\u7528\u65bc\u6cbb\u7642\u7b26\u5408\u689d\u4ef6\u7684\u942e\u72c0\u7d30\u80de\u75c5\u6216\u8f38\u8840\u4f9d\u8cf4\u6027\u03b2\u5730\u4e2d\u6d77\u8ca7\u8840\u60a3\u8005\u3002\u8afe\u8c9d\u723e\u734e\u5f97\u4e3b\u7684CRISPR\u6280\u8853\u5fb9\u5e95\u6539\u8b8a\u4e86\u751f\u7269\u91ab\u5b78\u7814\u7a76\uff0c\u4ee3\u8868\u4e86\u4e00\u7a2e\u7d93\u904e\u81e8\u5e8a\u9a57\u8b49\u7684\u5f37\u5927\u65b9\u6cd5\uff0c\u5177\u6709\u6f5b\u529b\u5275\u9020\u4e00\u985e\u5168\u65b0\u7684\u3001\u53ef\u80fd\u5177\u6709\u8b8a\u9769\u6027\u7684\u85e5\u7269\u3002\u70ba\u4e86\u52a0\u901f\u548c\u64f4\u5927\u5176\u52aa\u529b\uff0cCRISPR Therapeutics\u8207Vertex Pharmaceuticals\u7b49\u9818\u5148\u516c\u53f8\u5efa\u7acb\u4e86\u6230\u7565\u5408\u4f5c\u5925\u4f34\u95dc\u4fc2\u3002CRISPR Therapeutics AG\u7e3d\u90e8\u4f4d\u65bc\u745e\u58eb\u695a\u683c\uff0c\u5176\u5168\u8cc7\u5b50\u516c\u53f8CRISPR Therapeutics, Inc.\u4f4d\u65bc\u7f8e\u570b\uff0c\u7814\u767c\u696d\u52d9\u5247\u8a2d\u5728\u7f8e\u570b\u99ac\u85a9\u8af8\u585e\u5dde\u6ce2\u58eb\u9813\u548c\u52a0\u5229\u798f\u5c3c\u4e9e\u5dde\u820a\u91d1\u5c71\u3002\u6b32\u77ad\u89e3\u66f4\u591a\u4fe1\u606f\uff0c\u8acb\u8a2a\u554fwww.crisprtx.com\u3002 \u5f15\u7528\u6587\u737b\uff1a 1. Lozano R, Naghavi M, Foreman K, et al. Global and regional mortality from 235 causes of death for 20 age groups in 1990 and 2010: a systematic analysis for the Global Burden of Disease Study 2010. Lancet. 2012;380, 2095-1128. \u5a92\u9ad4\u806f\u7e6b\u4eba\uff1a +86 21 61207756info@siriusrna.com \u00a0\" \/>\n<meta property=\"og:url\" content=\"https:\/\/www.franchise.com.hk\/en\/%e9%9d%96%e5%9b%a0%e8%97%a5%e6%a5%ad%e8%88%87crispr-therapeutics%e5%ae%a3%e4%bd%88%e5%90%88%e4%bd%9c%ef%bc%8c%e5%85%b1%e5%90%8c%e6%8e%a8%e9%80%b2sirna%e5%89%b5%e6%96%b0%e7%99%82%e6%b3%95%e9%96%8b\/26175\" \/>\n<meta property=\"og:site_name\" content=\"\u9999\u6e2f\u7279\u8a31\u7d93\u71df\" \/>\n<meta property=\"article:published_time\" content=\"2025-05-19T20:00:00+00:00\" \/>\n<meta name=\"twitter:card\" content=\"summary_large_image\" \/>\n<meta name=\"twitter:label1\" content=\"Est. reading time\" \/>\n\t<meta name=\"twitter:data1\" content=\"1 minute\" \/>\n<script type=\"application\/ld+json\" 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\u662f\u4e00\u5bb6\u81e8\u5e8a\u968e\u6bb5\u7684\u751f\u7269\u6280\u8853\u516c\u53f8\uff0c\u4ee5\u4eba\u985e\u5065\u5eb7\u798f\u7949\u70ba\u4f7f\u547d\uff0c\u81f4\u529b\u958b\u767c\u985b\u8986\u6162\u75c5\u9632\u6cbb\u7684siRNA\u65b0\u7642\u6cd5\u7684\u9818\u8ecd\u8005\u3002\u76ee\u524d\u5df2\u9032\u5165\u81e8\u5e8a\u958b\u767c\u968e\u6bb5\u7684\u7522\u54c1\u5305\u62ec\u7528\u65bc\u6cbb\u7642\u8840\u6813\u6813\u585e\u6027\u75be\u75c5\u7684SRSD107\uff0c\u9ad8\u8102\u86cb\u767d(a)\u7684SRSD216\uff0c\u548c\u8840\u8102\u7570\u5e38\u7684SRSD101\u3002 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\u95dc\u65bcCRISPR Therapeutics \u81ea\u5341\u591a\u5e74\u524d\u6210\u7acb\u4ee5\u4f86\uff0cCRISPR Therapeutics\u5df2\u5f9e\u4e00\u5bb6\u81f4\u529b\u65bc\u63a8\u9032\u57fa\u56e0\u7de8\u8f2f\u8a08\u5283\u7684\u7814\u7a76\u578b\u516c\u53f8\u767c\u5c55\u6210\u70ba\u4e00\u5bb6\u884c\u696d\u9818\u5c0e\u8005\uff0c\u4e26\u6176\u795d\u4e86\u5168\u7403\u9996\u500bCRISPR\u57fa\u56e0\u7642\u6cd5\u7372\u6279\u7684\u6b77\u53f2\u6027\u6642\u523b\u3002\u8a72\u516c\u53f8\u64c1\u6709\u591a\u5143\u5316\u7684\u7522\u54c1\u5019\u9078\u7d44\u5408\uff0c\u6db5\u84cb\u5ee3\u6cdb\u7684\u75be\u75c5\u9818\u57df\uff0c\u5305\u62ec\u8840\u7d05\u86cb\u767d\u75c5\u3001\u816b\u7624\u5b78\u3001\u518d\u751f\u91ab\u5b78\u3001\u5fc3\u8840\u7ba1\u75be\u75c5\u3001\u81ea\u8eab\u514d\u75ab\u75be\u75c5\u548c\u7f55\u898b\u75c5\u30022018\u5e74\uff0cCRISPR Therapeutics\u5c07\u9996\u500bCRISPR\/Cas9\u57fa\u56e0\u7de8\u8f2f\u7642\u6cd5\u63a8\u9032\u81e8\u5e8a\uff0c\u7528\u65bc\u7814\u7a76\u942e\u72c0\u7d30\u80de\u75c5\u548c\u8f38\u8840\u4f9d\u8cf4\u6027\u03b2\u5730\u4e2d\u6d77\u8ca7\u8840\u7684\u6cbb\u7642\u3002\u81ea2023\u5e74\u5e95\u8d77\uff0cCASGEVY\u00ae\uff08exagamglogene autotemcel [exa-cel]\uff09\u5728\u591a\u500b\u570b\u5bb6\u7372\u6279\uff0c\u7528\u65bc\u6cbb\u7642\u7b26\u5408\u689d\u4ef6\u7684\u942e\u72c0\u7d30\u80de\u75c5\u6216\u8f38\u8840\u4f9d\u8cf4\u6027\u03b2\u5730\u4e2d\u6d77\u8ca7\u8840\u60a3\u8005\u3002\u8afe\u8c9d\u723e\u734e\u5f97\u4e3b\u7684CRISPR\u6280\u8853\u5fb9\u5e95\u6539\u8b8a\u4e86\u751f\u7269\u91ab\u5b78\u7814\u7a76\uff0c\u4ee3\u8868\u4e86\u4e00\u7a2e\u7d93\u904e\u81e8\u5e8a\u9a57\u8b49\u7684\u5f37\u5927\u65b9\u6cd5\uff0c\u5177\u6709\u6f5b\u529b\u5275\u9020\u4e00\u985e\u5168\u65b0\u7684\u3001\u53ef\u80fd\u5177\u6709\u8b8a\u9769\u6027\u7684\u85e5\u7269\u3002\u70ba\u4e86\u52a0\u901f\u548c\u64f4\u5927\u5176\u52aa\u529b\uff0cCRISPR Therapeutics\u8207Vertex Pharmaceuticals\u7b49\u9818\u5148\u516c\u53f8\u5efa\u7acb\u4e86\u6230\u7565\u5408\u4f5c\u5925\u4f34\u95dc\u4fc2\u3002CRISPR Therapeutics AG\u7e3d\u90e8\u4f4d\u65bc\u745e\u58eb\u695a\u683c\uff0c\u5176\u5168\u8cc7\u5b50\u516c\u53f8CRISPR Therapeutics, Inc.\u4f4d\u65bc\u7f8e\u570b\uff0c\u7814\u767c\u696d\u52d9\u5247\u8a2d\u5728\u7f8e\u570b\u99ac\u85a9\u8af8\u585e\u5dde\u6ce2\u58eb\u9813\u548c\u52a0\u5229\u798f\u5c3c\u4e9e\u5dde\u820a\u91d1\u5c71\u3002\u6b32\u77ad\u89e3\u66f4\u591a\u4fe1\u606f\uff0c\u8acb\u8a2a\u554fwww.crisprtx.com\u3002 \u5f15\u7528\u6587\u737b\uff1a 1. Lozano R, Naghavi M, Foreman K, et al. Global and regional mortality from 235 causes of death for 20 age groups in 1990 and 2010: a systematic analysis for the Global Burden of Disease Study 2010. 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