{"id":164386,"date":"2023-07-18T17:13:00","date_gmt":"2023-07-18T09:13:00","guid":{"rendered":"https:\/\/pingtungtimes.com.tw\/?p=164386"},"modified":"2023-07-18T17:13:00","modified_gmt":"2023-07-18T09:13:00","slug":"%e7%b6%ad%e4%ba%9e%e7%94%9f%e7%89%a9%e6%8a%95%e8%b3%87%e5%ad%b5%e5%8c%96%e5%85%ac%e5%8f%b8dtx-pharma%e5%ae%a3%e5%b8%83%e8%88%87%e8%ab%be%e8%8f%af%e9%81%94%e6%88%90%e6%94%b6%e8%b3%bc%e5%8d%94%e8%ad%b0","status":"publish","type":"post","link":"https:\/\/pingtungtimes.com.tw\/?p=164386","title":{"rendered":"\u7dad\u4e9e\u751f\u7269\u6295\u8cc7\u5b75\u5316\u516c\u53f8DTx Pharma\u5ba3\u5e03\u8207\u8afe\u83ef\u9054\u6210\u6536\u8cfc\u5354\u8b70"},"content":{"rendered":"
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\u8056\u5730\u7259\u54e5<\/span>2023\u5e747\u670818\u65e5<\/span> \/\u7f8e\u901a\u793e\/ — \u7dad\u4e9e\u751f\u7269\u53c3\u8207\u6295\u8cc7\u5b75\u5316\u7684 DTx Pharma \u662f\u4e00\u5bb6\u81e8\u5e8a\u524d\u968e\u6bb5\u751f\u7269\u6280\u8853\u516c\u53f8\uff0c\u5229\u7528\u5176\u8102\u80aa\u9178\u914d\u9ad4\u5171\u8edb\u5be1\u6838\u82f7\u9178 (FALCON\u2122) \u5e73\u53f0\u89e3\u6c7a\u5be1\u6838\u82f7\u9178\u7642\u6cd5\u7684\u905e\u9001\u6311\u6230\u30022023\u5e747\u670817\u65e5\uff0c<\/span>\u8a72\u516c\u53f8\u5ba3\u5e03\u8207\u8afe\u83ef\u9054\u6210\u6536\u8cfc\u5354\u8b70\u3002\u6839\u64da\u96d9\u65b9\u5354\u8b70\u689d\u6b3e\uff0c\u8afe\u83ef\u5c07\u652f\u4ed85\u5104\u7f8e\u5143\u7684\u9810\u4ed8\u6b3e\uff0c\u4e26\u5728\u5b8c\u6210\u9810\u5148\u6307\u5b9a\u7684\u91cc\u7a0b\u7891\u5f8c\u984d\u5916\u652f\u4ed8\u6700\u591a5\u5104\u7f8e\u5143\u3002FALCON \u5e73\u53f0\u80fd\u5920\u5c07\u5c0f\u5e72\u64fe RNA (siRNA) \u6cbb\u7642\u85e5\u7269\u8f38\u9001\u5230\u809d\u81df\u4ee5\u5916\u7684\u7d44\u7e54\u4e26\u767c\u63ee\u5176\u6d3b\u6027\uff0c\u5f9e\u800c\u589e\u5f37siRNA\u85e5\u7269\u7684\u751f\u7269\u5206\u4f48\u548c\u7d30\u80de\u651d\u53d6\u3002DTx Pharma \u7684\u9818\u5148\u7ba1\u7dda\u76ee\u524d\u6b63\u5728\u9032\u884c\u81e8\u5e8a\u524d\u958b\u767c\uff0c\u4e26\u7372\u5f97 FDA \u5b64\u5152\u85e5\u8cc7\u683c\u8a8d\u5b9a\uff0c\u7528\u65bc\u6cbb\u76421A \u578b\u8153\u9aa8\u808c\u840e\u7e2e\u75c7 (CMT1A)\u3002\u9664\u4e86 CMT1A \u9805\u76ee\u5916\uff0c\u8afe\u83ef\u9084\u7372\u5f97\u4e86 FALCON \u5e73\u53f0\u548c\u53e6\u5916\u5169\u500b\u65e9\u671f\u9805\u76ee\uff08\u91dd\u5c0d\u795e\u7d93\u808c\u8089\u548c\u4e2d\u6a1e\u795e\u7d93\u7cfb\u7d71\u9069\u61c9\u75c7\uff09\u7684\u5168\u90e8\u6b0a\u529b\u3002<\/p>\n

CMT1A \u662f\u4e00\u7a2e\u9032\u884c\u6027\u795e\u7d93\u808c\u8089\u5e38\u67d3\u8272\u9ad4\u986f\u6027\u907a\u50b3\u75be\u75c5\uff0c\u53ef\u5c0e\u81f4\u7d42\u751f\u808c\u8089\u529f\u80fd\u55aa\u5931\u548c\u6b98\u75be\u3002\u76ee\u524d\uff0c\u7f8e\u570b\u548c\u6b50\u6d32\u7d04\u6709150,000\u540d CMT1A \u60a3\u8005\uff0c\u76ee\u524d\u5c1a\u7121\u91dd\u5c0dPMP22\uff08CMT1A\u7684\u6f5b\u5728\u907a\u50b3\u539f\u56e0\uff09\u7684\u6279\u51c6\u7642\u6cd5\u3002DTx Pharma \u7684\u9818\u5148\u7ba1\u7dda DTx-1252\u662f\u4e00\u7a2e\u91dd\u5c0d PMP22\u7684\u65b0\u578b\u7684\u3001\u9996\u5275\u7684 siRNA \u5019\u9078\u85e5\u7269\u3002\u8a72\u8cc7\u7522\u64c1\u6709\u7a69\u5065\u7684\u81e8\u5e8a\u524d\u6578\u64da\u7814\u7a76\uff0c\u4e0d\u50c5\u5c55\u793a\u4e86\u5728\u81e8\u5e8a\u524d\u9f67\u9f52\u52d5\u7269\u6a21\u578b\u4e2d\u75be\u75c5\u7684\u9006\u8f49\uff0c\u9084\u70ba\u5411\u81e8\u5e8a\u7814\u7a76\u7684\u8f49\u5316\u63d0\u4f9b\u4e86\u53ef\u9760\u7684\u6578\u64da\u3002<\/p>\n

\u300c\u6211\u5f88\u9ad8\u8208\u8afe\u83ef\u5c07\u63a8\u9032\u6211\u5011\u7684 CMT1A \u6cbb\u7642\u7ba1\u7dda\u548c FALCON \u5e73\u53f0\u3002\u6191\u85c9\u5728\u795e\u7d93\u808c\u8089\u75be\u75c5\u9818\u57df\u7684\u8cc7\u6e90\u548c\u80fd\u529b\uff0c\u8afe\u83ef\u5b8c\u5168\u6709\u80fd\u529b\u52a0\u901f DTx-1252\u7684\u958b\u767c\uff0c\u70ba\u6025\u9700\u6cbb\u7642\u7684\u60a3\u8005\u5e36\u4f86\u5e0c\u671b\u3002\u300dDTx Pharm \u7684\u806f\u5408\u5275\u59cb\u4eba\u517c\u9996\u5e2d\u57f7\u884c\u5b98 Artie Suckow<\/span> \u535a\u58eb\u8868\u793a\uff0c\u300c\u6211\u4e5f\u611f\u5230\u975e\u5e38\u81ea\u8c6a\uff0c\u5728\u6211\u5011\u5718\u968a\u7684\u627f\u8afe\u548c\u52aa\u529b\u4e0b\uff0cDTx Pharma \u6210\u70ba\u809d\u5916 siRNA \u905e\u9001\u9818\u57df\u7684\u9818\u5c0e\u8005\uff0c\u6b63\u5982\u6211\u5011\u63a8\u9032\u7b2c\u4e00\u500b\u7814\u7a76\u6027 FALCON siRNA \u5de5\u4f5c\u6240\u8b49\u660e\u7684\u90a3\u6a23\u2014\u2014FALCON\u65e8\u5728\u905e\u9001 siRNA \u81f3\u5916\u5468\u795e\u7d93\u7cfb\u7d71\u4f86\u6cbb\u7642 CMT1A\u3002\u300d<\/p>\n

\u300c\u6b64\u6b21\u6536\u8cfc\u51f8\u986f\u4e86\u8afe\u83ef\u81f4\u529b\u65bc\u70ba\u60a3\u6709\u795e\u7d93\u808c\u8089\u75be\u75c5\u548c\u5176\u4ed6\u795e\u7d93\u7cfb\u7d71\u75be\u75c5\u7684\u60a3\u8005\uff0c\u63d0\u4f9b\u6539\u8b8a\u751f\u6d3b\u7684\u85e5\u7269\u7684\u627f\u8afe\u3002\u300d\u8afe\u83ef\u751f\u7269\u91ab\u5b78\u7814\u7a76\u6240\u795e\u7d93\u79d1\u5b78\u5168\u7403\u8ca0\u8cac\u4eba Robert Baloh<\/span> \u8868\u793a\uff0c\u300c\u6211\u5011\u5f88\u9ad8\u8208\u80fd\u5920\u63a8\u52d5\u9019\u4e9b\u6709\u524d\u666f\u7684\u81e8\u5e8a\u524d\u9805\u76ee\u4e26\u63a2\u7d22 FALCON \u5e73\u53f0\u7684\u6f5b\u529b\u3002\u300d<\/p>\n

\u300c\u8afe\u83ef\u5718\u968a\u5c0d CMT1A \u751f\u7269\u5b78\u7684\u6df1\u5165\u4e86\u89e3\uff0c\u4ee5\u53ca\u5c0d\u60a3\u8005\u8ca0\u64d4\u7684\u6df1\u523b\u7406\u89e3\uff0c\u5f9e\u6211\u5011\u7684\u7b2c\u4e00\u6b21\u4e92\u52d5\u4e2d\u5c31\u53ef\u898b\u4e00\u6591\uff0c\u6211\u5011\u5c0d\u4ed6\u5011\u652f\u6301\u63a8\u9032DTx-1252\u7684\u958b\u767c\u611f\u5230\u8208\u596e\u3002\u300dDTx Pharma \u7684 CBO Peter Condon \u8868\u793a:\u300c\u9019\u9805\u5354\u8b70\u4e5f\u662f\u5c0d\u6211\u5011 FALCON \u5e73\u53f0\u53ca\u901a\u904e\u5176\u5728\u809d\u81df\u4e4b\u5916\u905e\u9001 siRNA \u4f86\u958b\u95e2\u65b0\u6cbb\u7642\u9818\u57df\u7684\u6f5b\u529b\u7684\u6709\u529b\u9a57\u8b49\u3002\u300d<\/p>\n

\u7dad\u4e9e\u751f\u7269\u9996\u5e2d\u5275\u65b0\u5b98\u517c\u7dad\u4e9e\u751f\u7269\u5275\u65b0\u4e2d\u5fc3\u8ca0\u8cac\u4eba\u6234\u6657\u535a\u58eb\u8868\u793a\uff1a\u300cDTx \u4f9d\u6258\u5275\u59cb\u5718\u968a\u5728 siRNA \u85e5\u7269\u591a\u5e74\u7684\u6df1\u8015\u548c\u7a4d\u7d2f\uff0c\u6210\u529f\u958b\u767c\u5275\u65b0\u6027 RNA \u85e5\u7269\u905e\u9001\u5e73\u53f0 FALCON\uff0c\u7528\u65bc\u5c07 RNA \u85e5\u7269\u905e\u9001\u5230\u809d\u81df\u4ee5\u5916\u7684\u5668\u5b98\uff0c\u89e3\u6c7a\u4e86 RNA \u85e5\u7269\u905e\u9001\u7684\u75db\u9ede\u3002\u6211\u5011\u5f88\u9ad8\u8208\u898b\u8b49\u4e26\u966a\u4f34\u4e86 DTx Pharma \u5f9e\u6982\u5ff5\u9a57\u8b49\u5230\u81e8\u5e8a\u524d\u7814\u7a76\u9019\u4e00\u8def\u7684\u6210\u9577\uff0c\u4e26\u53c3\u8207\u5176\u591a\u8f2a\u878d\u8cc7\u3002\u672c\u6b21\u9054\u6210\u6536\u8cfc\uff0c\u4e5f\u518d\u6b21\u9a57\u8b49\u4e86\u7dad\u4e9e\u6295\u8cc7\u5718\u968a\u7684\u6230\u7565\u773c\u5149\u53ca\u5c08\u696d\u7684\u6295\u5f8c\u652f\u6301\u3002\u501f\u52a9\u8afe\u83ef\u5728\u7f55\u898b\u75c5\u9818\u57df\u7684\u7a4d\u7d2f\uff0c\u6211\u5011\u76f8\u4fe1 DTx \u7ba1\u7dda\u7684\u7814\u767c\u6709\u671b\u9032\u4e00\u6b65\u52a0\u901f\uff0c\u66f4\u597d\u5730\u958b\u62d3\u7f55\u898b\u75c5\u9818\u57df\u3002\u300d<\/p>\n

\u95dc\u65bc<\/b>DTx Pharma
<\/b>DTx Pharma, Inc.\u662f\u4e00\u5bb6\u79c1\u71df\u751f\u7269\u6280\u8853\u516c\u53f8\uff0c\u5229\u7528\u8a72\u516c\u53f8\u5c08\u6709\u7684\u8102\u80aa\u9178\u914d\u9ad4\u5171\u8edb\u5be1\u6838\u82f7\u9178 (FALCON\u2122) \u6280\u8853\u5e73\u53f0\u89e3\u6c7a RNAi \u7642\u6cd5\u6cbb\u7642\u809d\u81df\u4ee5\u5916\u75be\u75c5\u7684\u76f8\u95dc\u6311\u6230\u3002FALCON \u5e73\u53f0\u5229\u7528\u8102\u80aa\u9178\u4f86\u589e\u5f37\u5168\u8eab\u7d44\u7e54\u548c\u7d30\u80de\u985e\u578b\u7684\u751f\u7269\u5206\u4f48\u548c\u7d30\u80de\u651d\u53d6\u3002\u5728\u81e8\u5e8a\u524d\u7814\u7a76\u4e2d\uff0cFALCON siRNA \u5df2\u8b49\u660e\u80fd\u5920\u6709\u6548\u4e14\u6301\u4e45\u5730\u6291\u5236\u5468\u570d\u795e\u7d93\u7cfb\u7d71 (PNS)\u3001\u9aa8\u9abc\u808c\u3001\u5fc3\u81df\u3001\u76ae\u819a\u548c\u4e2d\u6a1e\u795e\u7d93\u7cfb\u7d71\u4e2d\u7684\u9776\u57fa\u56e0\u3002FALCON siRNA \u53ef\u4ee5\u901a\u904e\u975c\u8108\u5167\u3001\u76ae\u4e0b\u548c\u9798\u5167\u7d66\u85e5\u9014\u5f91\u905e\u9001\uff0c\u4e26\u4e14\u53ef\u4ee5\u4ee5\u76f8\u5c0d\u8f03\u4f4e\u7684\u6210\u672c\u88fd\u9020\u3002\u8a72\u516c\u53f8\u64c1\u6709\u5c08\u6ce8\u65bc\u5916\u5468\u795e\u7d93\u7cfb\u7d71\u3001\u4e2d\u6a1e\u795e\u7d93\u7cfb\u7d71\u548c\u795e\u7d93\u808c\u8089\u75be\u75c5\u7684\u7522\u54c1\u7dda\u3002DTx Pharma\u5df2\u5f9e\u591a\u5bb6\u5168\u7403\u9818\u5148\u7684\u751f\u7269\u91ab\u85e5\u6295\u8cc7\u8005\u90a3\u88e1\u7c4c\u96c6\u4e86\u8d85\u904e1.15\u5104\u7f8e\u5143\u7684\u6295\u8cc7\uff0c\u5305\u62ecRA Capital Management\u3001Access Biotechnology\u3001Surveyor Capital\uff08Citadel \u516c\u53f8\uff09\u3001Eli Lilly and Company\u3001Friedman Bioventure\u3001Viva Biotech Holdings\u4ee5\u53ca\u4f86\u81eaCMT\u7814\u7a76\u57fa\u91d1\u6703 (CMTRF) \u548c\u7f8e\u570b\u570b\u7acb\u885b\u751f\u7814\u7a76\u9662\u7b49\u7814\u7a76\u57fa\u91d1\u6703\u7684\u652f\u6301\u3002\u6b32\u4e86\u89e3\u6709\u95dc DTx Pharma \u7684\u66f4\u591a\u4fe1\u606f\uff0c\u8acb\u8a2a\u554f www.dtxpharma.com<\/a> \u6216\u8005\u5728 LinkedIn \u548c Twitter \u4e0a\u95dc\u6ce8 DTx@DTxPharma\u3002<\/p>\n

\u95dc\u65bc\u7dad\u4e9e\u751f\u7269
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HK\uff09\u6210\u7acb\u65bc2008\u5e74\uff0c\u5411\u5168\u7403\u5275\u65b0\u85e5\u7814\u767c\u4f01\u696d\u63d0\u4f9b\u5f9e\u65e9\u671f\u57fa\u65bc\u7d50\u69cb\u7684\u85e5\u7269\u7814\u767c\u5230\u5546\u696d\u5316\u85e5\u7269\u4ea4\u4ed8\u7684\u4e00\u7ad9\u5f0f\u7d9c\u5408\u670d\u52d9\u3002\u6191\u85c9\u5728\u57fa\u65bc\u7d50\u69cb\u7684\u85e5\u7269\u7814\u767c (SBDD) \u6280\u8853\u9818\u57df\u7684\u9818\u5148\u512a\u52e2\uff0c\u6211\u5011\u5411\u5168\u7403\u5408\u4f5c\u5925\u4f34\u63d0\u4f9b\u65b0\u85e5\u7814\u7a76\u968e\u6bb5\u7684 CRO \u670d\u52d9\uff0c\u642d\u5efa\u4e86X \u5c04\u7dda\u86cb\u767d\u6676\u9ad4\u6280\u8853\u3001\u51b7\u51cd\u96fb\u93e1\u6280\u8853 (Cryo-EM)\u3001\u89aa\u548c\u529b\u8cea\u8b5c\u7be9\u9078\u6280\u8853 (ASMS)\u3001\u8868\u9762\u7b49\u96e2\u5b50\u5171\u632f\u6280\u8853 (SPR)\u3001\u6c2b\u6c18\u4ea4\u63db\u8cea\u8b5c\u6280\u8853 (HDX-MS)\u3001\u8a08\u7b97\u6a5f\u8f14\u52a9\u85e5\u7269\u8a2d\u8a08\u7b49\u591a\u500b\u5148\u9032\u6280\u8853\u5e73\u53f0\uff0c\u4e26\u6709\u8cc7\u6df1\u85e5\u7269\u5316\u5b78\u5bb6\u8207\u85e5\u7269\u767c\u73fe\u751f\u7269\u5c08\u5bb6\u9818\u8ecd\u7684\u5718\u968a\u63d0\u4f9b\u85e5\u7269\u8a2d\u8a08\u3001\u85e5\u7269\u5316\u5b78\uff08H2L\uff0cLO\uff09\u3001\u5316\u5408\u7269\u5408\u6210\u3001\u5316\u5b78\u5206\u6790\u53ca\u7d14\u5316\u3001\u516c\u65a4\u7d1a\u653e\u5927\u53ca\u591a\u80bd\u5408\u6210\u53ca\u76f8\u61c9\u7684\u751f\u7269\u6d3b\u6027\u6e2c\u8a66\u670d\u52d9\u3002\u901a\u904e\u5b50\u516c\u53f8\u6717\u83ef\u88fd\u85e5\uff0c\u6211\u5011\u63d0\u4f9b\u5f9e\u81e8\u5e8a\u524d\u958b\u767c\u5230\u5546\u696d\u5316\u751f\u7522\u7684\u4e00\u7ad9\u5f0f CMC\/CDMO \u89e3\u6c7a\u65b9\u6848\u3002\u540c\u6642\uff0c\u6211\u5011\u5c08\u6ce8\u65bc\u767c\u73fe\u3001\u6295\u8cc7\u9ad8\u6f5b\u529b\u751f\u7269\u91ab\u85e5\u521d\u5275\u516c\u53f8\uff0c\u4ee5\u7368\u5275\u7684\u6280\u8853\u670d\u52d9\u63db\u53d6\u80a1\u6b0a (EFS) \u7684\u5546\u696d\u6a21\u5f0f\uff0c\u89e3\u6c7a\u672a\u6eff\u8db3\u7684\u81e8\u5e8a\u9700\u6c42\u3002\" \/>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/pingtungtimes.com.tw\/?p=164386\" \/>\n<meta property=\"og:locale\" content=\"zh_TW\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"\u7dad\u4e9e\u751f\u7269\u6295\u8cc7\u5b75\u5316\u516c\u53f8DTx Pharma\u5ba3\u5e03\u8207\u8afe\u83ef\u9054\u6210\u6536\u8cfc\u5354\u8b70 - \u5c4f\u6771\u6642\u5831\u96fb\u5b50\u5831\" \/>\n<meta property=\"og:description\" content=\"\u8056\u5730\u7259\u54e52023\u5e747\u670818\u65e5 \/\u7f8e\u901a\u793e\/ -- \u7dad\u4e9e\u751f\u7269\u53c3\u8207\u6295\u8cc7\u5b75\u5316\u7684 DTx Pharma \u662f\u4e00\u5bb6\u81e8\u5e8a\u524d\u968e\u6bb5\u751f\u7269\u6280\u8853\u516c\u53f8\uff0c\u5229\u7528\u5176\u8102\u80aa\u9178\u914d\u9ad4\u5171\u8edb\u5be1\u6838\u82f7\u9178 (FALCON\u2122) \u5e73\u53f0\u89e3\u6c7a\u5be1\u6838\u82f7\u9178\u7642\u6cd5\u7684\u905e\u9001\u6311\u6230\u30022023\u5e747\u670817\u65e5\uff0c\u8a72\u516c\u53f8\u5ba3\u5e03\u8207\u8afe\u83ef\u9054\u6210\u6536\u8cfc\u5354\u8b70\u3002\u6839\u64da\u96d9\u65b9\u5354\u8b70\u689d\u6b3e\uff0c\u8afe\u83ef\u5c07\u652f\u4ed85\u5104\u7f8e\u5143\u7684\u9810\u4ed8\u6b3e\uff0c\u4e26\u5728\u5b8c\u6210\u9810\u5148\u6307\u5b9a\u7684\u91cc\u7a0b\u7891\u5f8c\u984d\u5916\u652f\u4ed8\u6700\u591a5\u5104\u7f8e\u5143\u3002FALCON \u5e73\u53f0\u80fd\u5920\u5c07\u5c0f\u5e72\u64fe RNA (siRNA) \u6cbb\u7642\u85e5\u7269\u8f38\u9001\u5230\u809d\u81df\u4ee5\u5916\u7684\u7d44\u7e54\u4e26\u767c\u63ee\u5176\u6d3b\u6027\uff0c\u5f9e\u800c\u589e\u5f37siRNA\u85e5\u7269\u7684\u751f\u7269\u5206\u4f48\u548c\u7d30\u80de\u651d\u53d6\u3002DTx Pharma \u7684\u9818\u5148\u7ba1\u7dda\u76ee\u524d\u6b63\u5728\u9032\u884c\u81e8\u5e8a\u524d\u958b\u767c\uff0c\u4e26\u7372\u5f97 FDA \u5b64\u5152\u85e5\u8cc7\u683c\u8a8d\u5b9a\uff0c\u7528\u65bc\u6cbb\u76421A \u578b\u8153\u9aa8\u808c\u840e\u7e2e\u75c7 (CMT1A)\u3002\u9664\u4e86 CMT1A \u9805\u76ee\u5916\uff0c\u8afe\u83ef\u9084\u7372\u5f97\u4e86 FALCON \u5e73\u53f0\u548c\u53e6\u5916\u5169\u500b\u65e9\u671f\u9805\u76ee\uff08\u91dd\u5c0d\u795e\u7d93\u808c\u8089\u548c\u4e2d\u6a1e\u795e\u7d93\u7cfb\u7d71\u9069\u61c9\u75c7\uff09\u7684\u5168\u90e8\u6b0a\u529b\u3002 CMT1A \u662f\u4e00\u7a2e\u9032\u884c\u6027\u795e\u7d93\u808c\u8089\u5e38\u67d3\u8272\u9ad4\u986f\u6027\u907a\u50b3\u75be\u75c5\uff0c\u53ef\u5c0e\u81f4\u7d42\u751f\u808c\u8089\u529f\u80fd\u55aa\u5931\u548c\u6b98\u75be\u3002\u76ee\u524d\uff0c\u7f8e\u570b\u548c\u6b50\u6d32\u7d04\u6709150,000\u540d CMT1A \u60a3\u8005\uff0c\u76ee\u524d\u5c1a\u7121\u91dd\u5c0dPMP22\uff08CMT1A\u7684\u6f5b\u5728\u907a\u50b3\u539f\u56e0\uff09\u7684\u6279\u51c6\u7642\u6cd5\u3002DTx Pharma \u7684\u9818\u5148\u7ba1\u7dda DTx-1252\u662f\u4e00\u7a2e\u91dd\u5c0d PMP22\u7684\u65b0\u578b\u7684\u3001\u9996\u5275\u7684 siRNA \u5019\u9078\u85e5\u7269\u3002\u8a72\u8cc7\u7522\u64c1\u6709\u7a69\u5065\u7684\u81e8\u5e8a\u524d\u6578\u64da\u7814\u7a76\uff0c\u4e0d\u50c5\u5c55\u793a\u4e86\u5728\u81e8\u5e8a\u524d\u9f67\u9f52\u52d5\u7269\u6a21\u578b\u4e2d\u75be\u75c5\u7684\u9006\u8f49\uff0c\u9084\u70ba\u5411\u81e8\u5e8a\u7814\u7a76\u7684\u8f49\u5316\u63d0\u4f9b\u4e86\u53ef\u9760\u7684\u6578\u64da\u3002 \u300c\u6211\u5f88\u9ad8\u8208\u8afe\u83ef\u5c07\u63a8\u9032\u6211\u5011\u7684 CMT1A \u6cbb\u7642\u7ba1\u7dda\u548c FALCON \u5e73\u53f0\u3002\u6191\u85c9\u5728\u795e\u7d93\u808c\u8089\u75be\u75c5\u9818\u57df\u7684\u8cc7\u6e90\u548c\u80fd\u529b\uff0c\u8afe\u83ef\u5b8c\u5168\u6709\u80fd\u529b\u52a0\u901f DTx-1252\u7684\u958b\u767c\uff0c\u70ba\u6025\u9700\u6cbb\u7642\u7684\u60a3\u8005\u5e36\u4f86\u5e0c\u671b\u3002\u300dDTx Pharm \u7684\u806f\u5408\u5275\u59cb\u4eba\u517c\u9996\u5e2d\u57f7\u884c\u5b98 Artie Suckow \u535a\u58eb\u8868\u793a\uff0c\u300c\u6211\u4e5f\u611f\u5230\u975e\u5e38\u81ea\u8c6a\uff0c\u5728\u6211\u5011\u5718\u968a\u7684\u627f\u8afe\u548c\u52aa\u529b\u4e0b\uff0cDTx Pharma \u6210\u70ba\u809d\u5916 siRNA \u905e\u9001\u9818\u57df\u7684\u9818\u5c0e\u8005\uff0c\u6b63\u5982\u6211\u5011\u63a8\u9032\u7b2c\u4e00\u500b\u7814\u7a76\u6027 FALCON siRNA \u5de5\u4f5c\u6240\u8b49\u660e\u7684\u90a3\u6a23\u2014\u2014FALCON\u65e8\u5728\u905e\u9001 siRNA \u81f3\u5916\u5468\u795e\u7d93\u7cfb\u7d71\u4f86\u6cbb\u7642 CMT1A\u3002\u300d \u300c\u6b64\u6b21\u6536\u8cfc\u51f8\u986f\u4e86\u8afe\u83ef\u81f4\u529b\u65bc\u70ba\u60a3\u6709\u795e\u7d93\u808c\u8089\u75be\u75c5\u548c\u5176\u4ed6\u795e\u7d93\u7cfb\u7d71\u75be\u75c5\u7684\u60a3\u8005\uff0c\u63d0\u4f9b\u6539\u8b8a\u751f\u6d3b\u7684\u85e5\u7269\u7684\u627f\u8afe\u3002\u300d\u8afe\u83ef\u751f\u7269\u91ab\u5b78\u7814\u7a76\u6240\u795e\u7d93\u79d1\u5b78\u5168\u7403\u8ca0\u8cac\u4eba Robert Baloh \u8868\u793a\uff0c\u300c\u6211\u5011\u5f88\u9ad8\u8208\u80fd\u5920\u63a8\u52d5\u9019\u4e9b\u6709\u524d\u666f\u7684\u81e8\u5e8a\u524d\u9805\u76ee\u4e26\u63a2\u7d22 FALCON \u5e73\u53f0\u7684\u6f5b\u529b\u3002\u300d \u300c\u8afe\u83ef\u5718\u968a\u5c0d CMT1A \u751f\u7269\u5b78\u7684\u6df1\u5165\u4e86\u89e3\uff0c\u4ee5\u53ca\u5c0d\u60a3\u8005\u8ca0\u64d4\u7684\u6df1\u523b\u7406\u89e3\uff0c\u5f9e\u6211\u5011\u7684\u7b2c\u4e00\u6b21\u4e92\u52d5\u4e2d\u5c31\u53ef\u898b\u4e00\u6591\uff0c\u6211\u5011\u5c0d\u4ed6\u5011\u652f\u6301\u63a8\u9032DTx-1252\u7684\u958b\u767c\u611f\u5230\u8208\u596e\u3002\u300dDTx Pharma \u7684 CBO Peter Condon \u8868\u793a:\u300c\u9019\u9805\u5354\u8b70\u4e5f\u662f\u5c0d\u6211\u5011 FALCON \u5e73\u53f0\u53ca\u901a\u904e\u5176\u5728\u809d\u81df\u4e4b\u5916\u905e\u9001 siRNA \u4f86\u958b\u95e2\u65b0\u6cbb\u7642\u9818\u57df\u7684\u6f5b\u529b\u7684\u6709\u529b\u9a57\u8b49\u3002\u300d \u7dad\u4e9e\u751f\u7269\u9996\u5e2d\u5275\u65b0\u5b98\u517c\u7dad\u4e9e\u751f\u7269\u5275\u65b0\u4e2d\u5fc3\u8ca0\u8cac\u4eba\u6234\u6657\u535a\u58eb\u8868\u793a\uff1a\u300cDTx \u4f9d\u6258\u5275\u59cb\u5718\u968a\u5728 siRNA \u85e5\u7269\u591a\u5e74\u7684\u6df1\u8015\u548c\u7a4d\u7d2f\uff0c\u6210\u529f\u958b\u767c\u5275\u65b0\u6027 RNA \u85e5\u7269\u905e\u9001\u5e73\u53f0 FALCON\uff0c\u7528\u65bc\u5c07 RNA \u85e5\u7269\u905e\u9001\u5230\u809d\u81df\u4ee5\u5916\u7684\u5668\u5b98\uff0c\u89e3\u6c7a\u4e86 RNA \u85e5\u7269\u905e\u9001\u7684\u75db\u9ede\u3002\u6211\u5011\u5f88\u9ad8\u8208\u898b\u8b49\u4e26\u966a\u4f34\u4e86 DTx Pharma \u5f9e\u6982\u5ff5\u9a57\u8b49\u5230\u81e8\u5e8a\u524d\u7814\u7a76\u9019\u4e00\u8def\u7684\u6210\u9577\uff0c\u4e26\u53c3\u8207\u5176\u591a\u8f2a\u878d\u8cc7\u3002\u672c\u6b21\u9054\u6210\u6536\u8cfc\uff0c\u4e5f\u518d\u6b21\u9a57\u8b49\u4e86\u7dad\u4e9e\u6295\u8cc7\u5718\u968a\u7684\u6230\u7565\u773c\u5149\u53ca\u5c08\u696d\u7684\u6295\u5f8c\u652f\u6301\u3002\u501f\u52a9\u8afe\u83ef\u5728\u7f55\u898b\u75c5\u9818\u57df\u7684\u7a4d\u7d2f\uff0c\u6211\u5011\u76f8\u4fe1 DTx \u7ba1\u7dda\u7684\u7814\u767c\u6709\u671b\u9032\u4e00\u6b65\u52a0\u901f\uff0c\u66f4\u597d\u5730\u958b\u62d3\u7f55\u898b\u75c5\u9818\u57df\u3002\u300d \u95dc\u65bcDTx PharmaDTx Pharma, Inc.\u662f\u4e00\u5bb6\u79c1\u71df\u751f\u7269\u6280\u8853\u516c\u53f8\uff0c\u5229\u7528\u8a72\u516c\u53f8\u5c08\u6709\u7684\u8102\u80aa\u9178\u914d\u9ad4\u5171\u8edb\u5be1\u6838\u82f7\u9178 (FALCON\u2122) \u6280\u8853\u5e73\u53f0\u89e3\u6c7a RNAi \u7642\u6cd5\u6cbb\u7642\u809d\u81df\u4ee5\u5916\u75be\u75c5\u7684\u76f8\u95dc\u6311\u6230\u3002FALCON \u5e73\u53f0\u5229\u7528\u8102\u80aa\u9178\u4f86\u589e\u5f37\u5168\u8eab\u7d44\u7e54\u548c\u7d30\u80de\u985e\u578b\u7684\u751f\u7269\u5206\u4f48\u548c\u7d30\u80de\u651d\u53d6\u3002\u5728\u81e8\u5e8a\u524d\u7814\u7a76\u4e2d\uff0cFALCON siRNA \u5df2\u8b49\u660e\u80fd\u5920\u6709\u6548\u4e14\u6301\u4e45\u5730\u6291\u5236\u5468\u570d\u795e\u7d93\u7cfb\u7d71 (PNS)\u3001\u9aa8\u9abc\u808c\u3001\u5fc3\u81df\u3001\u76ae\u819a\u548c\u4e2d\u6a1e\u795e\u7d93\u7cfb\u7d71\u4e2d\u7684\u9776\u57fa\u56e0\u3002FALCON siRNA 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