\u8981\u8aaa2022\u5e74\u6295\u8cc7\u8005\u6700\u4e0d\u7f3a\u4ec0\u9ebc\uff1f\u4fbf\u5b9c\u7684\u80a1\u7968\uff01\u4f46\u80a1\u5e02\u66b4\u8dcc\u662f\u6ce5\u6c99\u4ff1\u4e0b\uff0c\u6709\u7684\u662f\u6253\u56de\u539f\u5f62\uff0c\u53ea\u6709\u771f\u6b63\u7684\u597d\u516c\u53f8\u624d\u80fd\u6296\u843d\u7070\u5875\uff0c\u91cd\u65b0\u4e0a\u8def\u3002\u80a1\u7968\u7be9\u9078\u65b9\u6cd5\u6709\u5f88\u591a\uff0c\u5176\u4e2d\u4e00\u500b\u7d93\u904e\u8003\u9a57\u88ab\u8b49\u660e\u9760\u8b5c\u7684\u65b9\u6cd5\u662f\u95dc\u6ce8\u5167\u90e8\u4eba\u7684\u80a1\u7968\u8cb7\u8ce3\u60c5\u6cc1\u3002<\/p>\n
\u9019\u4e9b\u9ad8\u7ba1\u548c\u8463\u4e8b\u7b49\u5167\u90e8\u4eba\u6700\u77ad\u89e3\u516c\u53f8\u7684\u7d93\u71df\u60c5\u6cc1\u3002\u5982\u679c\u4ed6\u5011\u9858\u610f\u81ea\u638f\u8170\u5305\u8cb7\u5165\u516c\u53f8\u80a1\u7968\uff0c\u81f3\u5c11\u8aaa\u660e\u4ed6\u5011\u8a8d\u70ba\u80a1\u7968\u88ab\u4f4e\u4f30\u4e86\u3002\u4e0b\u9762\u9019\u5169\u96bb\u7f8e\u570b\u751f\u7269\u79d1\u6280\u80a1\u7968\u7684\u80a1\u50f9\u4eca\u5e74\u5927\u5e45\u4e0b\u8dcc\uff0c\u6700\u8fd1\u5167\u90e8\u4eba\u958b\u59cb\u9022\u4f4e\u8cb7\u5165\u9019\u4e9b\u80a1\u7968\uff0c\u9019\u53ef\u80fd\u9810\u793a\u8457\u80a1\u50f9\u5373\u5c07\u898b\u5e95\u3002<\/p>\n
2018\u5e74\uff0c\u5c08\u6ce8\u65bc\u7f55\u898b\u75c5\u85e5\u7269\u958b\u767c\u7684\u751f\u7269\u79d1\u6280\u516c\u53f8Insmed\u7684\u6297\u83cc\u85e5Arikayce\u2014\u2014\u9069\u61c9\u75c7\u70ba\u9ce5\u80de\u5167\u5206\u679d\u687f\u83cc\u8907\u5408\u9ad4\uff08MAC\uff09\u611f\u67d3\u5f15\u767c\u7684\u96e3\u6cbb\u6027\u975e\u7d50\u6838\u5206\u679d\u687f\u83cc\uff08NTM\uff09\u80ba\u75c5\u2014\u2014\u7372\u5f97\u6279\u51c6\uff0c\u56e0\u6b64\u7372\u5f97\u4e86\u7a69\u5b9a\u7684\u6536\u5165\u4f86\u6e90\u3002\u6700\u65b0\u767c\u4f48\u7684\u4e09\u5b63\u5831\u986f\u793a\uff0cArikayce\u7684\u92b7\u552e\u984d\u540c\u6bd4\u589e\u957745%\u81f3$6770\u842c\uff0c\u6709\u671b\u5be6\u73fe\u516c\u53f82022\u5e74\u92b7\u552e\u984d\u81f3\u5c11\u589e\u957730%\u7684\u76ee\u6a19\u3002<\/p>\n
\u9664\u4e86\u5df2\u7d93\u4e0a\u5e02\u7684\u85e5\uff0cInsmed\u7684\u5728\u7814\u85e5\u7269\u5305\u62ec\u652f\u6c23\u7ba1\u64f4\u5f35\u85e5\u7269brensocatib\uff0c\u4e09\u671fASPEN\u7814\u7a76\u7684\u62db\u52df\u5de5\u4f5c\u9810\u8a08\u5c07\u65bc1Q23\u5b8c\u6210\uff0c\u8a66\u9a57\u6578\u64da2Q24\u51fa\u7210\u3002\u6b64\u5916\uff0cbrensocatib\u6cbb\u7642\u56ca\u6027\u7e96\u7dad\u5316\uff08CF\uff09\u60a3\u8005\u7684\u4e8c\u671f\u85e5\u4ee3\u52d5\u529b\u5b78\/\u85e5\u6548\u5b78\u8a66\u9a57\u4e5f\u5728\u9032\u884c\u4e2d\uff0c\u6578\u64da\u9810\u8a08\u5c07\u57282022\u5e74\u5e95\u767c\u4f48\u3002TPIP\u5438\u5165\u5291\u7684\u5169\u9805\u4e8c\u671f\u7814\u7a76\u6b63\u5728\u62db\u52df\u88ab\u8a66\uff0c\u6578\u64da\u9810\u8a08\u5c07\u57282023\u5e74\u767c\u4f48\u3002\u6700\u5f8c\uff0cArikayce\u7684\u4e0a\u5e02\u5f8c\u78ba\u8b49\u6027\u81e8\u5e8a\u8a66\u9a57\uff08ARISE\u548cENCORE\u8a66\u9a57\uff09\u6b63\u5728\u9032\u884c\u4e2d\u3002<\/p>\n
\u5118\u7ba1\u5982\u6b64\uff0cINSM\u80a1\u50f9\u4eca\u5e74\u4e0b\u8dcc34%\u3002\u597d\u6d88\u606f\u662f\uff0c\u672c\u5468\u5169\u4f4d\u5167\u90e8\u4eba\u8a8d\u70ba\u8cb7\u5165\u6642\u6a5f\u5230\u4e86\uff0c\u5176\u4e2d\u8463\u4e8bMelvin Sharoky\u8cb7\u51653\u842c\u80a1\uff0c\u50f9\u503c$527700\u7684\u80a1\u7968\uff0c\u53e6\u5916\u8463\u4e8bLeo Lee\u8cb7\u51654.5\u842c\u80a1\uff0c\u50f9\u503c$791550\u7684\u80a1\u7968\u3002<\/p>\n
\u53e6\u4e00\u96bb\u7f8e\u570b\u751f\u7269\u79d1\u6280\u516c\u53f8Taysha Gene Therapies\u65e8\u5728\u958b\u767c\u591a\u7a2e\u6cbb\u7642\u55ae\u57fa\u56e0\u4e2d\u6a1e\u795e\u7d93\u7cfb\u7d71\u75be\u75c5\u7684\u57fa\u56e0\u7642\u6cd5\u4e26\u4e0a\u5e02\u3002<\/p>\n
Taysha\u7684\u817a\u76f8\u95dc\u75c5\u6bd2\uff08AAV\uff09\u57fa\u56e0\u7642\u6cd5\u5728\u7814\u85e5\u7269\u7576\u4e2d\uff0cTSHA-120\u6cbb\u7642\u7684\u662f\u4e00\u7a2e\u7f55\u898b\u7684\u907a\u50b3\u6027\u57fa\u56e0\u75be\u75c5\u2014\u2014\u5de8\u8ef8\u7d22\u795e\u7d93\u75c5\uff08GAN\uff09\uff0c\u4e26\u4e14\u5df2\u7d93\u7372\u5f97\u4e86FDA\u7684\u5b64\u5152\u85e5\u548c\u7f55\u898b\u5c0f\u5152\u75be\u75c5\u8cc7\u683c\u8a8d\u5b9a\u30021\/2\u671f\u7684\u81e8\u5e8a\u8a66\u9a57\u6578\u64da\u986f\u793a\uff0c\u63a5\u53d7\u6cbb\u7642\u7684\u60a3\u8005\u91cd\u65b0\u7372\u5f97\u4e86\u611f\u89ba\u795e\u7d93\u6ce2\u5e45\uff08SNAPs\uff09\uff0c\u540c\u6642\u986f\u793a\u81e8\u5e8a\u6539\u5584\u3002\u8a72\u516c\u53f8\u9810\u8a08\u5c07\u572812\u670813\u65e5\u8207FDA\u6703\u9762\u3002<\/p>\n
\u6b64\u5916\uff0cTSHA-102\u662f\u7b2c\u4e00\u4e5f\u662f\u552f\u4e00\u6b63\u5728\u958b\u767c\u6cbb\u7642\u96f7\u7279\u7d9c\u5408\u5f81\u2014\u2014\u4e00\u7a2e\u5f71\u97ff\u5927\u8166\u767c\u80b2\u7684\u7f55\u898b\u907a\u50b3\u75c5\u2014\u2014\u7684\u57fa\u56e0\u7642\u6cd5\u3002TSHA-102\u5df2\u7d93\u7372\u5f97\u5b64\u5152\u85e5\u548c\u7f55\u898b\u5c0f\u5152\u75be\u75c5\u7684\u8cc7\u683c\u8a8d\u5b9a\uff0c1\/2\u671f\u7814\u7a76\u7684\u6578\u64da\u9810\u8a08\u5c07\u65bc1H23\u51fa\u7210\u3002<\/p>\n
10\u6708\uff0cTaysha\u5ba3\u4f48\u7372\u5f97\u5b89\u65af\u6cf0\u4f86\u88fd\u85e5Astellas Pharma\uff08JP:4503\uff09$5000\u842c\u7684\u6230\u7565\u6295\u8cc7\uff0c\u7372\u5f97\u8a72\u516c\u53f8\u5927\u7d0415%\u7684\u80a1\u6b0a\uff0c\u4ee5\u53caTSHA-102\uff08\u96f7\u7279\u7d9c\u5408\u5f81\uff09\u548cTSHA-120\uff08\u5de8\u8ef8\u7d22\u795e\u7d93\u75c5\uff09\u7684\u7368\u4f54\u8a31\u53ef\u9078\u64c7\u6b0a\u3002\u6b64\u5916\u5728\u672c\u5468\uff0c\u8463\u4e8bPaul B Manning\u8cb7\u5165150\u842c\u80a1\uff0c\u7e3d\u984d\u9054$300\u842c\u7684Taysha\u80a1\u7968\u3002\u8a72\u80a1\u80a1\u50f9\u4eca\u5e74\u8fc4\u4eca\u4e0b\u8dcc83%\u3002<\/p>\n","protected":false},"excerpt":{"rendered":"
\u8981\u8aaa2022\u5e74\u6295\u8cc7\u8005\u6700\u4e0d\u7f3a\u4ec0\u9ebc\uff1f\u4fbf\u5b9c\u7684\u80a1\u7968\uff01\u4f46\u80a1\u5e02\u66b4\u8dcc\u662f\u6ce5\u6c99\u4ff1\u4e0b\uff0c\u6709\u7684\u662f\u6253\u56de\u539f\u5f62\uff0c\u53ea\u6709\u771f\u6b63\u7684\u597d\u516c\u53f8\u624d\u80fd\u6296\u843d\u7070\u5875 […]<\/p>\n","protected":false},"author":27,"featured_media":426794,"comment_status":"closed","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"footnotes":"","_links_to":"","_links_to_target":""},"categories":[1522,431],"tags":[1818,927,563,2001,564],"class_list":{"0":"post-442414","1":"post","2":"type-post","3":"status-publish","4":"format-standard","5":"has-post-thumbnail","6":"hentry","7":"category-1522","8":"category-trends-strategies-zh-hant","9":"tag-in-depth-analysis-zh-hant","10":"tag-927","11":"tag--zh-hant","13":"tag-564","14":"sectors-life-science-zh-hant","15":"locations-us-zh-hant","16":"exchange-nasdaq-zh-hant"},"acf":[],"_links":{"self":[{"href":"https:\/\/nai500.com\/zh-hant\/wp-json\/wp\/v2\/posts\/442414","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/nai500.com\/zh-hant\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/nai500.com\/zh-hant\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/nai500.com\/zh-hant\/wp-json\/wp\/v2\/users\/27"}],"replies":[{"embeddable":true,"href":"https:\/\/nai500.com\/zh-hant\/wp-json\/wp\/v2\/comments?post=442414"}],"version-history":[{"count":0,"href":"https:\/\/nai500.com\/zh-hant\/wp-json\/wp\/v2\/posts\/442414\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/nai500.com\/zh-hant\/wp-json\/wp\/v2\/media\/426794"}],"wp:attachment":[{"href":"https:\/\/nai500.com\/zh-hant\/wp-json\/wp\/v2\/media?parent=442414"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/nai500.com\/zh-hant\/wp-json\/wp\/v2\/categories?post=442414"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/nai500.com\/zh-hant\/wp-json\/wp\/v2\/tags?post=442414"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}