{"id":3088,"date":"2025-03-07T12:49:51","date_gmt":"2025-03-07T09:49:51","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=3088"},"modified":"2025-03-08T13:35:34","modified_gmt":"2025-03-08T10:35:34","slug":"solid-biosciences-plans-meeting-with-fda-in-mid-2025-for-sgt-003-gene-therapy","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/zh\/solid-biosciences-plans-meeting-with-fda-in-mid-2025-for-sgt-003-gene-therapy\/","title":{"rendered":"Solid Biosciences \u8ba1\u5212\u4e8e 2025 \u5e74\u4e2d\u671f\u4e0e FDA \u4e3e\u884c\u4f1a\u8bae\uff0c\u8ba8\u8bba SGT-003 \u7684\u6f5c\u5728\u52a0\u901f\u5ba1\u6279\u9014\u5f84"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Solid Biosciences \u662f\u4e00\u5bb6\u5f00\u53d1\u7528\u4e8e\u6cbb\u7597\u795e\u7ecf\u808c\u8089\u548c\u5fc3\u810f\u75be\u75c5\u7684\u7cbe\u51c6\u4e0b\u4e00\u4ee3\u57fa\u56e0\u836f\u7269\u7684\u751f\u547d\u79d1\u5b66\u516c\u53f8\uff0c\u8be5\u516c\u53f8\u516c\u5e03\u4e86\u622a\u81f3 2024 \u5e74 12 \u6708 31 \u65e5\u7684\u7b2c\u56db\u5b63\u5ea6\u548c\u5168\u5e74\u8d22\u52a1\u4e1a\u7ee9\uff0c\u5e76\u63d0\u4f9b\u4e86\u4e1a\u52a1\u66f4\u65b0\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Solid \u603b\u88c1\u517c\u9996\u5e2d\u6267\u884c\u5b98 Bo Cumbo \u8bc4\u8bba\u9053\uff1a\u201c\u8fc7\u53bb 18 \u4e2a\u6708\u6765\uff0c\u6211\u4eec\u4e00\u76f4\u8ba4\u771f\u6267\u884c\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u548c FA \u5f00\u53d1\u8ba1\u5212\uff0c\u8fd9\u8ba9 Solid \u53d1\u751f\u4e86\u7ffb\u5929\u8986\u5730\u7684\u53d8\u5316\uff0c\u5e76\u4e3a\u6211\u4eec\u8fc8\u5411 2025 \u5e74\u53ca\u4ee5\u540e\u7684\u53d1\u5c55\u63d0\u4f9b\u4e86\u5de8\u5927\u7684\u52a8\u529b\u3002\u6211\u4eec\u4e0b\u4e00\u4ee3\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (Duchenne) \u9879\u76ee SGT-003 \u7684 1\/2 \u671f INSPIRE DUCHENNE \u8bd5\u9a8c\u7684\u524d\u4e09\u540d\u53c2\u4e0e\u8005\u7684\u521d\u59cb 90 \u5929\u6570\u636e\u867d\u7136\u8fd8\u5904\u4e8e\u65e9\u671f\u9636\u6bb5\uff0c\u4f46\u5df2\u663e\u793a\u51fa\u4ee4\u4eba\u653e\u5fc3\u7684\u5b89\u5168\u4f53\u9a8c\uff0c\u5e76\u4e14\u808c\u8089\u5b8c\u6574\u6027\u548c\u5065\u5eb7\u7684\u751f\u7269\u6807\u5fd7\u7269\u6709\u5e0c\u671b\u6539\u5584\u3002\u6211\u4eec\u76f8\u4fe1\u8fd9\u4e9b\u65e9\u671f\u6307\u6807\u652f\u6301 SGT-003 \u6210\u4e3a\u6cbb\u7597\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684\u4e00\u6d41\u57fa\u56e0\u6cbb\u7597\u5019\u9009\u836f\u7269\u7684\u6f5c\u529b\u3002\u6211\u4eec\u7406\u89e3\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u60a3\u8005\u7fa4\u4f53\u5bf9\u8fd9\u4e9b\u6570\u636e\u7684\u5174\u594b\uff0c\u6211\u4eec\u81f4\u529b\u4e8e\u6709\u76ee\u7684\u6027\u548c\u7d27\u8feb\u6027\u5730\u91c7\u53d6\u884c\u52a8\uff0c\u5c3d\u5feb\u5c06 SGT-003 \u63a8\u5411\u4e34\u5e8a\u3002\u201d(<a href=\"https:\/\/dmdwarrior.com\/zh\/solid-biosciences-reports-positive-initial-clinical-data-from-next-generation-duchenne-gene-therapy-candidate-sgt-003\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u9605\u8bfb\u66f4\u591a<\/a>)<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">SGT-003 \u7684\u521d\u6b65\u4e34\u5e8a\u6570\u636e\u79ef\u6781<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u89c2\u5bdf\u5230\u6bcf\u4e2a\u7ec6\u80de\u6838\u7684\u9ad8\u8f7d\u4f53\u57fa\u56e0\u7ec4\u62f7\u8d1d\u6570\u3001\u5f3a\u52b2\u7684\u5fae\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u8868\u8fbe\u4ee5\u53ca\u808c\u8089\u5b8c\u6574\u6027\u5176\u4ed6\u6307\u6807\u7684\u65e9\u671f\u6539\u5584\uff0c\u5305\u62ec\uff1a<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u6bcf\u4e2a\u7ec6\u80de\u6838\u7684\u5e73\u5747\u8f7d\u4f53\u62f7\u8d1d\u6570\uff1a18.7 (N=3)\uff0c<\/li>\n\n\n\n<li>\u5e73\u5747\u5fae\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u8868\u8fbe\uff1a110%\uff08N=3\uff09\uff0c\u901a\u8fc7\u86cb\u767d\u8d28\u5370\u8ff9\u6cd5\u6d4b\u91cf\uff0c<\/li>\n\n\n\n<li>\u5e73\u5747\u5fae\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u8868\u8fbe\uff1a108%\uff08N=3\uff09\uff0c\u7ecf\u8d28\u8c31\u6d4b\u91cf\uff0c<\/li>\n\n\n\n<li>\u6297\u808c\u840e\u7f29\u86cb\u767d\u9633\u6027\u7ea4\u7ef4\u5e73\u5747\u767e\u5206\u6bd4\uff1a78%\uff08N=3\uff09\uff0c\u901a\u8fc7\u514d\u75ab\u8367\u5149\u6d4b\u91cf\uff0c<\/li>\n\n\n\n<li>\u5e73\u5747 \u03b2 \u808c\u805a\u7cd6\u9633\u6027\u7ea4\u7ef4\u767e\u5206\u6bd4\uff1a70% (N=3)\uff0c<\/li>\n\n\n\n<li>\u5e73\u5747 nNOS\uff08\u795e\u7ecf\u5143\u4e00\u6c27\u5316\u6c2e\u5408\u9176\uff09\u9633\u6027\u7ea4\u7ef4\u767e\u5206\u6bd4\uff1a42%\uff08N=3\uff09\uff0c<\/li>\n\n\n\n<li>\u53e6\u5916 7 \u79cd\u808c\u8089\u5b8c\u6574\u6027\u751f\u7269\u6807\u5fd7\u7269\u5f97\u5230\u6539\u5584\uff08N=3\uff09\uff0c\u5e76\u4e14<\/li>\n\n\n\n<li>\u7b2c 180 \u5929\u5de6\u5fc3\u5ba4\u5c04\u8840\u5206\u6570 (LVEF) \u8f83\u57fa\u7ebf\u6709 8% \u7684\u65e9\u671f\u5e73\u5747\u6539\u5584 (N=2)\u3002<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">\u4e86\u89e3\u66f4\u591a\uff1a <a href=\"https:\/\/dmdwarrior.com\/zh\/faq-solid-biosciences-sgt-003-gene-therapy\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u5173\u4e8e Solid Biosciences SGT-003 \u57fa\u56e0\u6cbb\u7597\u7684\u5e38\u89c1\u95ee\u9898<\/a><\/p>\n\n\n\n<h2 class=\"wp-block-heading\">\u4e0e FDA \u4e3e\u884c\u4f1a\u8bae\uff0c\u8ba8\u8bba SGT-003 \u7684\u6f5c\u5728\u52a0\u901f\u5ba1\u6279\u9014\u5f84<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u622a\u81f3 2025 \u5e74 2 \u6708 11 \u65e5\u6570\u636e\u622a\u6b62\u65e5\u671f\uff0c6 \u540d\u63a5\u53d7\u6cbb\u7597\u7684\u53c2\u4e0e\u8005\u5bf9 SGT-003 \u8010\u53d7\u6027\u826f\u597d\uff0c\u672a\u89c2\u5bdf\u5230\u4e25\u91cd\u4e0d\u826f\u4e8b\u4ef6 (SAE)\u3001\u7591\u4f3c\u610f\u5916\u4e25\u91cd\u4e0d\u826f\u53cd\u5e94 (SUSAR) \u6216\u6025\u6027\u809d\u635f\u4f24 AE\u3002<\/li>\n\n\n\n<li>\u8bd5\u9a8c\u62db\u52df\u5de5\u4f5c\u6b63\u5728\u8fdb\u884c\u4e2d\uff0c\u516c\u53f8\u9884\u8ba1\u5230 2025 \u5e74\u7b2c\u4e8c\u5b63\u5ea6\u521d\u5c06\u62db\u52df\u8d85\u8fc7 10 \u540d\u53c2\u4e0e\u8005\uff0c\u5230 2025 \u5e74\u7b2c\u56db\u5b63\u5ea6\u5c06\u62db\u52df\u7ea6 20 \u540d\u53c2\u4e0e\u8005\u3002<\/li>\n\n\n\n<li><strong>2025 \u5e74\u4e2d\uff0c\u516c\u53f8\u8ba1\u5212\u8bf7\u6c42\u4e0e FDA \u4e3e\u884c\u4f1a\u8bae\uff0c\u8ba8\u8bba SGT-003 \u7684\u6f5c\u5728\u52a0\u901f\u5ba1\u6279\u9014\u5f84\u3002<\/strong><\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u4e86\u89e3\u66f4\u591a\uff1a<\/strong>&nbsp;<a href=\"https:\/\/dmdwarrior.com\/zh\/new-gene-therapies-for-duchenne-muscular-dystrophy\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684\u6f5c\u5728\u65b0\u57fa\u56e0\u7597\u6cd5<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>Solid Biosciences, a life sciences company developing precision, next generation, genetic medicines for neuromuscular and cardiac diseases, reported financial results for the fourth quarter and full year ended December 31, 2024, and provided a business update. Bo Cumbo, President and CEO of Solid, commented: &#8220;The diligent execution of our Duchenne and FA development programs over [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":3090,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[88,61,110,170,109],"class_list":["post-3088","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-fda","tag-gene-therapy","tag-sgt-003","tag-sgt-003-gene-therapy","tag-solid-biosciences"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/3088","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=3088"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/3088\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media\/3090"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media?parent=3088"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/categories?post=3088"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/tags?post=3088"}],"curies":[{"name":"\u53ef\u6e7f\u6027\u7c89\u5242","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}