{"id":8916,"date":"2026-11-05T16:37:41","date_gmt":"2026-11-05T13:37:41","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=8916"},"modified":"2026-11-05T16:40:46","modified_gmt":"2026-11-05T13:40:46","slug":"pbgene-dmd-no-serious-adverse-events-week-8","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/zh\/pbgene-dmd-no-serious-adverse-events-week-8\/","title":{"rendered":"Precision BioSciences\u516c\u53f8\u62a5\u544aPBGENE-DMD\u5b89\u5168\u6027\u8bc4\u4f30\u7ed3\u679c\u79ef\u6781\uff1a\u7b2c\u516b\u5468\u672a\u53d1\u751f\u4e25\u91cd\u4e0d\u826f\u4e8b\u4ef6"},"content":{"rendered":"<p class=\"wp-block-paragraph\"><strong>Precision BioSciences\u5ba3\u5e03\u5bf9\u5176\u7528\u4e8e\u6cbb\u7597\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD) \u7684\u5728\u7814\u57fa\u56e0\u7f16\u8f91\u7597\u6cd5PBGENE-DMD\u8fdb\u884c\u4e86\u79ef\u6781\u7684\u5b89\u5168\u6027\u5ba1\u67e5\u3002<\/strong> \u4e00\u4e2a\u72ec\u7acb\u7684\u6570\u636e\u5b89\u5168\u76d1\u6d4b\u59d4\u5458\u4f1a (DSMB) \u5ba1\u67e5\u4e86 1\/2 \u671fFUNCTION-DMD\u4e34\u5e8a\u8bd5\u9a8c\u4e2d\u9996\u4f8b\u63a5\u53d7\u6cbb\u7597\u7684\u60a3\u8005\u7684\u5b89\u5168\u6570\u636e\uff0c\u5e76\u62a5\u544a\u79f0\u5728\u6cbb\u7597\u540e\u7684\u524d\u516b\u5468\u5185\u6ca1\u6709\u53d1\u751f\u4e25\u91cd\u4e0d\u826f\u4e8b\u4ef6 (SAE) \u6216\u7279\u522b\u5173\u6ce8\u7684\u4e0d\u826f\u4e8b\u4ef6 (AESI)\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u5728\u5b8c\u6210\u60a3\u8005\u6700\u521d\u4e3a\u671f\u516b\u5468\u7684\u5b89\u5168\u89c2\u5bdf\u671f\u540e\uff0c\u6570\u636e\u5b89\u5168\u76d1\u5bdf\u59d4\u5458\u4f1a (DSMB) \u672a\u53d1\u73b0\u4efb\u4f55\u5b89\u5168\u9690\u60a3\uff0c\u5e76\u6279\u51c6\u7ee7\u7eed\u8fdb\u884c\u7b2c\u4e00\u90e8\u5206\u7814\u7a76\u4e2d\u5176\u4ed6\u60a3\u8005\u7684\u6cbb\u7597\u3002\u8fd9\u4e00\u51b3\u5b9a\u662f\u6b63\u5728\u8fdb\u884c\u7684\u4e34\u5e8a\u8bd5\u9a8c\u7684\u4e00\u4e2a\u91cd\u8981\u91cc\u7a0b\u7891\uff0c\u5e76\u652f\u6301\u7ee7\u7eed\u62db\u52df\u60a3\u8005\u3002\u4e86\u89e3\u66f4\u591a\uff1a <a href=\"https:\/\/dmdwarrior.com\/zh\/faq-pbgene-dmd-gene-editing-therapy\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u4ec0\u4e48\u662f PBGENE-DMD\uff1f<\/a><\/p>\n\n\n\n<h3 class=\"wp-block-heading\">PBGENE-DMD \uff1a\u4e00\u79cd\u7528\u4e8e\u6cbb\u7597\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684\u57fa\u56e0\u7f16\u8f91\u65b9\u6cd5<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">PBGENE-DMD\u7597\u6cd5\u65e8\u5728\u5229\u7528\u4eba\u4f53\u81ea\u8eab\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u57fa\u56e0\u6765\u89e3\u51b3\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\uff08DMD\uff09\u7684\u6839\u672c\u9057\u4f20\u75c5\u56e0\u3002\u8be5\u7597\u6cd5\u91c7\u7528\u4e24\u79cd\u4e92\u8865\u7684ARCUS\u6838\u9178\u9176\uff0c\u901a\u8fc7\u5355\u4e00\u817a\u76f8\u5173\u75c5\u6bd2\uff08AAV\uff09\u9012\u9001\uff0c\u4ee5\u5207\u9664\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u57fa\u56e0\u7684\u7b2c45-55\u53f7\u5916\u663e\u5b50\u3002\u5176\u76ee\u6807\u662f\u6062\u590d\u8fd1\u4e4e\u5168\u957f\u7684\u529f\u80fd\u6027\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u4ea7\u751f\uff0c\u800c\u4e0d\u662f\u4f9d\u8d56\u5408\u6210\u7684\u3001\u7f29\u77ed\u7684\u5fae\u578b\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u3002<\/p>\n\n\n\n<h3 class=\"wp-block-heading\">\u5173\u4e8eFUNCTION-DMD\u4e34\u5e8a\u8bd5\u9a8c<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">FUNCTION-DMD 1\/2 \u671f\u7814\u7a76\u65e8\u5728\u8bc4\u4f30PBGENE-DMD\u5728 2 \u81f3 7 \u5c81\u60a3\u6709 DMD \u4e14\u80fd\u72ec\u7acb\u884c\u8d70\u7684\u7537\u5b69\u4e2d\u7684\u5b89\u5168\u6027\u3001\u8010\u53d7\u6027\u548c\u6709\u6548\u6027\uff0c\u8fd9\u4e9b\u7537\u5b69\u7684\u57fa\u56e0\u7a81\u53d8\u6d89\u53ca\u5916\u663e\u5b50 45-55\u3002\u8be5\u7814\u7a76\u5c06\u8bc4\u4f30\u53c2\u4e0e\u8005\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u8868\u8fbe\u548c\u529f\u80fd\u7ed3\u679c\u3002 <strong>\u4e86\u89e3\u66f4\u591a\uff1a<\/strong> <a href=\"https:\/\/dmdwarrior.com\/clinical-trials\/pbgene-dmd-phase-1-2a-clinical-trial-for-duchenne-function-dmd\/\" target=\"_blank\" rel=\"noreferrer noopener\">PBGENE-DMD 1\/2a\u671f\u4e34\u5e8a\u8bd5\u9a8c<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Precision BioSciences\u516c\u53f8\u8868\u793a\uff0c\u6700\u521d\u7684\u516b\u5468\u76d1\u6d4b\u671f\u5bf9\u4e8e\u8bc6\u522b\u4e0eAAV\u7597\u6cd5\u76f8\u5173\u7684\u6f5c\u5728\u65e9\u671f\u5b89\u5168\u4e8b\u4ef6\u81f3\u5173\u91cd\u8981\u3002\u968f\u7740\u7814\u7a76\u7684\u8fdb\u884c\uff0c\u8be5\u516c\u53f8\u5c06\u7ee7\u7eed\u8fdb\u884c\u5b89\u5168\u76d1\u6d4b\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u8be5\u516c\u53f8\u9884\u8ba1\u5c06\u5728 2027 \u5e74\u5168\u5e74\u63d0\u4f9bFUNCTION-DMD\u7814\u7a76\u7684\u8fdb\u4e00\u6b65\u5b89\u5168\u6027\u548c\u6709\u6548\u6027\u66f4\u65b0\u4fe1\u606f\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u6700\u65b0\u516c\u5e03\u7684\u6570\u636e\u63d0\u4f9b\u4e86\u4e00\u4e2a\u4ee4\u4eba\u9f13\u821e\u7684\u521d\u6b65\u5b89\u5168\u6027\u4fe1\u53f7\uff1a\u5728\u9996\u4f8b\u60a3\u8005\u4e2d\uff0c\u622a\u81f3\u7b2c\u516b\u5468\u672a\u62a5\u544a\u4efb\u4f55\u4e25\u91cd\u4e0d\u826f\u4e8b\u4ef6\u6216\u7279\u522b\u503c\u5f97\u5173\u6ce8\u7684\u4e0d\u826f\u4e8b\u4ef6\u3002\u7136\u800c\uff0c\u8fd9\u4e9b\u65e9\u671f\u7ed3\u679c\u5e76\u4e0d\u80fd\u786e\u5b9a\u8be5\u7597\u6cd5\u7684\u603b\u4f53\u5b89\u5168\u6027\u548c\u6709\u6548\u6027\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u4e86\u89e3\u66f4\u591a<\/strong>:&nbsp;<a href=\"https:\/\/dmdwarrior.com\/clinical-trials\/map\/\" target=\"_blank\" rel=\"noopener\">\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u4e34\u5e8a\u8bd5\u9a8c\u5730\u70b9\u5730\u56fe<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>Precision BioSciences has announced a positive safety review of PBGENE-DMD, its investigational gene-editing treatment for Duchenne muscular dystrophy (DMD). An independent Data Safety Monitoring Board (DSMB) reviewed safety data from the first patient treated in the Phase 1\/2 FUNCTION-DMD clinical trial and reported no serious adverse events (SAEs) or adverse events of special interest (AESIs) [&hellip;]<\/p>\n","protected":false},"author":4,"featured_media":8918,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[479,314,724,313],"class_list":["post-8916","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-function-dmd","tag-pbgene-dmd","tag-pbgene-dmd-therapy","tag-precision-biosciences"],"subtitle":"Precision BioSciences reports an encouraging safety milestone for PBGENE-DMD in the FUNCTION-DMD trial. The first patient experienced no serious adverse events or events of special interest through Week 8, supporting the study's progression.","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/8916","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\/4"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/comments?post=8916"}],"version-history":[{"count":2,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/8916\/revisions"}],"predecessor-version":[{"id":8920,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/8916\/revisions\/8920"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media\/8918"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media?parent=8916"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/categories?post=8916"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/tags?post=8916"}],"curies":[{"name":"\u53ef\u6e7f\u6027\u7c89\u5242","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}