{"id":3230,"date":"2025-03-17T14:28:46","date_gmt":"2025-03-17T11:28:46","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=3230"},"modified":"2025-03-17T14:28:48","modified_gmt":"2025-03-17T11:28:48","slug":"avidity-biosciences-announces-positive-topline-del-zota-data-showing-consistent-statistically-significant-improvements-in-dystrophin","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/zh\/avidity-biosciences-announces-positive-topline-del-zota-data-showing-consistent-statistically-significant-improvements-in-dystrophin\/","title":{"rendered":"Avidity Biosciences \u516c\u5e03 Del-zota \u7684\u6b63\u9762\u9876\u7ebf\u6570\u636e\uff0c\u663e\u793a\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u6c34\u5e73\u6301\u7eed\u3001\u7edf\u8ba1\u4e0a\u663e\u8457\u6539\u5584"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Avidity Biosciences \u5ba3\u5e03\u79ef\u6781\u7684 Del-zota \u9876\u7ebf\u6570\u636e\u663e\u793a\uff0c\u5728 1\/2 \u671f EXPLORE44\u00ae \u8bd5\u9a8c\u4e2d\uff0c\u9002\u5408\u8fdb\u884c\u5916\u663e\u5b50 44 \u8df3\u8dc3\u7684\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u53c2\u4e0e\u8005\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u3001\u5916\u663e\u5b50\u8df3\u8dc3\u548c\u808c\u9178\u6fc0\u9176\u53d6\u5f97\u4e86\u6301\u7eed\u3001\u7edf\u8ba1\u5b66\u4e0a\u663e\u7740\u7684\u6539\u5584\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u8be5\u6570\u636e\u5c06\u5728 2025 \u5e74 3 \u6708 16 \u65e5\u81f3 19 \u65e5\u5728\u5fb7\u514b\u8428\u65af\u5dde\u8fbe\u62c9\u65af\u4e3e\u884c\u7684 2025 \u5e74\u808c\u8089\u840e\u7f29\u75c7\u534f\u4f1a (MDA) \u4e34\u5e8a\u4e0e\u79d1\u5b66\u4f1a\u8bae\u4e0a\u4ee5\u53e3\u5934\u548c\u6d77\u62a5\u5c55\u793a\u7684\u5f62\u5f0f\u91cd\u70b9\u5c55\u793a\u3002<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Avidity Biosciences EXPLORE44\uff08Del-zota\uff09\u4e34\u5e8a\u8bd5\u9a8c<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u963f\u80af\u8272\u5927\u5b66\u533b\u5b66\u79d1\u5b66\u9662\u548c\u963f\u80af\u8272\u5dde\u513f\u7ae5\u533b\u9662\u513f\u79d1\u526f\u6559\u6388 Aravindhan Veerapandiyan \u533b\u5b66\u535a\u58eb\u8868\u793a\uff1a\u201cDel-zota \u5728\u591a\u9879\u6307\u6807\u4e0a\u90fd\u8868\u73b0\u51fa\u663e\u8457\u6539\u5584\uff0c\u5305\u62ec\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u751f\u6210\u91cf\u5927\u5e45\u589e\u52a0\uff0c\u8840\u6e05\u808c\u9178\u6fc0\u9176\u6c34\u5e73\u663e\u8457\u964d\u4f4e\u81f3\u4ec5\u670d\u7528\u4e09\u6b21\u540e\u63a5\u8fd1\u6b63\u5e38\u6c34\u5e73\u3002\u5982\u6b64\u77ed\u7684\u65f6\u95f4\u5185\u53d6\u5f97\u5982\u6b64\u4e00\u81f4\u7684\u7ed3\u679c\uff0c\u518d\u52a0\u4e0a\u826f\u597d\u7684\u5b89\u5168\u6027\u548c\u8010\u53d7\u6027\uff0c\u51f8\u663e\u4e86 del-zota \u6709\u671b\u6210\u4e3a\u4e00\u79cd\u7a81\u7834\u6027\u7597\u6cd5\uff0c\u7528\u4e8e\u6cbb\u7597\u643a\u5e26\u53ef\u8fdb\u884c\u5916\u663e\u5b50 44 \u8df3\u8dc3\u7684\u9057\u4f20\u53d8\u5f02\u7684 DMD \u60a3\u8005\u3002\u201d<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u201c\u8fd9\u4e9b\u7ed3\u679c\u4e3a\u53d7 DMD \u5f71\u54cd\u7684\u60a3\u8005\u548c\u5bb6\u5ead\u5e26\u6765\u4e86\u65b0\u7684\u5e0c\u671b\uff0c\u4ed6\u4eec\u8feb\u5207\u9700\u8981\u9488\u5bf9\u6027\u7684\u6cbb\u7597\u6765\u4fdd\u6301\u808c\u8089\u5b8c\u6574\u6027\uff0c\u5e76\u53ef\u80fd\u9884\u9632\u6216\u5ef6\u7f13\u4e0e\u8be5\u75be\u75c5\u76f8\u5173\u7684\u808c\u8089\u65e0\u529b\u548c\u529f\u80fd\u4e27\u5931\u7684\u8fdb\u5c55\u3002\u201d<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">\u4ec0\u4e48\u662f Del-Zota\uff1f<\/h2>\n\n\n\n<figure class=\"wp-block-image size-large\"><img loading=\"lazy\" decoding=\"async\" width=\"1024\" height=\"576\" src=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/Avidity-Biosciences-Del-Zota-1024x576.jpg\" alt=\"Avidity Biosciences Del-zota EXPLORE44 \u5916\u663e\u5b50 44 \u8df3\u8dc3\" class=\"wp-image-3234\" title=\"\" srcset=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/Avidity-Biosciences-Del-Zota-1024x576.jpg 1024w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/Avidity-Biosciences-Del-Zota-300x169.jpg 300w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/Avidity-Biosciences-Del-Zota-768x432.jpg 768w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/Avidity-Biosciences-Del-Zota-1536x864.jpg 1536w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/Avidity-Biosciences-Del-Zota-18x10.jpg 18w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/Avidity-Biosciences-Del-Zota-747x420.jpg 747w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/Avidity-Biosciences-Del-Zota-150x84.jpg 150w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/Avidity-Biosciences-Del-Zota-696x392.jpg 696w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/Avidity-Biosciences-Del-Zota-1068x601.jpg 1068w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/Avidity-Biosciences-Del-Zota.jpg 1920w\" sizes=\"auto, (max-width: 1024px) 100vw, 1024px\" \/><\/figure>\n\n\n\n<p class=\"wp-block-paragraph\">Del-zota \u65e8\u5728\u5c06\u78f7\u4e8c\u9170\u80fa\u5417\u5549\u5be1\u805a\u4f53 (PMO) \u9012\u9001\u81f3\u9aa8\u9abc\u548c\u5fc3\u808c\u7ec4\u7ec7\uff0c\u4ee5\u7279\u5f02\u6027\u8df3\u8fc7\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u57fa\u56e0\u7684\u5916\u663e\u5b50 44\uff0c\u5e76\u5b9e\u73b0\u8fd1\u4e4e\u5168\u957f\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u4ea7\u751f\u3002Del-zota \u5df2\u83b7\u5f97\u7f8e\u56fd\u98df\u54c1\u836f\u54c1\u7ba1\u7406\u5c40 (FDA) \u548c\u6b27\u6d32\u836f\u54c1\u7ba1\u7406\u5c40 (EMA) \u7684\u5b64\u513f\u836f\u8d44\u683c\u8ba4\u5b9a\u3002FDA \u8fd8\u83b7\u5f97\u4e86 del-zota \u7f55\u89c1\u513f\u79d1\u75be\u75c5\u548c\u5feb\u901f\u901a\u9053\u8d44\u683c\u8ba4\u5b9a\u3002Del-zota \u662f Avidity \u76ee\u524d\u6b63\u5728\u5f00\u53d1\u7684\u7528\u4e8e\u6cbb\u7597 DMD \u7684\u591a\u79cd AOC \u4e2d\u7684\u7b2c\u4e00\u4e2a\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>MDA \u4e0a\u5448\u73b0\u7684\u6570\u636e\u7a81\u51fa\u663e\u793a\u4e86 del-zota 5 mg \/ kg \u548c 10 mg \/ kg \u7ec4\u6240\u6709\u53c2\u6570\u7684\u4e00\u81f4\u6570\u636e\uff0c\u5305\u62ec\uff1a<\/strong><\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u9776\u5411\u9012\u9001 PMO \u5bfc\u81f4\u9aa8\u9abc\u808c\u4e2d\u7684\u7ec4\u7ec7\u6d53\u5ea6\u8fbe\u5230\u7ea6 200nM\uff1b<\/li>\n\n\n\n<li>\u5916\u663e\u5b50 44 \u8df3\u8dc3\u4e2d\u7ea6 40% \u5177\u6709\u7edf\u8ba1\u5b66\u663e\u8457\u589e\u52a0\uff1b<\/li>\n\n\n\n<li>\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u4ea7\u751f\u6bd4\u6b63\u5e38\u503c\u663e\u8457\u589e\u52a0\u7ea6 25%\uff0c\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u603b\u91cf\u6062\u590d\u81f3\u6b63\u5e38\u503c\u7684 58%\uff1b<\/li>\n\n\n\n<li>\u4e0e\u57fa\u7ebf\u76f8\u6bd4\uff0c\u808c\u9178\u6fc0\u9176\u6c34\u5e73\u964d\u4f4e\u81f3\u63a5\u8fd1\u6b63\u5e38\u6c34\u5e73\uff0c\u4e14\u964d\u4f4e\u5e45\u5ea6\u8d85\u8fc7 80%\uff1a\n<ul class=\"wp-block-list\">\n<li>\u540c\u6837\u5730\uff0c\u63a5\u53d7\u5b89\u6170\u5242\u6cbb\u7597\u7684\u53d7\u8bd5\u8005\u5728\u63a5\u53d7 del-zota \u6cbb\u7597\u540e\uff0c\u808c\u9178\u6fc0\u9176\u6c34\u5e73\u4e5f\u964d\u4f4e\u81f3\u63a5\u8fd1\u6b63\u5e38\u6c34\u5e73\uff1b<\/li>\n\n\n\n<li>\u5728 EXPLORE44-OLE \u8bd5\u9a8c\u4e2d\uff0c\u808c\u9178\u6fc0\u9176\u6c34\u5e73\u663e\u8457\u964d\u4f4e\uff0c\u6301\u7eed\u6cbb\u7597\u957f\u8fbe\u4e00\u5e74\uff1b\u5e76\u4e14\uff0c<\/li>\n<\/ul>\n<\/li>\n\n\n\n<li>Del-zota \u5728\u4e24\u79cd\u5242\u91cf\u4e0b\u5747\u8868\u73b0\u51fa\u826f\u597d\u7684\u5b89\u5168\u6027\u548c\u8010\u53d7\u6027\uff0c\u5927\u591a\u6570\u6cbb\u7597\u51fa\u73b0\u7684\u4e0d\u826f\u4e8b\u4ef6 (TEAE) \u5747\u4e3a\u8f7b\u5ea6\u6216\u4e2d\u5ea6\u3002<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u4e86\u89e3\u66f4\u591a\uff1a<\/strong> <a href=\"https:\/\/dmdwarrior.com\/zh\/next-generation-exon-skipping-therapies-for-duchenne-muscular-dystrophy\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u4e3a\u6cbb\u7597\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u800c\u5f00\u53d1\u7684\u4e0b\u4e00\u4ee3\u5916\u663e\u5b50\u8df3\u8dc3\u7597\u6cd5<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>Avidity Biosciences Announces Positive Topline Del-zota Data Showing Consistent, Statistically Significant Improvements in Dystrophin, Exon Skipping, and Creatine Kinase in Phase 1\/2 EXPLORE44\u00ae Trial Participants with Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping. The data will be highlighted in an oral and poster presentation at the 2025 Muscular Dystrophy Association (MDA) Clinical &amp; Scientific [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":3232,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[126,298,141,127,246,299],"class_list":["post-3230","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-avidity-biosciences","tag-del-zota","tag-exon-44","tag-exon-44-skipping","tag-exon-44-skipping-therapies","tag-explore44"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/3230","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=3230"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/3230\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media\/3232"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media?parent=3230"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/categories?post=3230"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/tags?post=3230"}],"curies":[{"name":"\u53ef\u6e7f\u6027\u7c89\u5242","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}