{"id":6203,"date":"2026-02-11T17:08:53","date_gmt":"2026-02-11T14:08:53","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=6203"},"modified":"2026-02-11T17:08:55","modified_gmt":"2026-02-11T14:08:55","slug":"precision-biosciences-pbgene-dmd-function-dmd-phase-1-2-clinical-study","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/zh\/precision-biosciences-pbgene-dmd-function-dmd-phase-1-2-clinical-study\/","title":{"rendered":"Precision BioSciences \u83b7\u5f97\u7f8e\u56fd FDA \u6279\u51c6\uff0c\u7528\u4e8e\u6cbb\u7597\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684\u9996\u521b\u836f\u7269 PBGENE-DMD \u7684\u7814\u7a76\u6027\u65b0\u836f\u7533\u8bf7\u83b7\u6279"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Precision BioSciences \u5ba3\u5e03\uff0c\u516c\u53f8\u5df2\u6536\u5230\u7f8e\u56fd\u98df\u54c1\u836f\u54c1\u76d1\u7763\u7ba1\u7406\u5c40 (FDA) \u7684\u201c\u7814\u7a76\u53ef\u4ee5\u8fdb\u884c\u201d\u901a\u77e5\u3002. <strong>\u8fd9\u4f7f\u5f97 Precision BioSciences \u80fd\u591f\u542f\u52a8 IRB \u6d3b\u52a8\u548c\u4e34\u5e8a\u8bd5\u9a8c\u4e2d\u5fc3\u6fc0\u6d3b\uff0c\u4ee5\u5f00\u5c55 FUNCTION-DMD \u7684 1\/2 \u671f\u4e34\u5e8a\u8bd5\u9a8c\uff0c\u7528\u4e8e\u6cbb\u7597\u5728\u9ad8\u5ea6\u4e13\u4e1a\u5316\u7684\u4e34\u5e8a\u8bd5\u9a8c\u4e2d\u5fc3\u63a5\u53d7 PBGENE-DMD \u6cbb\u7597\u7684\u53ef\u884c\u8d70\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD) \u60a3\u8005\u3002.<\/strong> \u8be5\u7814\u7a76\u7684\u76ee\u7684\u662f\u8bc4\u4f30\u5b89\u5168\u6027\u3001\u8010\u53d7\u6027\u548c\u6709\u6548\u6027\uff0c\u5305\u62ec\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u8868\u8fbe\u548cDMD\u60a3\u8005\u7684\u529f\u80fd\u7ed3\u679c\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u2013 \u7f8e\u56fd FDA \u7814\u7a76\u53ef\u80fd\u7ee7\u7eed\u8fdb\u884c\u7684\u901a\u77e5\uff0c\u4f7f\u5f97\u9488\u5bf9\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD) \u60a3\u8005\u7684 FUNCTION-DMD 1\/2 \u671f\u4e34\u5e8a\u7814\u7a76\u7684\u4e34\u5e8a\u8bd5\u9a8c\u4e2d\u5fc3\u542f\u52a8\u6210\u4e3a\u53ef\u80fd \u2013<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u2013 \u591a\u4e2a\u4e16\u754c\u4e00\u6d41\u7684DMD\u4e34\u5e8a\u8bd5\u9a8c\u4e2d\u5fc3\u6b63\u5728\u8fdb\u884c\u673a\u6784\u5ba1\u67e5\u59d4\u5458\u4f1a\uff08IRB\uff09\u5ba1\u6279\u6d41\u7a0b \u2013<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u2013 PBGENE-DMD \u662f\u4e00\u79cd\u4f53\u5185\u57fa\u56e0\u7f16\u8f91\u7814\u7a76\u4ea7\u54c1\uff0c\u65e8\u5728\u901a\u8fc7\u4e00\u79cd\u65b0\u578b\u7684\u4e00\u6b21\u6027\u57fa\u56e0\u7f16\u8f91\u7597\u6cd5\u6765\u7ea0\u6b63 DMD \u7684\u6f5c\u5728\u9057\u4f20\u75c5\u56e0\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u2013 \u516c\u53f8\u8ba1\u5212\u5728\u808c\u8089\u840e\u7f29\u75c7\u534f\u4f1a\u4f1a\u8bae\u4e4b\u540e\uff0c\u4e8e 3 \u6708\u4efd\u4e3e\u529e\u4e00\u573a\u7ebf\u4e0a\u6d3b\u52a8\uff0c\u8ba8\u8bba PBGENE-DMD \u548c FUNCTION-DMD \u4e34\u5e8a\u7814\u7a76\uff0c\u5c4a\u65f6\u5c06\u9080\u8bf7\u5305\u62ec\u60a3\u8005\u6743\u76ca\u5021\u5bfc\u8005\u5728\u5185\u7684\u5173\u952e\u610f\u89c1\u9886\u8896\u53c2\u4e0e\u3002<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">\u4ec0\u4e48\u662f PBGENE-DMD\uff1f<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">PBGENE-DMD \u662f Precision \u516c\u53f8\u5168\u8d44\u62e5\u6709\u7684\u9996\u521b\u4f53\u5185\u57fa\u56e0\u7f16\u8f91\u7a0b\u5e8f\uff0c\u91c7\u7528\u57fa\u56e0\u5207\u9664\u65b9\u6cd5\uff0c\u65e8\u5728\u6c38\u4e45\u7ea0\u6b63 45 \u81f3 55 \u53f7\u5916\u663e\u5b50\u4e4b\u95f4\u7684\u7a81\u53d8\u60a3\u8005\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u57fa\u56e0\uff0c\u8be5\u7a81\u53d8\u4f4d\u4e8e\u201c\u70ed\u70b9\u201d\u533a\u57df\uff0c\u7ea6\u5360 DMD \u60a3\u8005\u4e2d\u6700\u5927\u7684\u5206\u5b50\u4e9a\u7fa4\uff08\u7ea6 60%\uff09\u3002. <strong>\u6cbb\u7597\u76ee\u6807\u662f\u6062\u590d\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u57fa\u56e0\u4ea7\u751f\u63a5\u8fd1\u5168\u957f\u7684\u529f\u80fd\u6027\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u80fd\u529b\u3002\u8fd9\u79cd\u86cb\u767d\u4fdd\u7559\u4e86\u5168\u957f\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684 80%\uff0c\u6bd4\u76ee\u524d\u5df2\u83b7\u6279\u51c6\u6216\u6b63\u5728\u5f00\u53d1\u7684\u5408\u6210\u5fae\u578b\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u6784\u5efa\u4f53\uff08\u5176\u5927\u5c0f\u7ea6\u4e3a\u5168\u957f\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684 34%\uff09\u5927\u5f97\u591a\u3002.<\/strong><\/p>\n\n\n\n<h2 class=\"wp-block-heading\">\u76ee\u6807\u662f\u57282026\u5e74\u4e0a\u534a\u5e74\u542f\u52a8\u9996\u4e2a\u4e34\u5e8a\u8bd5\u9a8c\u70b9\u3002<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u201cPBGENE-DMD\u83b7\u5f97FDA\u7684\u201d\u7814\u7a76\u53ef\u8fdb\u884c\u2019\u901a\u77e5\uff0c\u6807\u5fd7\u7740Precision BioSciences\u5728\u63a8\u8fdb\u5176\u7b2c\u4e8c\u4e2a\u5168\u8d44\u7814\u53d1\u9879\u76ee\u8fdb\u5165\u4e34\u5e8a\u8bd5\u9a8c\u9636\u6bb5\u65b9\u9762\u53c8\u53d6\u5f97\u4e86\u4e00\u9879\u76d1\u7ba1\u6210\u5c31\u3002\u5c3d\u7ba1\u76ee\u524d\u5df2\u6709\u83b7\u6279\u7597\u6cd5\uff0c\u4f46\u60a3\u6709\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\uff08DMD\uff09\u7684\u7537\u5b69\u4ecd\u7136\u7f3a\u4e4f\u80fd\u591f\u957f\u671f\u6539\u5584\u5176\u529f\u80fd\u72b6\u51b5\u7684\u6cbb\u7597\u65b9\u6848\u3002\u6211\u4eec\u5f88\u9ad8\u5174\u80fd\u5c06\u8fd9\u79cd\u9488\u5bf9DMD\u7684\u65b0\u578b\u57fa\u56e0\u5207\u9664\u7597\u6cd5\u5f15\u5165\u4e34\u5e8a\uff0c\u76ee\u6807\u662f\u57282026\u5e74\u4e0a\u534a\u5e74\u542f\u52a8\u7f8e\u56fd\u9996\u4e2a\u4e34\u5e8a\u8bd5\u9a8c\u4e2d\u5fc3\u3002\u6211\u4e3a\u6211\u4eec\u7684\u56e2\u961f\u611f\u5230\u81ea\u8c6a\uff0c\u4ed6\u4eec\u5b9e\u73b0\u4e86\u5728\u5e74\u5e95\u524d\u63d0\u4ea4\u65b0\u836f\u4e34\u5e8a\u8bd5\u9a8c\u7533\u8bf7\uff08IND\uff09\u7684\u76ee\u6807\uff0c\u5e76\u5728\u7b2c\u4e00\u5b63\u5ea6\u83b7\u5f97\u4e86FDA\u7684\u2018\u7814\u7a76\u53ef\u8fdb\u884c\u2019\u901a\u77e5\u3002\u516c\u53f8\u5c06\u7ee7\u7eed\u4e0e\u591a\u4e2a\u4f26\u7406\u5ba1\u67e5\u59d4\u5458\u4f1a\uff08IRB\uff09\u548cFDA\u5408\u4f5c\uff0c\u542f\u52a8\u4e34\u5e8a\u8bd5\u9a8c\u4e2d\u5fc3\u7684\u542f\u52a8\u5de5\u4f5c\u3002\u201d Precision BioSciences\u9996\u5e2d\u6267\u884c\u5b98Michael Amoroso\u8868\u793a\u3002.<\/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>Precision BioSciences announced that the Company received a Study May Proceed notification from the U.S. Food and Drug Administration (FDA). This allows Precision BioSciences to initiate IRB activities and clinical trial site activation for the FUNCTION-DMD Phase 1\/2 clinical trial for PBGENE-DMD for the treatment of ambulatory Duchenne muscular dystrophy (DMD) patients at highly specialized [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":6205,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[359,479,72,314,313],"class_list":["post-6203","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-full-length-dystrophin","tag-function-dmd","tag-gene-editing","tag-pbgene-dmd","tag-precision-biosciences"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/6203","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=6203"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/6203\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media\/6205"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media?parent=6203"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/categories?post=6203"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/tags?post=6203"}],"curies":[{"name":"\u53ef\u6e7f\u6027\u7c89\u5242","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}