{"id":306798,"date":"2019-11-26T16:38:20","date_gmt":"2019-11-27T00:38:20","guid":{"rendered":"https:\/\/nai500.com\/?p=306798"},"modified":"2019-11-26T16:38:20","modified_gmt":"2019-11-27T00:38:20","slug":"crispr-therapeutics-ctx001-%e5%9f%ba%e5%9b%a0%e7%bc%96%e8%be%91%e6%a6%82%e5%bf%b5%e8%82%a1","status":"publish","type":"post","link":"https:\/\/nai500.com\/zh-hans\/blog\/2019\/11\/crispr-therapeutics-ctx001-%e5%9f%ba%e5%9b%a0%e7%bc%96%e8%be%91%e6%a6%82%e5%bf%b5%e8%82%a1\/","title":{"rendered":"CTX001\u8bd5\u9a8c\u7ed3\u679c\u79ef\u6781\uff0cCRISPR Therapeutics\u662f\u76ee\u524d\u6700\u4f73\u7684\u57fa\u56e0\u7f16\u8f91\u6982\u5ff5\u80a1\u5417\uff1f"},"content":{"rendered":"<p>\u57fa\u56e0\u7f16\u8f91\u516c\u53f8<a href=\"https:\/\/nai500.com\/zh-hans\/\u80a1\u7968\u67e5\u8be2\/?qm_symbol=CRSP\">CRISPR Therapeutics AG\uff08NASDAQ: CRSP\uff09<\/a>\u6700\u8fd1\u53d1\u5e03\u57fa\u56e0\u7f16\u8f91\u9020\u8840\u5e72\u7ec6\u80de\u7597\u6cd5CTX001\u7684\u8bd5\u9a8c\u7ed3\u679c\u975e\u5e38\u79ef\u6781\uff0c\u53e0\u52a0\u6b64\u524dOppenheimer\u5206\u6790\u5e08\u8c03\u5347\u8be5\u80a1\u8bc4\u7ea7\uff0c\u8be5\u80a1\u80a1\u4ef7\u572811\u670819\u65e5\u5f53\u5929\u66b4\u6da8\u8d85\u8fc725%\u3002\u4e0d\u8fc7\uff0c\u6b64\u540eCRISPR\u56de\u5410\u591a\u6570\u6da8\u5e45\uff0c\u6700\u8fd1\u7684\u56de\u8c03\u662f\u6295\u8d44\u8005\u9022\u4f4e\u4e70\u5165\u8fd9\u53ea\u57fa\u56e0\u7f16\u8f91\u6982\u5ff5\u80a1\u7684\u673a\u4f1a\u5417\uff1f<\/p>\n<h2>\u57fa\u56e0\u7f16\u8f91\u836f\u7269CTX001\u7684\u8bd5\u9a8c\u7ed3\u679c<\/h2>\n<p>\u672c\u6708\u65e9\u4e9b\u65f6\u5019\uff0cCRISPR\u53d1\u5e03\u5019\u9009\u836f\u7269CTX001\u7684\u7b2c\u4e00\u4e2a\u4e34\u5e8a\u8bd5\u9a8c\u6570\u636e\uff0c\u5176\u4e2d\u5305\u62ec\u4e00\u540d\u9570\u72b6\u7ec6\u80de\u75c5\uff08SCD\uff09\u60a3\u8005\u4ee5\u53ca\u4e00\u540d\u8f93\u8840\u4f9d\u8d56\u578b\u03b2\u5730\u4e2d\u6d77\u8d2b\u8840\u60a3\u8005\u7684\u7ed3\u679c\u3002\u7ed3\u679c\u663e\u793a\uff0c\u4e24\u540d\u60a3\u8005\u5747\u62a5\u544a\u4e3b\u8981\u75c7\u72b6\u5b8c\u5168\u6d88\u5931\u3002<\/p>\n<p>\u5177\u4f53\u6765\u770b\uff0c\u9570\u72b6\u7ec6\u80de\u75c5\u60a3\u8005\u6b64\u524d\u6bcf\u5e74\u5e73\u5747\u53d1\u751f7\u6b21\u8840\u7ba1\u5835\u585e\uff08\u8840\u7ba1\u95ed\u585e\u5371\u673a\uff09\uff0c\u4f46\u5728\u6cbb\u7597\u76849\u4e2a\u6708\u671f\u95f4\uff0c\u60a3\u8005\u6ca1\u6709\u53d1\u751f\u8fc71\u6b21\u5835\u585e\u3002\u03b2\u5730\u4e2d\u6d77\u8d2b\u8840\u60a3\u8005\u6b64\u524d\u6bcf\u5e74\u5e73\u5747\u9700\u8981\u8f93\u884016.5\u6b21\uff0c\u4e0d\u8fc7\u5728\u63a5\u53d7CTX001\u6cbb\u7597\u4e24\u5e74\u540e\uff0c\u8fd9\u4e00\u6570\u5b57\u964d\u81f3\u96f6\uff0c\u5373\u5b8c\u5168\u6446\u8131\u4e86\u5bf9\u4e8e\u8f93\u8840\u7684\u4f9d\u8d56\u3002<\/p>\n<p>\u8bd5\u9a8c\u7ed3\u679c\u8fd8\u6709\u66f4\u591a\u7684\u7ec6\u8282\uff0c\u4f46\u7b80\u5355\u6765\u8bf4\uff0c\u521d\u6b65\u7ed3\u679c\u975e\u5e38\u4ee4\u4eba\u632f\u594b\u3002<\/p>\n<h2>\u6f5c\u5728\u7684\u7ade\u4e89\u5bf9\u624b<\/h2>\n<p>\u5c3d\u7ba1\u5230\u76ee\u524d\u4e3a\u6b62\uff0cCTX001\u7684\u8bd5\u9a8c\u7ed3\u679c\u79ef\u6781\uff0c\u4f46\u76f8\u8f83\u6b63\u5904\u4e8e\u5f00\u53d1\u8fc7\u7a0b\u4e2d\u7684\u5176\u4ed6\u7ade\u4e89\u836f\u7269\u9886\u5148\u4f18\u52bf\u5e76\u6ca1\u6709\u90a3\u4e48\u660e\u663e\u3002\u8840\u6db2\u75c5\u662f\u57fa\u56e0\u7f16\u8f91\u516c\u53f8\u4e3b\u653b\u7684\u4e00\u4e2a\u65b9\u5411\uff0c\u5176\u4e2d<a href=\"https:\/\/nai500.com\/zh-hans\/\u80a1\u7968\u67e5\u8be2\/?qm_symbol=EDIT\">Editas Medicine Inc\uff08NASDAQ: EDIT\uff09<\/a>\u548c<a href=\"https:\/\/nai500.com\/zh-hans\/\u80a1\u7968\u67e5\u8be2\/?qm_symbol=SGMO\">Sangamo Therapeutics Inc\uff08NASDAQ: SGMO\uff09<\/a>\u90fd\u5728\u5f00\u53d1\u81ea\u5df1\u7684\u9570\u72b6\u7ec6\u80de\u75c5\u548c\u03b2\u5730\u4e2d\u6d77\u8d2b\u8840\u6cbb\u7597\u65b9\u6cd5\u3002<\/p>\n<p>\u4eca\u5e74\u65e9\u4e9b\u65f6\u5019\uff0cEditas\u7684\u9570\u72b6\u7ec6\u80de\u75c5\u836f\u7269EDIT-301\u663e\u793a\u4e0d\u9519\u7684\u4e34\u5e8a\u524d\u7ed3\u679c\uff0c\u9884\u8ba1\u5e74\u5e95\u524d\u5c06\u63d0\u4f9b\u66f4\u591a\u7684\u7814\u7a76\u4fe1\u606f\u3002\u4e0d\u8fc7\uff0c\u8ddfCRISPR\u4e0d\u540c\u7684\u662f\uff0cEditas\u7684\u65d7\u8230\u836f\u7269\u5e76\u4e0d\u662f\u9488\u5bf9\u8840\u6db2\u75c5\u7684EDIT-301\uff0c\u800c\u662f\u4e3b\u653b\u7f55\u89c1\u513f\u7ae5\u773c\u75c5\u2014\u2014Leber\u5148\u5929\u6027\u9ed1\u6726\u7684EDIT-101\u3002Leber\u5148\u5929\u6027\u9ed1\u6726\u662f\u4e00\u79cd\u4e25\u91cd\u7684\u9057\u4f20\u6027\u89c6\u7f51\u819c\u53d8\u6027\u75be\u75c5\uff0c\u5bfc\u81f4\u5a74\u5e7c\u513f\u5148\u5929\u6027\u76f2\u3002<\/p>\n<p>\u81f3\u4e8eSangamo\uff0c\u8be5\u516c\u53f8\u7684\u836f\u7269\u7814\u53d1\u7ba1\u7ebf\u76f8\u5f53\u5bbd\uff0c\u5176\u4e2d\u5305\u62ec\u5904\u4e8e\u5f00\u53d1\u8fc7\u7a0b\u4e2d\u7684\u03b2\u5730\u4e2d\u6d77\u8d2b\u8840\u836f\u7269ST-400\u548c\u9570\u72b6\u7ec6\u80de\u75c5\u836f\u7269BIVV-003\u3002\u4e0d\u8fc7\uff0c\u8fd9\u4e24\u6b3e\u836f\u7269\u4ecd\u5904\u4e8e\u4e34\u5e8a\u8bd5\u9a8c\u975e\u5e38\u521d\u671f\u7684\u9636\u6bb5\uff0c\u5176\u4e2d\u4e00\u9879\u7814\u7a76\u62db\u52df\u4e86\u4e24\u540d\u60a3\u8005\u8bc4\u4f30ST-400\u6cbb\u7597\u03b2-\u5730\u4e2d\u6d77\u8d2b\u8840\u3002\u521d\u671f\u6570\u636e\u9884\u8ba1\u5c06\u4e8e\u5e74\u5e95\u524d\u51fa\u7089\uff0c\u751f\u7269\u79d1\u6280\u6295\u8d44\u8005\u5e94\u5f53\u4e88\u4ee5\u5173\u6ce8\u3002<\/p>\n<p>\u4e0d\u8fc7\uff0c\u8ddfEditas\u7c7b\u4f3c\u7684\u662f\uff0cSangamo\u7684\u03b2\u5730\u4e2d\u6d77\u8d2b\u8840\u548c\u9570\u72b6\u7ec6\u80de\u75c5\u7597\u6cd5\u5e76\u4e0d\u662f\u8be5\u516c\u53f8\u7684\u4e3b\u8981\u5019\u9009\u836f\u7269\u3002\u76f8\u53cd\uff0c\u8be5\u516c\u53f8\u7684\u65d7\u8230\u7597\u6cd5\u662f\u7532\u578b\u8840\u53cb\u75c5\u836f\u7269SB-525\u3002\u7532\u578b\u8840\u53cb\u75c5\u662f\u4e00\u79cd\u7f55\u89c1\u7684\u51fa\u8840\u6027\u75be\u75c5\uff0c\u81f4\u75c5\u539f\u56e0\u662f\u8840\u6d46\u4e2d\u7684\u51dd\u8840\u56e0\u5b50VIII\u4e0d\u8db3\u3002<\/p>\n<h2>CRISPR\u662f\u76ee\u524d\u6700\u4f73\u7684\u57fa\u56e0\u7f16\u8f91\u6982\u5ff5\u80a1\u5417\uff1f<\/h2>\n<p>CRISPR\u76ee\u524d\u662f\u5426\u662f\u7edd\u5bf9\u7684\u6700\u4f73\u57fa\u56e0\u7f16\u8f91\u6982\u5ff5\u80a1\u8fd8\u5b58\u7591\u3002\u8be5\u516c\u53f8\u5728\u9570\u72b6\u7ec6\u80de\u75c5\u548c\u03b2\u5730\u4e2d\u6d77\u8d2b\u8840\u6cbb\u7597\u9886\u57df\u9886\u8dd1\uff08\u672a\u6765\u51e0\u5e74\u8fd9\u4e24\u4e2a\u9886\u57df\u7684\u5e02\u573a\u89c4\u6a21\u4f30\u8ba1\u5c06\u5206\u522b\u8fbe$35\u4ebf\u548c$55\u4ebf\uff09\u3002\u4e0d\u8fc7\uff0c\u5728\u5176\u4ed6\u6cbb\u7597\u9886\u57df\uff0c\u5176\u4ed6\u57fa\u56e0\u7f16\u8f91\u516c\u53f8\u5177\u5907CRISPR\u6ca1\u6709\u7684\u4f18\u52bf\u3002Sangamo\u7684\u8840\u53cb\u75c5\u6cbb\u7597\u5c31\u662f\u4e00\u4e2a\u6781\u597d\u7684\u4f8b\u5b50\uff1a\u8be5\u9886\u57df\u7684\u5e02\u573a\u89c4\u6a21\u9884\u8ba1\u52302025\u5e74\u5c06\u8fbe$148\u4ebf\u3002<\/p>\n<p>\u4e0e\u6b64\u540c\u65f6\uff0c\u6709\u4e9b\u6740\u5165\u57fa\u56e0\u7f16\u8f91\u9886\u57df\u7684\u5236\u836f\u516c\u53f8\u5df2\u7ecf\u5f00\u59cb\u5f03\u7528CRISPR Therapeutics\u5f00\u53d1\u7684CRISPR\/Cas9\u57fa\u56e0\u7f16\u8f91\u5de5\u5177\u3002<a href=\"https:\/\/nai500.com\/zh-hans\/\u80a1\u7968\u67e5\u8be2\/?qm_symbol=NVO\">\u8bfa\u548c\u8bfa\u5fb7Novo Nordisk\uff08NYSE: NVO\uff09<\/a>\u6700\u8fd1\u5c31\u5ba3\u5e03\uff0c\u8be5\u516c\u53f8\u6ca1\u6709\u9009\u7528CRISPR\/Cas9\u6280\u672f\uff0c\u800c\u5c06\u91c7\u7528<a href=\"https:\/\/nai500.com\/zh-hans\/\u80a1\u7968\u67e5\u8be2\/?qm_symbol=BLUE\">Bluebird bio Inc\uff08NASDAQ: BLUE\uff09<\/a>\u5f00\u53d1\u7684\u53e6\u4e00\u79cd\u57fa\u4e8emRNA\u7684\u6280\u672fmegaTALs\u3002\u6709\u7814\u7a76\u663e\u793a\uff0c\u8fd9\u4e00\u66f4\u65b0\u7684\u57fa\u56e0\u7f16\u8f91\u6280\u672f\u76f8\u8f83CRISPR\/Cas9\u51c6\u786e\u6027\u66f4\u9ad8\u3002<\/p>\n<p>\u76ee\u524d\u54ea\u53ea\u57fa\u56e0\u7f16\u8f91\u4e2a\u80a1\u662f\u6700\u4f73\u7684\u6295\u8d44\u9009\u62e9\uff0c\u8fd9\u5f88\u96be\u8bf4\uff0c\u4f46\u4ec5\u4ece\u9570\u72b6\u7ec6\u80de\/\u03b2\u5730\u4e2d\u6d77\u8d2b\u8840\u7684\u8bd5\u9a8c\u7ed3\u679c\u51fa\u53d1\uff0cCRISPR Therapeutics\u7684\u4f18\u52bf\u8fd8\u662f\u6bd4\u8f83\u7a81\u51fa\u7684\u3002\u4e0d\u8fc7\uff0c\u6295\u8d44\u8005\u6700\u597d\u8fd8\u662f\u5e94\u5f53\u91c7\u53d6\u9e1f\u67aa\u7b56\u7565\uff0c\u5373\u591a\u5143\u5316\u6301\u80a1\uff0c\u5206\u6563\u5c0f\u989d\u6301\u6709\u5305\u62ecCRISPR\u3001Editas\u548cSangamo\u7b49\u5934\u90e8\u7684\u57fa\u56e0\u7f16\u8f91\u6982\u5ff5\u80a1\u3002<\/p>\n","protected":false},"excerpt":{"rendered":"<p>\u57fa\u56e0\u7f16\u8f91\u516c\u53f8CRISPR Therapeutics AG\uff08NASDAQ: CRSP\uff09\u6700\u8fd1\u53d1\u5e03\u57fa\u56e0\u7f16\u8f91\u9020\u8840\u5e72\u7ec6\u80de [&hellip;]<\/p>\n","protected":false},"author":27,"featured_media":306800,"comment_status":"closed","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"footnotes":"","_links_to":"","_links_to_target":""},"categories":[1521,430],"tags":[455,747,925,459],"class_list":["post-306798","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-1521","category-trends-strategies-zh-hans","tag-455","tag-747","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\/306798","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\/27"}],"replies":[{"embeddable":true,"href":"https:\/\/nai500.com\/zh-hans\/wp-json\/wp\/v2\/comments?post=306798"}],"version-history":[{"count":0,"href":"https:\/\/nai500.com\/zh-hans\/wp-json\/wp\/v2\/posts\/306798\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/nai500.com\/zh-hans\/wp-json\/wp\/v2\/media\/306800"}],"wp:attachment":[{"href":"https:\/\/nai500.com\/zh-hans\/wp-json\/wp\/v2\/media?parent=306798"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/nai500.com\/zh-hans\/wp-json\/wp\/v2\/categories?post=306798"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/nai500.com\/zh-hans\/wp-json\/wp\/v2\/tags?post=306798"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}