{"id":5315,"date":"2025-12-12T17:44:59","date_gmt":"2025-12-12T14:44:59","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=5315"},"modified":"2025-12-12T17:51:07","modified_gmt":"2025-12-12T14:51:07","slug":"ractigen-therapeutics-study-of-rag-18-sarna-for-duchenne-muscular-dystrophy","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/zh\/ractigen-therapeutics-study-of-rag-18-sarna-for-duchenne-muscular-dystrophy\/","title":{"rendered":"Ractigen Therapeutics \u5728 IIT \u7814\u7a76\u4e2d\u4e3a\u9996\u4f4d\u60a3\u8005\u6ce8\u5c04 RAG-18\uff0c\u8fd9\u662f\u4e00\u79cd\u53ef\u80fd\u6539\u53d8\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u6cbb\u7597\u683c\u5c40\u7684 saRNA \u7597\u6cd5"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Ractigen Therapeutics\uff0c\u4e00\u5bb6\u5f00\u521b\u6027\u7684\u5c0f\u6fc0\u6d3bRNA\uff08saRNA\uff09\u7597\u6cd5\u5f00\u53d1\u5546\uff0c\u4eca\u5929\u5ba3\u5e03\u5176\u7814\u7a76\u8005\u53d1\u8d77\u7684RAG-18\u8bd5\u9a8c\uff08IIT\uff09\u7684\u9996\u4f8b\u60a3\u8005\u5728\u5317\u4eac\u534f\u548c\u533b\u9662\uff08\u4e2d\u56fd\u533b\u5b66\u79d1\u5b66\u9662\uff09\u6210\u529f\u63a5\u53d7\u7ed9\u836f\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u8fd9\u4e00\u91cc\u7a0b\u7891\u6807\u5fd7\u7740\u5bf9\u6297\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD) \u7684\u6597\u4e89\u8fce\u6765\u4e86\u4e00\u4e2a\u53d8\u9769\u6027\u7684\u65f6\u523b\uff0c\u4f7f RAG-18 \u6210\u4e3a\u8be5\u9886\u57df\u6f5c\u5728\u7684\u53d8\u9769\u8005\u3002.<\/strong> RAG-18 \u5229\u7528\u4e00\u79cd\u65b0\u578b saRNA \u673a\u5236\u6765\u4e0a\u8c03 UTRN \u8868\u8fbe\uff0c\u4ee3\u8868\u4e86\u4e00\u79cd\u9769\u547d\u6027\u7684\u6cbb\u7597\u65b9\u6cd5\uff0c\u65e8\u5728\u514b\u670d\u5f53\u524d\u6cbb\u7597\u7684\u5c40\u9650\u6027\uff0c\u5e76\u53ef\u80fd\u4e3a\u6240\u6709\u53d7\u8fd9\u79cd\u6bc1\u706d\u6027\u75be\u75c5\u5f71\u54cd\u7684\u60a3\u8005\u63d0\u4f9b\u666e\u904d\u7684\u89e3\u51b3\u65b9\u6848\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u8be5\u521b\u65b0\u6027\u7814\u7a76\u5c0f\u7ec4\u7531\u5317\u4eac\u534f\u548c\u533b\u9662\u795e\u7ecf\u5185\u79d1\u526f\u4e3b\u4efb\u3001\u795e\u7ecf\u808c\u8089\u75be\u75c5\u9886\u57df\u6770\u51fa\u4e13\u5bb6\u6234\u6bc5\u6559\u6388\u9886\u5bfc\u3002\u8be5\u7814\u7a76\u65e8\u5728\u8bc4\u4f30RAG-18\u7684\u5b89\u5168\u6027\u548c\u836f\u4ee3\u52a8\u529b\u5b66\u7279\u5f81\uff0c\u5e76\u63a2\u7d22\u5176\u6539\u5584\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\uff08DMD\uff09\u60a3\u8005\u808c\u8089\u529f\u80fd\u7684\u6f5c\u529b\u3002. <strong>\u6234\u6559\u6388\u8868\u793a\uff1a\u201c\u6211\u4eec\u5f88\u9ad8\u5174\u80fd\u542f\u52a8RAG-18\u7684\u4e34\u5e8a\u8bd5\u9a8c\uff0c\u8fd9\u662f\u4e00\u79cd\u6cbb\u7597\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684\u524d\u6cbf\u7597\u6cd5\u3002\u6211\u4eec\u671f\u5f85\u79ef\u6781\u7684\u4e34\u5e8a\u7ed3\u679c\uff0c\u8fd9\u5c06\u4e3a\u60a3\u8005\u5f00\u8f9f\u65b0\u7684\u521b\u65b0\u6cbb\u7597\u9009\u62e9\uff0c\u5e76\u663e\u8457\u63d0\u9ad8\u4ed6\u4eec\u7684\u751f\u6d3b\u8d28\u91cf\u3002\u201d<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Ractigen Therapeutics\u521b\u59cb\u4eba\u517c\u9996\u5e2d\u6267\u884c\u5b98\u674e\u9f99\u6210\u535a\u58eb\u5bf9\u8fd9\u9879\u8bd5\u9a8c\u5145\u6ee1\u70ed\u60c5\uff0c\u4ed6\u8868\u793a\uff1a\u201c\u542f\u52a8\u8fd9\u9879\u7814\u7a76\u662fRAG-18\u53d1\u5c55\u5386\u7a0b\u4e2d\u7684\u5173\u952e\u4e00\u6b65\uff0c\u51f8\u663e\u4e86\u5176\u5728\u514b\u670dDMD\u6311\u6218\u65b9\u9762\u7684\u72ec\u7279\u4f5c\u7528\u3002\u8fd9\u79cd\u7597\u6cd5\u6709\u671b\u89e3\u51b3\u6240\u6709\u4e0eDMD\u76f8\u5173\u7684\u57fa\u56e0\u7a81\u53d8\uff0c\u5c55\u73b0\u4e86RNA\u6fc0\u6d3b\uff08RNAa\uff09\u6280\u672f\u5728\u4e34\u5e8a\u5b9e\u8df5\u4e2d\u7684\u9769\u547d\u6027\u4f5c\u7528\uff0c\u5e76\u4e3a\u4ee5\u5f80\u65e0\u6cd5\u6cbb\u6108\u7684\u75be\u75c5\u5e26\u6765\u4e86\u5e0c\u671b\u3002\u201d<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">\u4ec0\u4e48\u662f RAG-18 saRNA\uff1f<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>RAG-18 \u662f\u9996\u4e2a\u6b64\u7c7b saRNA \u5019\u9009\u836f\u7269\uff0c\u65e8\u5728\u901a\u8fc7 RNAa \u673a\u5236\u7279\u5f02\u6027\u5730\u9776\u5411\u548c\u6fc0\u6d3b\u808c\u8089\u7ec6\u80de\u4e2d\u7684 UTRN \u57fa\u56e0\u8868\u8fbe\u3002.<\/strong> UTRN \u57fa\u56e0\u7f16\u7801\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u5728\u7ed3\u6784\u548c\u529f\u80fd\u4e0a\u4e0e\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u76f8\u4f3c\uff0c\u5176\u4e0a\u8c03\u53ef\u80fd\u4f5c\u4e3a DMD \u808c\u8089\u7ec6\u80de\u4e2d\u7f3a\u5931\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u529f\u80fd\u66ff\u4ee3\u54c1\uff0c\u4ece\u800c\u4e3a\u6240\u6709 DMD \u60a3\u8005\u63d0\u4f9b\u6cbb\u7597\uff0c\u800c\u65e0\u9700\u8003\u8651\u5177\u4f53\u7684\u7a81\u53d8\u4f4d\u7f6e\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u4e34\u5e8a\u524d\u6570\u636e\u8868\u660e\uff0c\u5229\u7528 Ractigen \u4e13\u6709\u7684 LiCO\u2122\uff08\u8102\u8d28\u5076\u8054\u5be1\u6838\u82f7\u9178\uff09\u6280\u672f\u9012\u9001\u7684 RAG-18 \u5df2\u6709\u6548\u4e0a\u8c03 UTRN \u8868\u8fbe\uff0c\u51cf\u8f7b\u808c\u8089\u635f\u4f24\u5e76\u6539\u5584\u808c\u8089\u529f\u80fd\uff0c\u5728\u6cbb\u7597 DMD \u60a3\u8005\u65b9\u9762\u8868\u73b0\u51fa\u5de8\u5927\u7684\u6f5c\u529b\u3002. <strong>RAG-18 \u4e8e 2024 \u5e74\u83b7\u5f97\u7f8e\u56fd\u98df\u54c1\u836f\u54c1\u76d1\u7763\u7ba1\u7406\u5c40 (FDA) \u7684\u5b64\u513f\u836f\u8d44\u683c\u8ba4\u5b9a (ODD) \u548c\u7f55\u89c1\u513f\u79d1\u75be\u75c5\u8d44\u683c\u8ba4\u5b9a (RPDD) (FDA)\uff0c\u51f8\u663e\u4e86\u5176\u4f5c\u4e3a DMD \u60a3\u8005\u6cbb\u7597\u9009\u62e9\u7684\u6f5c\u529b\u3002.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u5173\u6ce8\u6b64\u9875\u9762<\/strong>&nbsp;&gt;&gt;&gt;&nbsp;<a href=\"https:\/\/dmdwarrior.com\/zh\/dmd-therapies\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u6240\u6709\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684\u4e34\u5e8a\u8bd5\u9a8c<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>Ractigen Therapeutics, a pioneering developer of small activating RNA (saRNA) therapeutics, today announced the successful dosing of the first patient in its investigator-initiated trial (IIT) of RAG-18 at Peking Union Medical College Hospital (PUMCH), Chinese Academy of Medical Sciences in Beijing. This milestone marks a transformative moment in the fight against Duchenne Muscular Dystrophy (DMD), [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":5316,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[27],"tags":[123,466,467,465,468],"class_list":["post-5315","post","type-post","status-publish","format-standard","has-post-thumbnail","category-research","tag-china","tag-ractigen-therapeutics","tag-rag-18","tag-sarna","tag-utrn-expression"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/5315","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\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/comments?post=5315"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/5315\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media\/5316"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media?parent=5315"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/categories?post=5315"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/tags?post=5315"}],"curies":[{"name":"\u53ef\u6e7f\u6027\u7c89\u5242","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}