{"id":5323,"date":"2025-12-15T21:53:45","date_gmt":"2025-12-15T18:53:45","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=5323"},"modified":"2025-12-15T21:57:24","modified_gmt":"2025-12-15T18:57:24","slug":"ractigen-therapeutics-rag-18-sarna-early-phase-1-clinical-trial-nct07282652-for-duchenne","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/zh\/ractigen-therapeutics-rag-18-sarna-early-phase-1-clinical-trial-nct07282652-for-duchenne\/","title":{"rendered":"\u603b\u90e8\u4f4d\u4e8e\u4e2d\u56fd\u7684Ractigen Therapeutics\u516c\u53f8\u542f\u52a8\u4e34\u5e8a\u8bd5\u9a8c\uff0c\u4ee5\u7814\u7a76RAG-18\u5728\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\uff08DMD\uff09\u60a3\u8005\u4e2d\u7684\u5b89\u5168\u6027\u548c\u8010\u53d7\u6027\u3002"},"content":{"rendered":"<p class=\"wp-block-paragraph\">\u6839\u636e Ractigen Therapeutics\uff0c\u8fd9\u9879\u7814\u7a76\u662f\u4e00\u9879\u5f00\u653e\u6807\u7b7e\u3001\u5355\u81c2\u3001\u5242\u91cf\u9012\u589e\u7814\u7a76\uff0c\u65e8\u5728\u8bc4\u4f30 RAG-18 \u5728\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD) \u513f\u79d1\u60a3\u8005\u4e2d\u7684\u5b89\u5168\u6027\u3001\u8010\u53d7\u6027\u3001\u836f\u4ee3\u52a8\u529b\u5b66 (PK)\u3001\u836f\u6548\u5b66 (PD) \u548c\u521d\u6b65\u7597\u6548\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u8be5\u7814\u7a76\u5c06\u62db\u52df\u7ea612\u540d\u53d7\u8bd5\u8005\uff0c\u5206\u4e3a\u56db\u4e2a\u961f\u5217\uff0c\u4ee5\u8bc4\u4f30\u9012\u589e\u9759\u8109\u5242\u91cf\u7684\u5b89\u5168\u6027\u548c\u8010\u53d7\u6027\u3002\u6b21\u8981\u76ee\u6807\u5305\u62ec\u63cf\u8ff0\u836f\u4ee3\u52a8\u529b\u5b66\uff08PK\uff09\/\u836f\u6548\u5b66\uff08PD\uff09\u7279\u5f81\uff0c\u5e76\u901a\u8fc7\u808c\u8089\u751f\u7269\u6807\u5fd7\u7269\u3001\u808c\u8089\u6210\u5206\u3001\u5fc3\u80ba\u529f\u80fd\u548c\u8fd0\u52a8\u8868\u73b0\u7684\u53d8\u5316\u6765\u8bc4\u4f30\u63a2\u7d22\u6027\u7597\u6548\u3002\u662f\u5426\u5347\u7ea7\u5230\u4e0b\u4e00\u5242\u91cf\u6c34\u5e73\u5c06\u57fa\u4e8e\u5bf9\u524d\u4e00\u961f\u5217\u7684\u5168\u9762\u5b89\u5168\u6027\u8bc4\u4f30\u3002. <strong>\u9605\u8bfb\u66f4\u591a<\/strong>: <a href=\"https:\/\/dmdwarrior.com\/zh\/ractigen-therapeutics-study-of-rag-18-sarna-for-duchenne-muscular-dystrophy\/\" target=\"_blank\" rel=\"noreferrer noopener\">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<\/a><\/p>\n\n\n\n<div class=\"wp-block-rank-math-toc-block\" id=\"rank-math-toc\"><h2>\u76ee\u5f55<\/h2><nav><ul><li><a href=\"#what-is-rag-18-sa-rna\">\u4ec0\u4e48\u662f RAG-18 saRNA\uff1f<\/a><\/li><li><a href=\"#rag-18-sa-rna-early-phase-1-nct-07282652\">RAG-18 saRNA \u65e9\u671f 1 \u671f\u4e34\u5e8a\u8bd5\u9a8c \u2013 NCT07282652<\/a><\/li><li><a href=\"#how-to-participate-in-the-wve-n-531-clinical-trial\">\u5982\u4f55\u53c2\u4e0e RAG-18 saRNA \u4e34\u5e8a\u8bd5\u9a8c<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"what-is-rag-18-sa-rna\">\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<h2 class=\"wp-block-heading\" id=\"rag-18-sa-rna-early-phase-1-nct-07282652\">RAG-18 saRNA \u65e9\u671f 1 \u671f\u4e34\u5e8a\u8bd5\u9a8c \u2013 NCT07282652<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u4e00\u9879\u7814\u7a76\u8005\u53d1\u8d77\u7684\u8bc4\u4f30 RAG-18 \u5728\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u513f\u79d1\u60a3\u8005\u4e2d\u7684\u5b89\u5168\u6027\/\u8010\u53d7\u6027\u3001\u836f\u4ee3\u52a8\u529b\u5b66\u3001\u836f\u6548\u5b66\u548c\u521d\u6b65\u7597\u6548\u7684\u7814\u7a76\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u5b66\u4e60\u5f00\u59cb\u65f6\u95f4\uff08\u9884\u8ba1\uff09<\/strong>: 2025-12<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u5c0f\u5b66\u5b8c\u6210\u60c5\u51b5\uff08\u9884\u8ba1\uff09<\/strong>: 2026-11<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u7814\u7a76\u5b8c\u6210\u60c5\u51b5\uff08\u9884\u8ba1\uff09<\/strong>: 2026-11<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u53c2\u4e0e\u4eba\u6570\uff08\u9884\u8ba1\uff09<\/strong>: 12<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u5b66\u4e60\u7c7b\u578b<\/strong>\uff1a\u4ecb\u5165<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u5730\u70b9<\/strong>\uff1a \u4e2d\u56fd<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u9002\u5408\u5b66\u4e60\u7684\u5e74\u9f84<\/strong>4\u5c81\u81f315\u5c81\uff08\u513f\u7ae5\uff09<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u4e3b\u8981\u7eb3\u5165\u548c\u6392\u9664\u6807\u51c6\u3002\u2013 <a href=\"https:\/\/clinicaltrials.gov\/study\/NCT07282652\" target=\"_blank\" data-type=\"link\" data-id=\"https:\/\/clinicaltrials.gov\/study\/NCT07282652\" rel=\"noreferrer noopener\">NCT07282652<\/a> \u2013<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"how-to-participate-in-the-wve-n-531-clinical-trial\">\u5982\u4f55\u53c2\u4e0e RAG-18 saRNA \u4e34\u5e8a\u8bd5\u9a8c<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u8981\u53c2\u4e0e RAG-18 saRNA \u65e9\u671f 1 \u671f\u4e34\u5e8a\u8bd5\u9a8c\uff0c\u60a8\u53ef\u4ee5\u4f7f\u7528\u4ee5\u4e0b\u8054\u7cfb\u4fe1\u606f\u3002.\u00a0<strong>\u4e86\u89e3\u66f4\u591a<\/strong>:\u00a0<a href=\"https:\/\/dmdwarrior.com\/zh\/how-to-participate-in-clinical-trials-for-duchenne\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u5982\u4f55\u53c2\u52a0\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD) \u4e34\u5e8a\u8bd5\u9a8c\uff1f<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u59d3\u540d<\/strong>: \u5cb1\u5cb1<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u7535\u8bdd\u53f7\u7801<\/strong>: +86 138 111 77 531<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u7535\u5b50\u90ae\u4ef6<\/strong>:\u00a0pumchdy@pumch.cn<\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u9605\u8bfb\u66f4\u591a<\/strong>:&nbsp;<a href=\"https:\/\/dmdwarrior.com\/zh\/dmd-therapies\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u4e34\u5e8a\u8bd5\u9a8c\uff08\u6240\u6709\u7814\u7a76\u5217\u8868\uff09<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><\/p>","protected":false},"excerpt":{"rendered":"<p>According to Ractigen Therapeutics, this study is an open-label, single-arm, dose-escalation study designed to evaluate the safety, tolerance, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary efficacy of RAG-18 in pediatric patients with Duchenne Muscular Dystrophy (DMD). The study will enroll approximately 12 subjects into four cohorts to assess the safety and tolerability of ascending intravenous doses. [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":5327,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[27],"tags":[123,137,242,466,467,465,468],"class_list":["post-5323","post","type-post","status-publish","format-standard","has-post-thumbnail","category-research","tag-china","tag-clinical-trial","tag-clinical-trials","tag-ractigen-therapeutics","tag-rag-18","tag-sarna","tag-utrn-expression"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/5323","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=5323"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/5323\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media\/5327"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media?parent=5323"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/categories?post=5323"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/tags?post=5323"}],"curies":[{"name":"\u53ef\u6e7f\u6027\u7c89\u5242","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}