{"id":6289,"date":"2026-04-29T17:14:16","date_gmt":"2026-04-29T14:14:16","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=6289"},"modified":"2026-04-29T17:16:44","modified_gmt":"2026-04-29T14:16:44","slug":"precision-biosciences-pbgene-dmd-phase-1-2a-function-dmd-clinical-trial-nct07429240","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/zh\/precision-biosciences-pbgene-dmd-phase-1-2a-function-dmd-clinical-trial-nct07429240\/","title":{"rendered":"Precision BioSciences\u516c\u53f8\u5ba3\u5e03\u542f\u52a8\u9488\u5bf9\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u95e8\u8bca\u60a3\u8005\u7684PBGENE-DMD\u836f\u72691\/2a\u671f\uff08FUNCTION-DMD\uff09\u7814\u7a76\u3002"},"content":{"rendered":"<p class=\"wp-block-paragraph\"><strong>Precision BioSciences \u5ba3\u5e03\u542f\u52a8 PBGENE-DMD (NCT07429240) \u7684 1\/2a \u671f\u8bd5\u9a8c\uff0c\u53d7\u8bd5\u8005\u4e3a 2 \u81f3 7 \u5c81\u7684\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u95e8\u8bca\u60a3\u8005\u3002.<\/strong> \u5728\u672c\u8bd5\u9a8c\u4e2d\uff0c\u5c06\u5bf9\u5355\u5242\u91cf FUNCTION-DMD \u8fdb\u884c\u4e3a\u671f 130 \u5468\u7684\u7814\u7a76\u3002. <strong>\u9605\u8bfb\u66f4\u591a\uff1a<\/strong> <a href=\"https:\/\/dmdwarrior.com\/zh\/precision-biosciences-pbgene-dmd-function-dmd-phase-1-2-clinical-study\/\" target=\"_blank\" rel=\"noreferrer noopener\">Precision BioSciences \u83b7\u5f97\u7f8e\u56fd FDA \u7814\u7a76\u6027\u65b0\u836f\u7533\u8bf7\u7684\u6279\u51c6<\/a><\/p>\n\n\n\n<div class=\"wp-block-rank-math-toc-block\" id=\"rank-math-toc\"><h2>\u76ee\u5f55<\/h2><nav><ul><li><a href=\"#what-is-pbgene-dmd-phase-1-2-a-clinical-trial\">PBGENE-DMD 1\/2a\u671f\u4e34\u5e8a\u8bd5\u9a8c\u662f\u4ec0\u4e48\uff1f<\/a><\/li><li><a href=\"#pbgene-dmd-phase-1-2-a-nct-07429240\">PBGENE-DMD \u9636\u6bb5 1\/\u200b2a \u2013 NCT07429240<\/a><\/li><li><a href=\"#how-to-participate-in-the-pbgene-dmd-phase-1-2-a-clinical-trial\">\u5982\u4f55\u53c2\u4e0e PBGENE-DMD 1\/2a \u671f\u4e34\u5e8a\u8bd5\u9a8c<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u8fd9\u9879 1\/2a \u671f\u7814\u7a76\u65e8\u5728\u8bc4\u4f30 PBGENE-DMD \u5728\u60a3\u6709 DMD \u4e14\u5177\u6709\u9002\u5408\u5207\u9664\u5916\u663e\u5b50 45-55 \u7684\u7a81\u53d8\u7684\u4e2a\u4f53\u4e2d\u7684\u5b89\u5168\u6027\u3001\u8010\u53d7\u6027\u548c\u65e9\u671f\u7597\u6548\u8ff9\u8c61\u3002.<\/strong> \u9274\u4e8e\u76ee\u524d\u6cbb\u7597\u65b9\u6848\u7684\u5c40\u9650\u6027\uff0cPBGENE-DMD \u63d0\u4f9b\u4e86\u4e00\u79cd\u6709\u5e0c\u671b\u7684\u65b0\u7b56\u7565\uff0c\u65e8\u5728\u5bf9\u60a3\u6709 DMD \u7684\u6700\u5927\u5206\u5b50\u4e9a\u7ec4\u4eba\u7fa4\u7684\u6f5c\u5728\u9057\u4f20\u7f3a\u9677\u8fdb\u884c\u4e00\u6b21\u6027\u3001\u6301\u4e45\u7684\u7ea0\u6b63\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u672c\u8bd5\u9a8c\u6700\u591a\u53ef\u62db\u52df 18 \u540d\u53c2\u4e0e\u8005\u3002\u6bcf\u4f4d\u53c2\u4e0e\u8005\u7684\u8bd5\u9a8c\u53c2\u4e0e\u603b\u65f6\u957f\u7ea6\u4e3a 130 \u5468\u3002.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"what-is-pbgene-dmd-phase-1-2-a-clinical-trial\">PBGENE-DMD 1\/2a\u671f\u4e34\u5e8a\u8bd5\u9a8c\u662f\u4ec0\u4e48\uff1f<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u8fd9\u662f\u4e00\u9879\u65e9\u671f\u4e34\u5e8a\u8bd5\u9a8c\uff0c\u65e8\u5728\u6d4b\u8bd5\u4e00\u79cd\u540d\u4e3a PBGENE-DMD \u7684\u65b0\u7597\u6cd5\u5bf9\u60a3\u6709\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD) \u7684\u7537\u5b69\u548c\u7537\u6027\u60a3\u8005\u7684\u7597\u6548\u3002\u8fd9\u4e9b\u60a3\u8005\u643a\u5e26\u7279\u5b9a\u7684\u57fa\u56e0\u7a81\u53d8\uff0c\u53ef\u80fd\u5bf9\u8be5\u7597\u6cd5\u6709\u53cd\u5e94\u3002\u53c2\u4e0e\u8005\u5c06\u901a\u8fc7\u9759\u8109\u6ce8\u5c04\uff08\u624b\u81c2\u4e0a\u7684\u9759\u8109\uff09\u63a5\u53d7\u5355\u6b21\u5242\u91cf\u7684\u6cbb\u7597\u3002\u533b\u751f\u5c06\u5bc6\u5207\u76d1\u6d4b\u5b89\u5168\u6027\u3001\u526f\u4f5c\u7528\u4ee5\u53ca\u6cbb\u7597\u8d77\u6548\u7684\u65e9\u671f\u8ff9\u8c61\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u4e3a\u4e86\u5e2e\u52a9\u51cf\u5c11\u53ef\u80fd\u7684\u514d\u75ab\u53cd\u5e94\uff0c\u53c2\u4e0e\u8005\u5728\u6cbb\u7597\u524d\u540e\u8fd8\u5c06\u63a5\u53d7\u77ed\u671f\u836f\u7269\u6cbb\u7597\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u8fd9\u9879\u7814\u7a76\u5206\u4e3a\u4e24\u90e8\u5206\u3002\u7b2c\u4e00\u90e8\u5206\uff0c\u7814\u7a76\u4eba\u5458\u5c06\u786e\u5b9a\u4e00\u4e2a\u5b89\u5168\u4e14\u8010\u53d7\u6027\u826f\u597d\u7684\u5242\u91cf\u3002\u5982\u679c\u8be5\u5242\u91cf\u5f97\u5230\u786e\u8ba4\uff0c\u7b2c\u4e8c\u90e8\u5206\u5c06\u7eb3\u5165\u66f4\u591a\u53d7\u8bd5\u8005\uff0c\u4ee5\u8fdb\u4e00\u6b65\u8bc4\u4f30\u6cbb\u7597\u6548\u679c\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u8fd9\u9879\u7814\u7a76\u5c06\u6709\u6700\u591a 18 \u540d\u53c2\u4e0e\u8005\u3002\u6bcf\u4f4d\u53c2\u4e0e\u8005\u5c06\u88ab\u8ddf\u8e2a\u7ea6 2 \u5e74\u534a\uff08\u7ea6 130 \u5468\uff09\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u4e86\u89e3\u66f4\u591a\uff1a <a href=\"https:\/\/dmdwarrior.com\/zh\/faq-pbgene-dmd-gene-editing-therapy\/\" target=\"_blank\" rel=\"noreferrer noopener\">PBGENE-DMD\u57fa\u56e0\u7f16\u8f91\u7597\u6cd5\uff1a\u76ca\u5904\u3001\u8d44\u683c\u548c\u5e38\u89c1\u95ee\u9898\u89e3\u7b54<\/a><\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"pbgene-dmd-phase-1-2-a-nct-07429240\">PBGENE-DMD \u9636\u6bb5 1\/\u200b2a \u2013 NCT07429240<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u4e00\u9879 1\/2a \u671f\u3001\u591a\u4e2d\u5fc3\u3001\u5f00\u653e\u6807\u7b7e\u7814\u7a76\uff0c\u65e8\u5728\u8bc4\u4f30 PBGENE-DMD \u5728\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (FUNCTION-DMD) \u60a3\u8005\u4e2d\u7684\u5b89\u5168\u6027\u3001\u8010\u53d7\u6027\u548c\u521d\u6b65\u7597\u6548\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u5b66\u4e60\u5f00\u59cb\u65f6\u95f4\uff08\u9884\u8ba1\uff09\uff1a<\/strong> 2026-03<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u5c0f\u5b66\u5b8c\u6210\u60c5\u51b5\uff08\u9884\u8ba1\uff09\uff1a<\/strong> 2029-11<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u7814\u7a76\u5b8c\u6210\u65f6\u95f4\uff08\u9884\u8ba1\uff09\uff1a<\/strong> 2029-12<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u53c2\u4e0e\u4eba\u6570\uff08\u9884\u4f30\uff09\uff1a<\/strong> 18<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u7814\u7a76\u7c7b\u578b\uff1a<\/strong> \u4ecb\u5165\u6cbb\u7597<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u5730\u70b9\uff1a<\/strong> \u963f\u80af\u8272\u5dde\u513f\u7ae5\u533b\u9662<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u7b26\u5408\u5b66\u4e60\u6761\u4ef6\u7684\u5e74\u9f84\uff1a<\/strong> 2\u5c81\u81f37\u5c81\uff08\u513f\u7ae5\uff09<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u4e3b\u8981\u7eb3\u5165\u548c\u6392\u9664\u6807\u51c6\u3002\u2013 <a href=\"https:\/\/clinicaltrials.gov\/study\/NCT07429240\" target=\"_blank\" rel=\"noreferrer noopener\">NCT07429240<\/a><\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"how-to-participate-in-the-pbgene-dmd-phase-1-2-a-clinical-trial\">\u5982\u4f55\u53c2\u4e0e PBGENE-DMD 1\/2a \u671f\u4e34\u5e8a\u8bd5\u9a8c<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u8981\u53c2\u4e0e PBGENE-DMD 1\/2a \u671f\u4e34\u5e8a\u8bd5\u9a8c\uff0c\u60a8\u53ef\u4ee5\u4f7f\u7528\u4ee5\u4e0b\u8054\u7cfb\u4fe1\u606f\u3002.&nbsp;<strong>\u4e86\u89e3\u66f4\u591a<\/strong>:&nbsp;<a href=\"https:\/\/dmdwarrior.com\/zh\/how-to-participate-in-clinical-trials-for-duchenne\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u5982\u4f55\u53c2\u52a0\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD) \u4e34\u5e8a\u8bd5\u9a8c\uff1f<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u59d3\u540d\uff1a<\/strong> \u827e\u7433\u00b7\u5e03\u91cc\u65af\u6258<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u7535\u8bdd\u53f7\u7801\uff1a<\/strong> +1-610-462-1004<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u7535\u5b50\u90ae\u4ef6\uff1a<\/strong> function-DMD@precisionbiosciences.com<\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u9605\u8bfb\u66f4\u591a<\/strong>:&nbsp;<a href=\"https:\/\/dmdwarrior.com\/zh\/dmd-therapies\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u4e34\u5e8a\u8bd5\u9a8c\uff08\u6240\u6709\u7814\u7a76\u5217\u8868\uff09<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>Precision BioSciences announced the commencement of a Phase 1\/\u200b2a trial of PBGENE-DMD (NCT07429240) in ambulatory patients with Duchenne aged 2 to 7 years. In this trial, single dose of FUNCTION-DMD will be studied over 130 weeks. Read More: Precision BioSciences Receives U.S. FDA Clearance of Investigational New Drug Application This Phase 1\/2a study is designed [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":6291,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[27],"tags":[137,242,359,479,72,493,314,313],"class_list":["post-6289","post","type-post","status-publish","format-standard","has-post-thumbnail","category-research","tag-clinical-trial","tag-clinical-trials","tag-full-length-dystrophin","tag-function-dmd","tag-gene-editing","tag-nct07429240","tag-pbgene-dmd","tag-precision-biosciences"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/6289","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=6289"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/6289\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media\/6291"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media?parent=6289"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/categories?post=6289"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/tags?post=6289"}],"curies":[{"name":"\u53ef\u6e7f\u6027\u7c89\u5242","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}