{"id":8843,"date":"2026-10-04T10:08:04","date_gmt":"2026-10-04T07:08:04","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=8843"},"modified":"2026-10-04T10:08:07","modified_gmt":"2026-10-04T07:08:07","slug":"rag-18-duchenne-first-in-human-data-wms-2026","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/zh\/rag-18-duchenne-first-in-human-data-wms-2026\/","title":{"rendered":"Ractigen Therapeutics \u62a5\u544a RAG-18 \u5728\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u60a3\u8005\u4e2d\u7684\u9996\u6b21\u4eba\u4f53\u8bd5\u9a8c\u4e2d\u53d6\u5f97\u79ef\u6781\u6570\u636e"},"content":{"rendered":"<p class=\"wp-block-paragraph\"><strong>Ractigen Therapeutics \u516c\u5e03\u4e86 RAG-18 (NCT07282652) \u7684\u79ef\u6781\u4eba\u4f53\u8bd5\u9a8c\u6570\u636e\uff0cRAG-18 \u662f\u4e00\u79cd\u7528\u4e8e\u6cbb\u7597\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (Duchenne) \u7684\u7814\u7a76\u6027\u7597\u6cd5\uff0c\u5b83\u5229\u7528\u5c0f\u6fc0\u6d3b RNA (saRNA) \u6765\u589e\u52a0\u808c\u8425\u517b\u86cb\u767d\u7684\u4ea7\u751f\u3002.<\/strong> I\u671f\u7814\u7a76\u6570\u636e\u5df2\u5728\u65e5\u672c\u5e7f\u5c9b\u4e3e\u884c\u7684\u7b2c31\u5c4a\u4e16\u754c\u808c\u8089\u5b66\u4f1a\u5e74\u4f1a\uff08WMS 2026\uff09\u4e0a\u516c\u5e03\u3002\u7814\u7a76\u7ed3\u679c\u63d0\u4f9b\u4e86\u65e9\u671f\u4e34\u5e8a\u8bc1\u636e\uff0c\u8868\u660esaRNA\u53ef\u4ee5\u5230\u8fbe\u4eba\u4f53\u9aa8\u9abc\u808c\u5e76\u6fc0\u6d3b\u4e00\u79cd\u5929\u7136\u5b58\u5728\u7684\u86cb\u767d\u8d28\u3002\u4e86\u89e3\u66f4\u591a\uff1a <a href=\"https:\/\/dmdwarrior.com\/clinical-trials\/rag-18-early-phase-1-clinical-trial-for-duchenne-muscular-dystrophy\/\">NCT07282652<\/a><\/p>\n\n\n\n<h2 class=\"wp-block-heading\">\u4ec0\u4e48\u662f\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\uff1f\u5b83\u4e3a\u4ec0\u4e48\u5982\u6b64\u91cd\u8981\uff1f<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u662f\u4e00\u79cd\u5929\u7136\u5b58\u5728\u7684\u86cb\u767d\u8d28\uff0c\u5176\u7ed3\u6784\u4e0e\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u76f8\u5173\u3002\u5728\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u4e2d\uff0c\u529f\u80fd\u6027\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u7f3a\u4e4f\u4f1a\u635f\u4f24\u808c\u8089\u7ea4\u7ef4\u3002\u65e0\u8bba\u60a3\u8005\u643a\u5e26\u4f55\u79cd\u5177\u4f53\u7684\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u57fa\u56e0\u7a81\u53d8\uff0c\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u90fd\u53ef\u4ee5\u5728\u808c\u8089\u7ec6\u80de\u819c\u4e0a\u66ff\u4ee3\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u53d1\u6325\u4f5c\u7528\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u56e0\u6b64\uff0c\u589e\u52a0\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\uff08utrophin\uff09\u7684\u6c34\u5e73\u53ef\u80fd\u63d0\u4f9b\u4e00\u79cd\u4e0d\u4f9d\u8d56\u4e8e\u60a3\u8005\u7279\u5b9a\u57fa\u56e0\u7a81\u53d8\u7684\u6cbb\u7597\u65b9\u6cd5\u3002RAG-18 \u7684\u8bbe\u8ba1\u76ee\u7684\u662f\u63d0\u9ad8\u4eba\u4f53\u81ea\u8eab\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u751f\u6210\uff0c\u800c\u4e0d\u662f\u5f15\u5165\u66ff\u4ee3\u57fa\u56e0\u3002.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">RAG-18 \u7684\u5de5\u4f5c\u539f\u7406\u662f\u4ec0\u4e48\uff1f<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">RAG-18\u91c7\u7528Ractigen Therapeutics&#039; RNA\u6fc0\u6d3b\u6280\u672f\uff0c\u5e76\u901a\u8fc7\u516c\u53f8\u81ea\u4e3b\u7814\u53d1\u7684\u8102\u8d28\u4f53\u5076\u8054\u5be1\u6838\u82f7\u9178\uff08LiCO\u2122\uff09\u6280\u672f\u9012\u9001\u3002\u8be5\u836f\u7269\u4ee5\u6bcf\u6708\u4e00\u6b21\u7684\u9759\u8109\u8f93\u6ce8\u65b9\u5f0f\u7ed9\u836f\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u8be5\u7597\u6cd5\u65e8\u5728\u5229\u7528\u7ec6\u80de\u81ea\u8eab\u7684\u8f6c\u5f55\u673a\u5236\u6765\u589e\u52a0\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u4ea7\u751f\uff0c\u65e0\u9700\u75c5\u6bd2\u8f7d\u4f53\u6216\u6c38\u4e45\u6027DNA\u7f16\u8f91\u3002RAG-18\u8fd8\u83b7\u5f97\u4e86\u7f8e\u56fd\u98df\u54c1\u836f\u54c1\u76d1\u7763\u7ba1\u7406\u5c40\uff08FDA\uff09\u6388\u4e88\u7684\u5b64\u513f\u836f\u8d44\u683c\u8ba4\u5b9a\u548c\u7f55\u89c1\u513f\u79d1\u75be\u75c5\u8d44\u683c\u8ba4\u5b9a\uff08FDA\uff1aFDA\uff09\u3002.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">\u524d\u4e09\u4f4d\u53c2\u8d5b\u8005\u5c55\u793a\u4e86\u4ec0\u4e48\uff1f<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u62a5\u544a\u7684\u6570\u636e\u6765\u81ea\u7b2c\u4e00\u6279\u7ecf\u57fa\u56e0\u786e\u8bca\u60a3\u6709\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684 4 \u81f3 15 \u5c81\u53ef\u884c\u8d70\u7537\u5b69\u3002. <strong>\u4ed6\u4eec\u6bcf\u6708\u63a5\u53d7 15 \u6beb\u514b RAG-18 \u6cbb\u7597\uff0c\u5e76\u63a5\u53d7\u968f\u8bbf\u81f3\u7b2c 169 \u5929\u3002.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u808c\u8089\u6d3b\u68c0\u663e\u793a\uff0c\u4e0e\u57fa\u7ebf\u76f8\u6bd4\uff0c\u808c\u8089\u819c\u4e0a\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u589e\u52a0\u4e86 3.5 \u81f3 5.3 \u500d\u3002.<\/strong> \u7814\u7a76\u4eba\u5458\u8fd8\u89c2\u5bdf\u5230\u808c\u8089\u7ed3\u6784\u7684\u53d8\u5316\uff0c\u5305\u62ec\u808c\u7ea4\u7ef4\u5c3a\u5bf8\u589e\u5927\u548c\u808c\u8089\u8102\u80aa\u51cf\u5c11\u3002\u808c\u8089\u6838\u78c1\u5171\u632f\u6210\u50cf\u663e\u793a\uff0c\u4e0e\u6d3b\u52a8\u6027\u808c\u8089\u6c34\u80bf\u548c\u708e\u75c7\u76f8\u5173\u7684\u6307\u6807\u6709\u6240\u964d\u4f4e\u3002\u9605\u8bfb\u66f4\u591a\uff1a <a href=\"https:\/\/dmdwarrior.com\/zh\/ractigen-therapeutics-rag-18-sarna-early-phase-1-clinical-trial-nct07282652-for-duchenne\/\" target=\"_blank\" rel=\"noreferrer noopener\">RAG-18\u7684\u5b89\u5168\u6027\u548c\u8010\u53d7\u6027<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u529f\u80fd\u6027\u7ed3\u679c\u663e\u793a\u51fa\u6df7\u5408\u4f46\u603b\u4f53\u79ef\u6781\u6216\u7a33\u5b9a\u7684\u4fe1\u53f7\u3002\u4e00\u540d\u53c2\u4e0e\u8005\u76846\u5206\u949f\u6b65\u884c\u8ddd\u79bb\u63d0\u9ad8\u4e8636.5\u7c73\uff0c\u53e6\u4e00\u540d\u53c2\u4e0e\u8005\u7684\u6b65\u884c\u8ddd\u79bb\u4fdd\u6301\u4e0d\u53d8\u3002\u7b2c\u4e09\u540d\u53c2\u4e0e\u8005\u7684\u6b65\u884c\u8ddd\u79bb\u4e0b\u964d\u4e8662.5\u7c73\u3002\u80ba\u529f\u80fd\u6d4b\u91cf\u7ed3\u679c\u663e\u793a\uff0c\u6240\u6709\u4e09\u540d\u53c2\u4e0e\u8005\u7684\u6570\u503c\u5747\u5448\u4e0a\u5347\u8d8b\u52bf\u3002\u5728\u4e3a\u671f24\u5468\u7684\u968f\u8bbf\u671f\u95f4\uff0c\u5fc3\u810f\u529f\u80fd\u5747\u4fdd\u6301\u5728\u6b63\u5e38\u8303\u56f4\u5185\u3002.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">\u5b89\u5168\u4e0e\u540e\u7eed\u6b65\u9aa4<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">RAG-18\u5728\u9996\u6279\u53d7\u8bd5\u8005\u4e2d\u663e\u793a\u51fa\u826f\u597d\u7684\u5b89\u5168\u6027\u548c\u8010\u53d7\u6027\u3002\u672a\u51fa\u73b0\u5242\u91cf\u9650\u5236\u6027\u6bd2\u6027\u3001\u4e25\u91cd\u4e0d\u826f\u4e8b\u4ef6\u62163\u7ea7\u53ca\u4ee5\u4e0a\u6cbb\u7597\u671f\u95f4\u51fa\u73b0\u7684\u4e0d\u826f\u4e8b\u4ef6\u3002\u62a5\u544a\u7684\u4e0d\u826f\u4e8b\u4ef6\u5747\u4e3a\u8f7b\u5fae\u4e14\u77ed\u6682\u7684\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u7b2c\u4e8c\u7ec4\u53d7\u8bd5\u8005\u6bcf\u6708\u670d\u7528 30 \u6beb\u514b\uff0c\u76ee\u524d\u5df2\u5168\u90e8\u5165\u7ec4\uff0c\u5b89\u5168\u6027\u968f\u8bbf\u4ecd\u5728\u8fdb\u884c\u4e2d\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u8fd9\u4e9b\u65e9\u671f\u53d1\u73b0\u4e3a\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u4e2d RNA \u6fc0\u6d3b\u7684\u673a\u5236\u63d0\u4f9b\u4e86\u4e34\u5e8a\u8bc1\u636e\uff0c\u8868\u660e RAG-18 \u53ef\u4ee5\u589e\u52a0\u4eba\u7c7b\u9aa8\u9abc\u808c\u4e2d\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u3002.<\/strong> \u7136\u800c\uff0c\u8fd9\u4e09\u540d\u53c2\u4e0e\u8005\u7684\u529f\u80fd\u7ed3\u679c\u4e0d\u4e00\uff0c\u800c\u4e14\u8be5\u7814\u7a76\u4ecd\u5904\u4e8e\u65e9\u671f\u9636\u6bb5\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u4e86\u89e3\u66f4\u591a<\/strong>:\u00a0<a href=\"https:\/\/dmdwarrior.com\/clinical-trials\/map\/\" target=\"_blank\" rel=\"noopener\">\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u4e34\u5e8a\u8bd5\u9a8c\u5730\u70b9\u5730\u56fe<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>Ractigen Therapeutics has presented positive first-in-human data for RAG-18 (NCT07282652), an investigational treatment for Duchenne muscular dystrophy (Duchenne) that uses small activating RNA (saRNA) to increase the production of utrophin. The Phase I study data were presented at the 31st Annual Congress of the World Muscle Society (WMS 2026) in Hiroshima, Japan. The results provide [&hellip;]<\/p>\n","protected":false},"author":11,"featured_media":8845,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[123,466,467,465,925],"class_list":["post-8843","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-china","tag-ractigen-therapeutics","tag-rag-18","tag-sarna","tag-utrophin"],"subtitle":"RAG-18 \u6b63\u5728\u6210\u4e3a\u6cbb\u7597\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684\u4e00\u79cd\u6f5c\u5728\u65b0\u65b9\u6cd5\u3002\u65e9\u671f\u6570\u636e\u663e\u793a\uff0c\u808c\u8089\u4e2d\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\uff08utrophin\uff09\u542b\u91cf\u589e\u52a0\uff0c\u8fd9\u9996\u6b21\u63ed\u793a\u4e86\u4eba\u4f53\u5185 RNA \u7684\u6fc0\u6d3b\u60c5\u51b5\u3002\u4f46\u8fd9\u5bf9\u4e8e\u60a3\u8005\u6765\u8bf4\u610f\u5473\u7740\u4ec0\u4e48\u5462\uff1f","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/8843","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/users\/11"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/comments?post=8843"}],"version-history":[{"count":2,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/8843\/revisions"}],"predecessor-version":[{"id":8846,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/8843\/revisions\/8846"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media\/8845"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media?parent=8843"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/categories?post=8843"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/tags?post=8843"}],"curies":[{"name":"\u53ef\u6e7f\u6027\u7c89\u5242","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}