{"id":3758,"date":"2025-04-24T14:48:38","date_gmt":"2025-04-24T11:48:38","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=3758"},"modified":"2025-04-24T14:57:12","modified_gmt":"2025-04-24T11:57:12","slug":"dyne-therapeutics-receives-european-medicines-agency-ema-orphan-drug-designation-for-dyne-251-in-duchenne-muscular-dystrophy","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/zh\/dyne-therapeutics-receives-european-medicines-agency-ema-orphan-drug-designation-for-dyne-251-in-duchenne-muscular-dystrophy\/","title":{"rendered":"Dyne Therapeutics \u83b7\u5f97\u6b27\u6d32\u836f\u54c1\u7ba1\u7406\u5c40 (EMA) \u5b64\u513f\u836f\u8d44\u683c\u8ba4\u5b9a\uff0c\u7528\u4e8e\u6cbb\u7597\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684 DYNE-251"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Dyne Therapeutics\u662f\u4e00\u5bb6\u81f4\u529b\u4e8e\u4e3a\u60a3\u6709\u9057\u4f20\u6027\u795e\u7ecf\u808c\u8089\u75be\u75c5\u7684\u60a3\u8005\u5f00\u53d1\u6539\u53d8\u4eba\u751f\u7597\u6cd5\u7684\u4e34\u5e8a\u9636\u6bb5\u516c\u53f8\uff0c\u8be5\u516c\u53f8\u4eca\u5929\u5ba3\u5e03\uff0cDYNE-251\u5df2\u88ab\u6b27\u76df\u59d4\u5458\u4f1a(EC)\u6307\u5b9a\u4e3a\u6cbb\u7597\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7(DMD)\u7684\u5b64\u513f\u836f\u3002DYNE-251\u6b63\u57281\/2\u671fDELIVER\u5168\u7403\u4e34\u5e8a\u7814\u7a76\u4e2d\uff0c\u9488\u5bf9\u6613\u53d751\u53f7\u5916\u663e\u5b50\u8df3\u8dc3\u5f71\u54cd\u7684DMD\u60a3\u8005\u8fdb\u884c\u6d4b\u8bd5\u3002<a href=\"https:\/\/dmdwarrior.com\/zh\/exon-51-skipping-therapies-dmd\/\" data-type=\"post\" data-id=\"3429\">\u5916\u663e\u5b50 51 \u8df3\u8dc3\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=\"#phase-1-2-trial-of-dyne-251\">DYNE-251 \u7684 I\/II \u671f\u4e34\u5e8a\u8bd5\u9a8c<\/a><\/li><li><a href=\"#what-is-orphan-drug\">\u4ec0\u4e48\u662f\u5b64\u513f\u836f\uff1f<\/a><\/li><li><a href=\"#key-milestones-for-the-deliver-trial\">DELIVER \u8bd5\u9a8c\u7684\u5173\u952e\u91cc\u7a0b\u7891<\/a><\/li><li><a href=\"#about-dyne-251\">\u5173\u4e8eDYNE-251<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"phase-1-2-trial-of-dyne-251\">DYNE-251 \u7684 I\/II \u671f\u4e34\u5e8a\u8bd5\u9a8c<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u57283\u6708\u4efd\u4e3e\u884c\u76842025\u5e74\u808c\u8089\u840e\u7f29\u75c7\u534f\u4f1a(MDA)\u4e34\u5e8a\u4e0e\u79d1\u5b66\u4f1a\u8bae\u4e0a\uff0c\u6b63\u5728\u8fdb\u884c\u7684DYNE-251 DELIVER\u7814\u7a76\u7684\u957f\u671f\u4e34\u5e8a\u6570\u636e\u88ab\u516c\u5e03\u3002\u8fd9\u4e9b\u6570\u636e\u8868\u660e\uff0c\u5728\u9009\u5b9a\u7684\u6ce8\u518c\u5242\u91cf\u4e0b\uff0c\u60a3\u8005\u83b7\u5f97\u4e86\u65e0\u4e0e\u4f26\u6bd4\u7684\u6301\u7eed\u529f\u80fd\u6539\u5584\u3002\u6b65\u5e45\u901f\u5ea6\u7b2c95\u767e\u5206\u4f4d\u6570(SV95C)\u662f\u4e00\u9879\u5ba2\u89c2\u7684\u6570\u5b57\u5316\u7ed3\u679c\uff0c\u88ab\u516c\u8ba4\u4e3a\u6b27\u6d32DMD\u4e34\u5e8a\u7814\u7a76\u7684\u4e3b\u8981\u7ec8\u70b9\uff0c\u4e5f\u662fDELIVER\u8bd5\u9a8c\u4e2d\u4f7f\u7528\u7684\u529f\u80fd\u8bc4\u4f30\u6307\u6807\u4e4b\u4e00\u3002\uff08\u9605\u8bfb\u66f4\u591a\uff1a <a href=\"https:\/\/dmdwarrior.com\/zh\/dyne-therapeutics-announces-phase-1-2-deliver-trial-of-dyne-251-in-duchenne-muscular-dystrophy\/\">DYNE-251 \u7684 1\/2 \u671f DELIVER \u8bd5\u9a8c<\/a>)<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"what-is-orphan-drug\">\u4ec0\u4e48\u662f\u5b64\u513f\u836f\uff1f<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u6b27\u76df\u59d4\u5458\u4f1a\u6388\u4e88\u7528\u4e8e\u6cbb\u7597\u3001\u8bca\u65ad\u6216\u9884\u9632\u7f55\u89c1\u3001\u5371\u53ca\u751f\u547d\u6216\u6162\u6027\u8870\u5f31\u6027\u75be\u75c5\u7684\u836f\u7269\u548c\u751f\u7269\u5236\u5242\u5b64\u513f\u836f\u8d44\u683c\uff0c\u8fd9\u4e9b\u75be\u75c5\u6216\u75c5\u75c7\u5728\u6b27\u76df\uff08EU\uff09\u7684\u53d1\u75c5\u7387\u4f4e\u4e8e\u4e07\u5206\u4e4b\u4e94\u3002\u5b64\u513f\u836f\u8d44\u683c\u4e3a\u516c\u53f8\u63d0\u4f9b\u4e86\u4e00\u5b9a\u7684\u76ca\u5904\uff0c\u5305\u62ec\u964d\u4f4e\u76d1\u7ba1\u8d39\u7528\u3001\u4e34\u5e8a\u65b9\u6848\u534f\u52a9\u3001\u7814\u7a76\u8865\u52a9\uff0c\u4ee5\u53ca\u4e00\u65e6\u83b7\u5f97\u6279\u51c6\uff0c\u5728\u6b27\u76df\u83b7\u5f97\u957f\u8fbe10\u5e74\u7684\u5e02\u573a\u72ec\u5360\u6743\u3002DYNE-251\u8fd8\u4e8e2023\u5e743\u6708\u83b7\u5f97\u4e86\u7f8e\u56fd\u98df\u54c1\u836f\u54c1\u76d1\u7763\u7ba1\u7406\u5c40\uff08FDA\uff09\u6388\u4e88\u7684\u5b64\u513f\u836f\u8d44\u683c\uff08FDA\uff09\u548c\u7f55\u89c1\u513f\u79d1\u75be\u75c5\u8d44\u683c\u3002<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"key-milestones-for-the-deliver-trial\">DELIVER \u8bd5\u9a8c\u7684\u5173\u952e\u91cc\u7a0b\u7891<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Dyne \u7ee7\u7eed\u5728\u5168\u7403\u8303\u56f4\u5185\u5bfb\u6c42\u9488\u5bf9\u9002\u5408\u8fdb\u884c\u5916\u663e\u5b50 51 \u8df3\u8dc3\u7684 DMD \u60a3\u8005\u7684 DYNE-251 \u7684\u5feb\u901f\u5ba1\u6279\u9014\u5f84\u3002<\/li>\n\n\n\n<li>\u4f5c\u4e3a DELIVER \u8bd5\u9a8c\u7684\u4e00\u90e8\u5206\uff0cDyne \u5df2\u5b8c\u6210 32 \u540d\u60a3\u8005\u7684\u6ce8\u518c\u6269\u5c55\u961f\u5217\u62db\u52df\u3002\u8be5\u961f\u5217\u7684\u6570\u636e\u8ba1\u5212\u4e8e 2025 \u5e74\u5e95\u516c\u5e03\u3002<\/li>\n\n\n\n<li>Dyne \u9884\u8ba1\u5c06\u4e8e 2026 \u5e74\u521d\u63d0\u4ea4\u751f\u7269\u5236\u54c1\u8bb8\u53ef\u7533\u8bf7 (BLA) \u4ee5\u83b7\u5f97\u7f8e\u56fd\u52a0\u901f\u6279\u51c6\u3002<\/li>\n<\/ul>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"about-dyne-251\">\u5173\u4e8eDYNE-251<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">DYNE-251 \u662f\u4e00\u79cd\u7814\u7a76\u6027\u6cbb\u7597\u836f\u7269\uff0c\u76ee\u524d\u6b63\u5728\u8fdb\u884c 1\/2 \u671f\u5168\u7403 DELIVER \u4e34\u5e8a\u8bd5\u9a8c\uff0c\u9488\u5bf9\u9002\u5408\u8fdb\u884c\u5916\u663e\u5b50 51 \u8df3\u8dc3\u7684 DMD \u60a3\u8005\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">DYNE-251 \u5df2\u88ab\u7f8e\u56fd\u98df\u54c1\u836f\u54c1\u76d1\u7763\u7ba1\u7406\u5c40\u6388\u4e88\u5feb\u901f\u901a\u9053\u3001\u5b64\u513f\u836f\u548c\u7f55\u89c1\u513f\u79d1\u75be\u75c5\u79f0\u53f7\uff0c\u7528\u4e8e\u6cbb\u7597\u53ef\u901a\u8fc7\u5916\u663e\u5b50 51 \u8df3\u8dc3\u6cbb\u7597\u7684 DMD \u7a81\u53d8\u3002<\/p>","protected":false},"excerpt":{"rendered":"<p>Today, Dyne Therapeutics, a clinical-stage company dedicated to developing life-changing treatments for individuals with genetically driven neuromuscular diseases, announced that DYNE-251 has been designated as an orphan drug by the European Commission (EC) for the treatment of Duchenne muscular dystrophy (DMD). DYNE-251 is being tested in DMD patients who are susceptible to exon 51 skipping [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":3760,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[143,208,145,91,213,122,193,194,195],"class_list":["post-3758","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-dyne","tag-dyne-therapeutics","tag-dyne-251","tag-ema","tag-european-medicines-agency","tag-exon-51","tag-exon-51-skipping","tag-exon-51-treatment","tag-orphan-drug"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/3758","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=3758"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/3758\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media\/3760"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media?parent=3758"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/categories?post=3758"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/tags?post=3758"}],"curies":[{"name":"\u53ef\u6e7f\u6027\u7c89\u5242","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}