{"id":4399,"date":"2025-09-04T14:17:40","date_gmt":"2025-09-04T11:17:40","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=4399"},"modified":"2025-09-04T14:30:44","modified_gmt":"2025-09-04T11:30:44","slug":"sqy-therapeutics-orphan-drug-ema-exon-51-skipping-therapy-sqy51-for-duchenne","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/zh\/sqy-therapeutics-orphan-drug-ema-exon-51-skipping-therapy-sqy51-for-duchenne\/","title":{"rendered":"SQY Therapeutics\u83b7\u5f97\u6b27\u6d32\u836f\u54c1\u7ba1\u7406\u5c40\u9881\u53d1\u7684\u5916\u663e\u5b5051\u8df3\u8dc3\u7597\u6cd5SQY51\u5b64\u513f\u836f\u8d44\u683c\u8ba4\u5b9a\uff0c\u7528\u4e8e\u6cbb\u7597\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7"},"content":{"rendered":"

SQY51\u5df2\u4e8e2024\u5e745\u6708\u83b7\u5f97\u7f8e\u56fd\u98df\u54c1\u836f\u54c1\u76d1\u7763\u7ba1\u7406\u5c40\u9881\u53d1\u7684\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u5b64\u513f\u836f\u8d44\u683c\u8ba4\u5b9a\uff08FDA\uff09\u3002<\/p>\n\n\n\n

SQY51 \u662f\u4e00\u79cd\u68d5\u6988\u9170\u7ed3\u5408\u7684 tc-DNA \u53cd\u4e49\u5be1\u6838\u82f7\u9178\uff0c\u65e8\u5728\u901a\u8fc7\u5728\u53d7\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u5f71\u54cd\u7684\u7537\u5b69\u4e9a\u7fa4\u7684 DMD \u57fa\u56e0 mRNA \u526a\u63a5\u8fc7\u7a0b\u4e2d\u8df3\u8fc7\u5916\u663e\u5b50 51 \u6765\u6062\u590d\u534a\u529f\u80fd\u6027\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u3002<\/p>\n\n\n\n

\u9605\u8bfb\u66f4\u591a<\/strong>: \u5373\u5c06\u63a8\u51fa\u7684\u7528\u4e8e\u6cbb\u7597\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684\u5916\u663e\u5b50 51 \u8df3\u8dc3\u7597\u6cd5<\/a><\/p>\n\n\n\n

SQY Therapeutics \u7279\u522b\u79d1\u5b66\u987e\u95ee Luis Garcia \u8868\u793a\uff1a\u201c\u6211\u4eec\u5f88\u9ad8\u5174 EMA \u83b7\u5f97\u5b64\u513f\u836f\u8d44\u683c\u8ba4\u5b9a\uff0c\u6b64\u524d\u6211\u4eec\u5df2\u7ecf\u83b7\u5f97 FDA \u7684\u5b64\u513f\u836f\u8d44\u683c\u8ba4\u5b9a\u3002\u8fd9\u4e9b\u8d44\u683c\u8ba4\u5b9a\u53cd\u6620\u4e86\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u9886\u57df\u5c1a\u672a\u6ee1\u8db3\u7684\u5de8\u5927\u9700\u6c42\uff0c\u4e5f\u589e\u5f3a\u4e86\u6211\u4eec\u5bf9 SQY51 \u6f5c\u529b\u7684\u4fe1\u5fc3\uff0c\u76f8\u4fe1\u5b83\u80fd\u591f\u4e3a\u53d7\u8fd9\u79cd\u6bc1\u706d\u6027\u75be\u75c5\u5f71\u54cd\u5e76\u62c5\u5fc3 51 \u53f7\u5916\u663e\u5b50\u8df3\u8dc3\u7684\u60a3\u8005\u53ca\u5176\u5bb6\u5ead\u5e26\u6765\u6709\u610f\u4e49\u7684\u6539\u53d8\u3002\u201d SQY51<\/strong>: \u4e00\u79cd\u6709\u671b\u6cbb\u7597\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684\u836f\u7269<\/a><\/p>\n\n\n\n

SQY51 \u76ee\u524d\u6b63\u5728\u8fdb\u884c 1\/2a \u671f AVANCE-1 \u4e34\u5e8a\u8bd5\u9a8c\uff0c\u8bd5\u9a8c\u5bf9\u8c61\u4e3a\u9002\u5408\u63a5\u53d7 51 \u53f7\u5916\u663e\u5b50\u8df3\u8dc3\u7597\u6cd5\u6cbb\u7597\u7684\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD) \u60a3\u8005\u3002\u8be5\u516c\u53f8\u5df2\u5b8c\u6210 12 \u540d 1\/2a \u671f\u60a3\u8005\u7684\u5165\u7ec4\uff0c\u9884\u8ba1 2026 \u5e74\u83b7\u5f97\u6570\u636e\u30021 \u671f\u8bd5\u9a8c\u5df2\u5b8c\u6210\uff0c2a \u671f\u8bd5\u9a8c\u6b63\u5728\u8fdb\u884c\u4e2d\u3002SQY Therapeutics \u8fd8\u8ba1\u5212\u4e3a\u5e0c\u671b\u7ee7\u7eed\u53d7\u76ca\u4e8e\u8be5\u6cbb\u7597\u7684\u53c2\u4e0e\u8005\u5f00\u5c55\u5ef6\u4f38\u671f\u8bd5\u9a8c\u3002<\/p>\n\n\n\n

\u4e86\u89e3\u66f4\u591a<\/strong>: \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":"

SQY51 had already obtained Orphan Drug Designation from the US Food and Drug Administration (FDA) in Duchenne Muscular Dystrophy in May 2024. SQY51 is a palmitoyl-conjugated tc-DNA antisense oligonucleotide designed to restore semi-functional dystrophin by skipping exon-51 during mRNA splicing of the DMD gene in a subpopulation of boys affected by Duchenne Muscular Dystrophy. Read […]<\/p>\n","protected":false},"author":1,"featured_media":4403,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[91,213,122,193,194,410,195,409,408],"class_list":["post-4399","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-ema","tag-european-medicines-agency","tag-exon-51","tag-exon-51-skipping","tag-exon-51-treatment","tag-france","tag-orphan-drug","tag-sqy-therapeutics","tag-sqy51"],"subtitle":"SQY Therapeutics \u5ba3\u5e03\uff0c\u6b27\u6d32\u836f\u54c1\u7ba1\u7406\u5c40 (EMA) \u5df2\u6388\u4e88 SQY51 \u5b64\u513f\u836f\u8d44\u683c (ODD)\uff0cSQY51 \u662f\u4e00\u79cd\u9488\u5bf9\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u524d mRNA \u5916\u663e\u5b50 51 \u7684\u9759\u8109\u6ce8\u5c04\u5206\u5b50\uff0c\u7528\u4e8e\u6cbb\u7597\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u60a3\u8005\u3002","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/4399","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=4399"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/4399\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media\/4403"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media?parent=4399"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/categories?post=4399"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/tags?post=4399"}],"curies":[{"name":"\u53ef\u6e7f\u6027\u7c89\u5242","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}