{"id":3218,"date":"2025-03-17T10:34:47","date_gmt":"2025-03-17T07:34:47","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=3218"},"modified":"2025-03-17T10:39:51","modified_gmt":"2025-03-17T07:39:51","slug":"dyne-therapeutics-announces-phase-1-2-deliver-trial-of-dyne-251-in-duchenne-muscular-dystrophy","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/zh\/dyne-therapeutics-announces-phase-1-2-deliver-trial-of-dyne-251-in-duchenne-muscular-dystrophy\/","title":{"rendered":"Dyne Therapeutics \u5ba3\u5e03\uff0c\u5728 DYNE-251 \u6cbb\u7597\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684 1\/2 \u671f DELIVER \u8bd5\u9a8c\u4e2d\uff0c\u60a3\u8005\u5728 18 \u4e2a\u6708\u5185\u83b7\u5f97\u4e86\u524d\u6240\u672a\u6709\u7684\u6301\u7eed\u529f\u80fd\u6539\u5584"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Dyne Therapeutics \u662f\u4e00\u5bb6\u4e34\u5e8a\u9636\u6bb5\u516c\u53f8\uff0c\u4e13\u6ce8\u4e8e\u4e3a\u60a3\u6709\u9057\u4f20\u6027\u795e\u7ecf\u808c\u8089\u75be\u75c5\u7684\u4eba\u63d0\u4f9b\u6539\u53d8\u751f\u6d3b\u7684\u6cbb\u7597\u65b9\u6cd5\uff0c\u4eca\u5929\uff0c\u8be5\u516c\u53f8\u516c\u5e03\u4e86\u5176\u6b63\u5728\u8fdb\u884c\u7684 1\/2 \u671f DELIVER \u8bd5\u9a8c\u7684\u65b0\u7684\u957f\u671f\u4e34\u5e8a\u6570\u636e\uff0c\u8be5\u8bd5\u9a8c\u5bf9 DYNE-251 \u8fdb\u884c\u4e86\u7814\u7a76\uff0c\u7ed3\u679c\u8868\u660e\uff0c\u5728\u9009\u5b9a\u7684\u6ce8\u518c\u5242\u91cf 20 mg\/kg Q4W\uff08\u8fd1\u4f3c PMO \u5242\u91cf\uff09\u4e0b\uff0c\u60a3\u8005\u83b7\u5f97\u4e86\u524d\u6240\u672a\u6709\u4e14\u6301\u7eed\u7684\u529f\u80fd\u6539\u5584\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">DELIVER \u8bd5\u9a8c\u6b63\u5728\u8bc4\u4f30 DYNE-251 \u5bf9\u9002\u5408\u8fdb\u884c\u5916\u663e\u5b50 51 \u8df3\u8dc3\u7684\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD) \u60a3\u8005\u7684\u7597\u6548\uff0c\u8be5\u8bd5\u9a8c\u7684\u6700\u65b0\u7ed3\u679c\u5c06\u4e8e\u672c\u5468\u5728 2025 \u5e74\u808c\u8425\u517b\u4e0d\u826f\u75c7\u534f\u4f1a (MDA) \u4e34\u5e8a\u548c\u79d1\u5b66\u4f1a\u8bae\u4e0a\u516c\u5e03\u3002<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">DELIVER 1\/2\u671f\u8bd5\u9a8c\u62a5\u544a\u7684\u4e3b\u8981\u53d1\u73b0\u5305\u62ec\uff1a<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>\u529f\u80fd\uff1a<\/strong> \u5206\u522b\u5728 12 \u4e2a\u6708\u548c 18 \u4e2a\u6708\u65f6\uff0c20 mg\/kg\uff08\u9009\u5b9a\u6ce8\u518c\u5242\u91cf\uff09\u548c 10 mg\/kg1 DYNE-251 Q4W \u961f\u5217\u4e2d\u89c2\u5bdf\u5230\u591a\u4e2a\u529f\u80fd\u7ec8\u70b9\u76f8\u5bf9\u4e8e\u57fa\u7ebf\u7684\u663e\u8457\u4e14\u6301\u7eed\u7684\u6539\u5584\u3002\u529f\u80fd\u8bc4\u4f30\u5305\u62ec\u6b65\u901f 95 \u767e\u5206\u4f4d\u6570 (SV95C)\u3001\u5317\u6781\u661f\u6b65\u884c\u8bc4\u4f30 (NSAA)\u300110 \u7c73\u6b65\u884c\/\u8dd1\u6b65\u65f6\u95f4 (10-MWR) \u548c\u4ece\u5730\u9762\u8d77\u7acb\u7684\u65f6\u95f4\u3002\n<ul class=\"wp-block-list\">\n<li>\u4ece 6 \u4e2a\u6708\u7684\u65f6\u95f4\u70b9\u5f00\u59cb\uff0c\u5728 DELIVER 10 mg\/kg \u548c 20 mg\/kg \u7ec4\u4e2d\u89c2\u5bdf\u5230\u7684 SV95C \u76f8\u5bf9\u4e8e\u57fa\u7ebf\u7684\u53d8\u5316\u8d85\u8fc7\u4e86\u5df2\u53d1\u5e03\u7684\u5efa\u8bae\u6700\u5c0f\u4e34\u5e8a\u91cd\u8981\u5dee\u5f02 (MCID)\u3002<\/li>\n\n\n\n<li>SV95C \u662f\u8861\u91cf\u60a3\u8005\u6b63\u5e38\u65e5\u5e38\u73af\u5883\u4e2d\u6b65\u884c\u80fd\u529b\u7684\u6570\u5b57\u5316\u5ba2\u89c2\u7ed3\u679c\u6d4b\u91cf\u6307\u6807\uff0c\u88ab\u63a5\u53d7\u4e3a\u6b27\u6d32 DMD \u4e34\u5e8a\u8bd5\u9a8c\u7684\u4e3b\u8981\u7ec8\u70b9\u3002<\/li>\n<\/ul>\n<\/li>\n<\/ul>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u8868\u8fbe\uff1a<\/strong> \u5982\u4e4b\u524d\u62a5\u9053\u7684\u90a3\u6837\uff0c\u5bf9\u4e8e\u63a5\u53d7\u5916\u663e\u5b50 51 \u8df3\u8dc3\u6cbb\u7597\u7684 DMD \u60a3\u8005\uff0cDYNE-251 \u663e\u793a\u51fa\u524d\u6240\u672a\u6709\u7684\u8fd1\u5168\u957f\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u8868\u8fbe\uff08\u901a\u8fc7\u86cb\u767d\u8d28\u5370\u8ff9\u6cd5\u6d4b\u91cf\uff09\u3002\u5728 6 \u4e2a\u6708\u7684\u65f6\u95f4\u70b9\uff0c\u63a5\u53d7 20 mg\/kg DYNE-251 Q4W \u6cbb\u7597\u7684\u60a3\u8005\u7684\u5e73\u5747\u7edd\u5bf9\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u8868\u8fbe\u4e3a\u6b63\u5e38\u503c\u7684 8.72%\uff08\u6839\u636e\u808c\u8089\u542b\u91cf\u8c03\u6574\uff09\u3002Dyne \u5df2\u786e\u8ba4\uff0c\u7f8e\u56fd\u98df\u54c1\u548c\u836f\u7269\u7ba1\u7406\u5c40\u4f7f\u7528\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u4f5c\u4e3a\u66ff\u4ee3\u751f\u7269\u6807\u5fd7\u7269\u4ee5\u52a0\u901f\u5ba1\u6279\u7684\u5148\u4f8b\u4ecd\u7136\u6709\u6548\u3002<\/li>\n<\/ul>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>\u5b89\u5168\u6027\u548c\u8010\u53d7\u6027\uff1a<\/strong> \u6839\u636e\u53c2\u52a0 DELIVER \u8bd5\u9a8c\u7684 54 \u540d\u53c2\u4e0e\u8005\u7684\u6570\u636e\uff0cDYNE-251 \u663e\u793a\u51fa\u826f\u597d\u7684\u5b89\u5168\u6027\u3002\u81ea 2024 \u5e74 11 \u6708 21 \u65e5\u63d0\u4f9b\u7684\u4e0a\u4e00\u6b21\u66f4\u65b0\u4ee5\u6765\uff0c\u5b89\u5168\u6027\u4fdd\u6301\u4e0d\u53d8\uff0c\u5e76\u4e14\u6ca1\u6709\u89c2\u5bdf\u5230\u65b0\u7684\u6cbb\u7597\u76f8\u5173\u4e25\u91cd\u4e0d\u826f\u4e8b\u4ef6\u3002\n<ul class=\"wp-block-list\">\n<li>\u8fc4\u4eca\u4e3a\u6b62\uff0c\u5728 77.1 \u4e2a\u60a3\u8005\u5e74\u7684\u968f\u8bbf\u671f\u5185\uff0c\u5df2\u7ecf\u65bd\u7528\u4e86 970 \u5242\u7814\u7a76\u836f\u7269\uff0c\u5176\u4e2d\u4e00\u4e9b\u60a3\u8005\u7684\u968f\u8bbf\u65f6\u95f4\u957f\u8fbe\u7ea6 2.5 \u5e74\u3002<\/li>\n\n\n\n<li>\u8fc4\u4eca\u4e3a\u6b62\uff0c\u5df2\u65bd\u7528 546 \u5242 20 mg\/kg \u5242\u91cf\u6c34\u5e73\u7684\u7814\u7a76\u836f\u7269\u3002<\/li>\n<\/ul>\n<\/li>\n<\/ul>","protected":false},"excerpt":{"rendered":"<p>Dyne Therapeutics, a clinical-stage company focused on advancing life-transforming therapeutics for people living with genetically driven neuromuscular diseases, today announced new long-term clinical data from its ongoing Phase 1\/2 DELIVER trial of DYNE-251 demonstrating unprecedented and sustained functional improvement at the selected registrational dose of 20 mg\/kg Q4W (approximate PMO dose). The DELIVER trial is [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":3221,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[208,145,122,193,194],"class_list":["post-3218","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-dyne-therapeutics","tag-dyne-251","tag-exon-51","tag-exon-51-skipping","tag-exon-51-treatment"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/3218","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=3218"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/3218\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media\/3221"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media?parent=3218"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/categories?post=3218"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/tags?post=3218"}],"curies":[{"name":"\u53ef\u6e7f\u6027\u7c89\u5242","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}