{"id":5101,"date":"2025-11-04T16:49:31","date_gmt":"2025-11-04T13:49:31","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=5101"},"modified":"2025-11-04T17:01:51","modified_gmt":"2025-11-04T14:01:51","slug":"solid-biosciences-provides-update-on-inspire-duchenne-clinical-trial-progress-and-planned-regulatory-discussions","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/zh\/solid-biosciences-provides-update-on-inspire-duchenne-clinical-trial-progress-and-planned-regulatory-discussions\/","title":{"rendered":"Solid Biosciences \u63d0\u4f9b\u5173\u4e8e INSPIRE DUCHENN \u4e34\u5e8a\u8bd5\u9a8c\u8fdb\u5c55\u548c\u8ba1\u5212\u4e2d\u7684\u76d1\u7ba1\u8ba8\u8bba\u7684\u6700\u65b0\u4fe1\u606f"},"content":{"rendered":"<p class=\"wp-block-paragraph\"><strong>Solid Biosciences \u516c\u5e03\u4e86 1\/2 \u671f INSPIRE DUCHENNE \u4e34\u5e8a\u8bd5\u9a8c\u7684\u79ef\u6781\u65b0\u4e2d\u671f\u6570\u636e\u3002.<\/strong> \u516c\u53f8\u8fd8\u66f4\u65b0\u4e86\u4e0e\u7f8e\u56fd\u98df\u54c1\u836f\u54c1\u76d1\u7763\u7ba1\u7406\u5c40 (FDA) \u4f1a\u9762\u7684\u8ba1\u5212\u65f6\u95f4\uff0c\u8ba8\u8bba SGT-003 \u7684\u6f5c\u5728\u6ce8\u518c\u9014\u5f84\uff0c\u5305\u62ec\u52a0\u901f\u5ba1\u6279\u9014\u5f84\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Solid Biosciences\u516c\u53f8\u603b\u88c1\u517c\u9996\u5e2d\u6267\u884c\u5b98Bo Cumbo\u8bc4\u8bba\u9053\uff1a\u201c\u4eca\u5929\u516c\u5e03\u7684INSPIRE DUCHENN\u4e2d\u671f\u6570\u636e\u589e\u5f3a\u4e86\u6211\u4eec\u5bf9SGT-003\u6cbb\u7597\u6f5c\u529b\u7684\u4fe1\u5fc3\u3002\u4ece\u89c2\u5bdf\u5230\u7684SGT-003\u5fae\u578b\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u8868\u8fbe\u6c34\u5e73\u4e0e\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u76f8\u5173\u86cb\u767d\u590d\u5408\u7269\u5173\u952e\u6210\u5206\u7684\u6b63\u786e\u5b9a\u4f4d\u6062\u590d\u4e4b\u95f4\u5b58\u5728\u663e\u8457\u7684\u751f\u7269\u5b66\u76f8\u5173\u6027\uff0c\u5230\u5fc3\u810f\u529f\u80fd\u6b63\u5e38\u5316\u7684\u65e9\u671f\u8bc1\u636e\uff0c\u6211\u4eec\u89c2\u5bdf\u5230\u4e86\u4eba\u4f53\u5185\u6e05\u6670\u4e14\u7ea7\u8054\u7684\u6548\u5e94\uff0c\u8868\u660e\u6cbb\u7597\u4f1a\u4ea7\u751f\u534f\u8c03\u7684\u5168\u8eab\u6027\u53cd\u5e94\u3002\u6211\u4eec\u76f8\u4fe1\uff0c\u8fd9\u4e9b\u4e2d\u671f\u6570\u636e\u4ee3\u8868\u4e86\u8fc4\u4eca\u4e3a\u6b62\u5bf9\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u57fa\u56e0\u7597\u6cd5\u6700\u5168\u9762\u7684\u65e9\u671f\u5206\u6790\u4e4b\u4e00\u3002\u8fd9\u4e9b\u4e2d\u671f\u7ed3\u679c\u7684\u8d28\u91cf\u548c\u4e00\u81f4\u6027\u8fdb\u4e00\u6b65\u5de9\u56fa\u4e86\u6211\u4eec\u5bf9SGT-003\u5c06\u5206\u5b50\u6c34\u5e73\u7684\u7597\u6548\u8f6c\u5316\u4e3a\u6709\u610f\u4e49\u7684\u4e34\u5e8a\u7ed3\u679c\u7684\u6f5c\u529b\u7684\u4fe1\u5ff5\u3002\u2019<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Cumbo\u5148\u751f\u7ee7\u7eed\u8bf4\u9053\uff1a\u201c\u9274\u4e8e\u76d1\u7ba1\u73af\u5883\u7684\u4e0d\u65ad\u53d8\u5316\u4ee5\u53caINSPIRE DUCHENNE\u8bd5\u9a8c\u5feb\u901f\u7684\u5165\u7ec4\u8fdb\u7a0b\uff0c\u6211\u4eec\u4e3b\u52a8\u51b3\u5b9a\u5c06\u539f\u5b9a\u4e0eFDA\u7684\u4f1a\u9762\u63a8\u8fdf\u81f32026\u5e74\u4e0a\u534a\u5e74\u3002\u989d\u5916\u7684\u65f6\u95f4\u5c06\u4f7f\u6211\u4eec\u80fd\u591f\uff1a1\uff09\u751f\u6210\u66f4\u5b8c\u6574\u7684\u6570\u636e\u96c6\uff0c\u4ee5\u4fbf\u4e0eFDA\u8fdb\u884c\u8ba8\u8bba\uff1b2\uff09\u57fa\u4e8e\u9ad8\u8d28\u91cf\u3001\u5339\u914d\u826f\u597d\u7684\u81ea\u7136\u53f2\u6570\u636e\uff0c\u6784\u5efa\u5168\u9762\u7684\u5916\u90e8\u5bf9\u7167\uff1b3\uff09\u4e0e\u6211\u4eec\u7684CDMO\u5408\u4f5c\u4f19\u4f34\u542f\u52a8\u5de5\u827a\u6027\u80fd\u9a8c\u8bc1\uff08PPQ\uff09\u751f\u4ea7\uff0c\u4e3a\u6f5c\u5728\u7684\u751f\u7269\u5236\u54c1\u8bb8\u53ef\u7533\u8bf7\uff08BLA\uff09\u63d0\u4ea4\u505a\u51c6\u5907\uff1b\u4ee5\u53ca4\uff09\u542f\u52a8IMPACT DUCHENNE\u8bd5\u9a8c\u7684\u7ed9\u836f\uff0c\u8fd9\u662f\u4e00\u9879\u5c06\u5728\u7f8e\u56fd\u5883\u5916\u8fdb\u884c\u76843\u671f\u968f\u673a\u3001\u53cc\u76f2\u3001\u5b89\u6170\u5242\u5bf9\u7167\u4e34\u5e8a\u8bd5\u9a8c\u3002\u6211\u4eec\u76f8\u4fe1\uff0c\u8fd9\u4e9b\u4e3e\u63aa\u5c06\u4f7fSolid\u516c\u53f8\u80fd\u591f\u5411\u76d1\u7ba1\u673a\u6784\u63d0\u4ea4\u6700\u5177\u8bf4\u670d\u529b\u7684\u65b9\u6848\u3002\u201d.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u201c\u6211\u4eec\u73b0\u5728\u7684\u9996\u8981\u4efb\u52a1\u662f\u8fc5\u901f\u6784\u5efa\u4e00\u4e2a\u5f3a\u5927\u7684\u6570\u636e\u96c6\uff0c\u4ee5\u652f\u6301SGT-003\u6f5c\u5728\u7684\u52a0\u901f\u76d1\u7ba1\u5ba1\u6279\u9014\u5f84\uff0c\u6211\u4eec\u81f4\u529b\u4e8e\u4ee5\u7d27\u8feb\u7684\u6b65\u4f10\u5c3d\u5feb\u5b9e\u73b0SGT-003\u7684\u6f5c\u529b\u3002\u9664\u4e86SGT-003\u4e4b\u5916\uff0c\u6211\u4eec\u4e3b\u8981\u7684\u5728\u7814\u9879\u76ee\u4e5f\u5728\u6301\u7eed\u63a8\u8fdb\u3002\u2019\u574e\u535a\u5148\u751f\u603b\u7ed3\u9053\u3002.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">SGT-003 \u2013 INSPIRE DUCHENN \u4e34\u5e8a\u8bd5\u9a8c<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u622a\u81f32025\u5e7410\u670831\u65e5\uff0cINSPIRE DUCHENNE\u8bd5\u9a8c\u5df2\u670923\u540d\u53d7\u8bd5\u8005\u63a5\u53d7\u4e86\u7ed9\u836f\uff1bSolid\u516c\u53f8\u9884\u8ba1\u52302026\u5e74\u521d\u5c06\u603b\u5171\u4e3a30\u540d\u53d7\u8bd5\u8005\u7ed9\u836f\uff0c\u7136\u540e\u8ba1\u5212\u57282026\u5e74\u4e0a\u534a\u5e74\u4e0eFDA\u516c\u53f8\u4f1a\u9762\uff0c\u8ba8\u8bba\u6f5c\u5728\u7684\u6ce8\u518c\u9014\u5f84\u3002<\/li>\n\n\n\n<li>\u4f7f\u7528\u4ec5\u542b\u7c7b\u56fa\u9187\u7684\u9884\u9632\u6027\u514d\u75ab\u8c03\u8282\u65b9\u6848\uff0cSGT-003 \u7684\u8010\u53d7\u6027\u603b\u4f53\u826f\u597d\uff1b\u5fc3\u810f\u5b89\u5168\u6027\u76d1\u6d4b\u6301\u7eed\u663e\u793a\u5fc3\u810f\u635f\u4f24\u51cf\u5c11\uff0c\u5e76\u51fa\u73b0\u5fc3\u810f\u529f\u80fd\u6b63\u5e38\u5316\u7684\u65e9\u671f\u8ff9\u8c61\u2014\u2014<\/li>\n\n\n\n<li>\u6765\u81ea 10 \u540d\u63a5\u53d7\u6cbb\u7597\u7684\u53c2\u4e0e\u8005\uff08\u5e74\u9f84 5-10 \u5c81\uff09\u7684\u7b2c 90 \u5929\u6d3b\u68c0\u6570\u636e\u663e\u793a\uff0c\u6240\u6709\u53c2\u4e0e\u8005\u5747\u5bf9\u6cbb\u7597\u6709\u53cd\u5e94\uff0c\u901a\u8fc7\u86cb\u767d\u8d28\u5370\u8ff9\u6cd5\u68c0\u6d4b\u7684\u5e73\u5747\u5fae\u578b\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u8868\u8fbe\u4e3a 58%\uff0c\u901a\u8fc7\u8d28\u8c31\u6cd5\u68c0\u6d4b\u7684\u5e73\u5747\u5fae\u578b\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u9633\u6027\u7ea4\u7ef4\u4e3a 58%\uff0c\u901a\u8fc7\u514d\u75ab\u8367\u5149\u6cd5\u68c0\u6d4b\u7684\u5e73\u5747\u5fae\u578b\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u9633\u6027\u7ea4\u7ef4\u4e3a 51%\uff0c\u901a\u8fc7\u514d\u75ab\u8367\u5149\u6cd5\u68c0\u6d4b\u7684\u5e73\u5747 \u03b2-\u808c\u805a\u7cd6\u9633\u6027\u7ea4\u7ef4\u4e3a 50% \u2013<\/li>\n\n\n\n<li>\u5728\u7b2c 90 \u5929\u89c2\u5bdf\u5230\u7684\u5fae\u578b\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u8868\u8fbe\u6c34\u5e73\u4e0e\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u76f8\u5173\u86cb\u767d\u590d\u5408\u7269 (DAPC) \u7684\u5173\u952e\u6210\u5206 \u03b2-\u808c\u805a\u7cd6 (r = 0.95) \u548c nNOS (r = 0.95) \u4e4b\u95f4\u5b58\u5728\u5f88\u5f3a\u7684\u7edf\u8ba1\u76f8\u5173\u6027\uff0c\u8fd9\u8868\u660e SGT-003 \u4ecb\u5bfc\u4e86 DAPC \u7684\u6062\u590d\uff0c\u5e76\u4e14\u901a\u8fc7\u964d\u4f4e CK \u6c34\u5e73 (r = -0.78) \u4e5f\u4ea7\u751f\u4e86\u808c\u8089\u4fdd\u62a4\u7684\u76f8\u5e94\u4fe1\u53f7\u3002<\/li>\n\n\n\n<li>Solid\u516c\u53f8\u5df2\u542f\u52a8\u9996\u4e2a\u4e34\u5e8a\u8bd5\u9a8c\u4e2d\u5fc3\uff0c\u76ee\u524d\u6b63\u5728\u4e3aIMPACT DUCHENNE\u8bd5\u9a8c\u7b5b\u9009\u53d7\u8bd5\u8005\u3002IMPACT DUCHENNE\u662f\u4e00\u9879\u5728\u7f8e\u56fd\u4ee5\u5916\u5730\u533a\u5f00\u5c55\u7684SGT-003\u76843\u671f\u968f\u673a\u3001\u53cc\u76f2\u3001\u5b89\u6170\u5242\u5bf9\u7167\u4e34\u5e8a\u8bd5\u9a8c\u3002<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">\u5728\u7b2c 90 \u5929\u548c\u7b2c 360 \u5929\uff0c\u5747\u89c2\u5bdf\u5230\u4e00\u7cfb\u5217\u808c\u8089\u635f\u4f24\u548c\u5206\u89e3\u751f\u7269\u6807\u5fd7\u7269\u51fa\u73b0\u6709\u5229\u7684\u4e0b\u964d\uff1a<\/p>\n\n\n\n<figure class=\"wp-block-table\"><table class=\"has-fixed-layout\"><tbody><tr><td><strong>\u8840\u6e05\u751f\u7269\u6807\u5fd7\u7269<\/strong><\/td><td class=\"has-text-align-center\" data-align=\"center\"><strong>\u7b2c 90 \u5929\u5e73\u5747\u51cf\u5c11\u91cf<\/strong><br><strong>\uff08\u9664\u975e\u53e6\u6709\u8bf4\u660e\uff0c\u5426\u5219 N=14\uff09<\/strong><\/td><td class=\"has-text-align-center\" data-align=\"center\"><strong>\u7b2c 360 \u5929\u5e73\u5747\u51cf\u5c11\u91cf<\/strong><br><strong>\uff08\u9664\u975e\u53e6\u6709\u8bf4\u660e\uff0c\u5426\u5219 N=3\uff09<\/strong><\/td><\/tr><tr><td>\u8840\u6e05\u808c\u9178\u6fc0\u9176\uff08CK\uff09<\/td><td class=\"has-text-align-center\" data-align=\"center\">34%<\/td><td class=\"has-text-align-center\" data-align=\"center\">42%<\/td><\/tr><tr><td>\u8840\u6e05\u4e19\u6c28\u9178\u8f6c\u6c28\u9176\uff08ALT\uff09<\/td><td class=\"has-text-align-center\" data-align=\"center\">41%<\/td><td class=\"has-text-align-center\" data-align=\"center\">29%<\/td><\/tr><tr><td>\u8840\u6e05\u5929\u51ac\u6c28\u9178\u6c28\u57fa\u8f6c\u79fb\u9176\uff08AST\uff09<\/td><td class=\"has-text-align-center\" data-align=\"center\">25%<\/td><td class=\"has-text-align-center\" data-align=\"center\">40%<\/td><\/tr><tr><td>\u8840\u6e05\u4e73\u9178\u8131\u6c22\u9176\uff08LDH\uff09*<\/td><td class=\"has-text-align-center\" data-align=\"center\">42%<\/td><td class=\"has-text-align-center\" data-align=\"center\">46%<\/td><\/tr><tr><td>\u8840\u6e05\u808c\u8054\u86cb\u767d**<\/td><td class=\"has-text-align-center\" data-align=\"center\">22%<\/td><td class=\"has-text-align-center\" data-align=\"center\">25%<\/td><\/tr><tr><td colspan=\"3\"><em>*\u7b2c 90 \u5929\u6709 12 \u4e2a\u53c2\u4e0e\u8005\u6837\u672c\u53ef\u7528\u4e8e\u68c0\u6d4b LDH\uff08\u5176\u4e2d\u4e24\u4e2a\u6837\u672c\u6eb6\u8840\uff09\uff1b**\u7b2c 90 \u5929\u6709 11 \u4e2a\u53c2\u4e0e\u8005\u6837\u672c\u53ef\u7528\u4e8e\u68c0\u6d4b\u808c\u8054\u86cb\u767d\uff0c\u7b2c 360 \u5929\u6709 2 \u4e2a\u6837\u672c\u53ef\u7528\u4e8e\u68c0\u6d4b\u808c\u8054\u86cb\u767d\uff0c\u8be5\u808c\u8054\u86cb\u767d\u5728\u8f83\u65e9\u7684\u622a\u6b62\u65e5\u671f\u8fdb\u884c\u4e86\u6279\u91cf\u5206\u6790\u3002.<\/em><\/td><\/tr><\/tbody><\/table><\/figure>\n\n\n\n<h2 class=\"wp-block-heading\">\u675c\u7533\u51b2\u51fb\u9879\u76ee\u7b2c\u4e09\u9636\u6bb5<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">2025\u5e7410\u6708\uff0cSolid\u516c\u53f8\u542f\u52a8\u4e86\u9996\u4e2a\u4e34\u5e8a\u8bd5\u9a8c\u4e2d\u5fc3\uff0c\u5e76\u5f00\u59cb\u5bf9IMPACT DUCHENNE\u8bd5\u9a8c\u8fdb\u884c\u53d7\u8bd5\u8005\u7b5b\u9009\u3002IMPACT DUCHENNE\u662f\u4e00\u9879\u8bc4\u4f30SGT-003\u76843\u671f\u968f\u673a\u3001\u53cc\u76f2\u3001\u5b89\u6170\u5242\u5bf9\u7167\u4e34\u5e8a\u8bd5\u9a8c\u3002\u8be5\u8bd5\u9a8c\u5c06\u5728\u7f8e\u56fd\u4ee5\u5916\u7684\u513f\u79d1\u53d7\u8bd5\u8005\u4e2d\u5f00\u5c55\uff0c\u65e8\u5728\u652f\u6301\u6f5c\u5728\u7684\u7f8e\u56fd\u4ee5\u5916\u5730\u533a\u7684\u76d1\u7ba1\u5ba1\u6279\u3002\u6211\u4eec\u5df2\u83b7\u5f97\u5728\u52a0\u62ff\u5927\u548c\u6fb3\u5927\u5229\u4e9a\u5f00\u5c55IMPACT DUCHENNE\u8bd5\u9a8c\u7684\u76d1\u7ba1\u6279\u51c6\uff0c\u5e76\u8ba1\u5212\u5728\u83b7\u5f97\u76d1\u7ba1\u6279\u51c6\u540e\uff0c\u5c06\u8bd5\u9a8c\u6269\u5c55\u5230\u5176\u4ed6\u56fd\u5bb6\u3002. <a href=\"https:\/\/dmdwarrior.com\/zh\/solid-biosciences-sgt-003-gene-therapy-phase-3-clinical-trial-nct07160634\/\" target=\"_blank\" rel=\"noreferrer noopener\">(IMPACT DUCHENNE)<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u5173\u6ce8\u6b64\u9875\u9762<\/strong>&nbsp;&gt;&gt;&gt;&nbsp;<a href=\"https:\/\/dmdwarrior.com\/zh\/dmd-therapies\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u6240\u6709\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684\u4e34\u5e8a\u8bd5\u9a8c<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>Solid Biosciences announced positive new interim data from the Phase 1\/2 INSPIRE DUCHENNE clinical trial. The Company also provided an update to the planned timing of its meeting with the U.S. Food and Drug Administration (FDA) to discuss potential registrational pathways, including accelerated approval pathways, for SGT-003. Bo Cumbo, President and CEO of Solid Biosciences [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":5102,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[61,110,170,109],"class_list":["post-5101","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","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\/5101","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=5101"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/5101\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media\/5102"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media?parent=5101"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/categories?post=5101"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/tags?post=5101"}],"curies":[{"name":"\u53ef\u6e7f\u6027\u7c89\u5242","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}