{"id":4132,"date":"2025-07-16T18:57:40","date_gmt":"2025-07-16T15:57:40","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=4132"},"modified":"2025-07-16T18:57:41","modified_gmt":"2025-07-16T15:57:41","slug":"precision-biosciences-highlights-new-preclinical-data-pbgene-dmd","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/zh\/precision-biosciences-highlights-new-preclinical-data-pbgene-dmd\/","title":{"rendered":"Precision BioSciences \u91cd\u70b9\u4ecb\u7ecd PBGENE-DMD \u6cbb\u7597\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684\u6700\u65b0\u4e34\u5e8a\u524d\u6570\u636e"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Precision BioSciences \u662f\u4e00\u5bb6\u4e34\u5e8a\u9636\u6bb5\u57fa\u56e0\u7f16\u8f91\u516c\u53f8\uff0c\u5229\u7528\u5176\u65b0\u9896\u7684\u4e13\u6709 ARCUS\u00ae \u5e73\u53f0\u5f00\u53d1\u9488\u5bf9\u9ad8\u5ea6\u672a\u6ee1\u8db3\u9700\u6c42\u75be\u75c5\u7684\u4f53\u5185\u57fa\u56e0\u7f16\u8f91\u7597\u6cd5\uff0c\u4eca\u5929\u5ba3\u5e03\u66f4\u65b0\u7684\u989d\u5916\u4e34\u5e8a\u524d\u6570\u636e\uff0c\u8fdb\u4e00\u6b65\u9a8c\u8bc1\u4e86\u652f\u6301\u5feb\u901f\u5f00\u53d1 PBGENE-DMD \u7684\u79d1\u5b66\u539f\u7406\uff0cPBGENE-DMD \u662f\u8be5\u516c\u53f8\u9488\u5bf9\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD) \u7684\u9996\u521b\u4f53\u5185\u57fa\u56e0\u7f16\u8f91\u65b9\u6cd5\uff0c\u7528\u4e8e\u9996\u6b21\u4eba\u4f53\u4e34\u5e8a\u7814\u7a76\u3002<\/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=\"#new-preclinical-data-for-pbgene-dmd\">PBGENE-DMD \u7684\u65b0\u4e34\u5e8a\u524d\u6570\u636e<\/a><\/li><li><a href=\"#pbgene-dmd-is-the-first-in-vivo-gene-editing-program\">PBGENE-DMD\u662f\u7b2c\u4e00\u4e2a\u4f53\u5185\u57fa\u56e0\u7f16\u8f91\u7a0b\u5e8f<\/a><\/li><li><a href=\"#pbgene-dmd-between-exons-45-55\">PBGENE-DMD\uff1a\u5916\u663e\u5b50 45-55 \u4e4b\u95f4<\/a><\/li><li><a href=\"#what-is-pbgene-dmd\">\u4ec0\u4e48\u662f PBGENE-DMD\uff1f<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"new-preclinical-data-for-pbgene-dmd\">PBGENE-DMD \u7684\u65b0\u4e34\u5e8a\u524d\u6570\u636e<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>PBGENE-DMD \u6700\u7ec8\u4e34\u5e8a\u5019\u9009\u836f\u7269\u7684\u6700\u65b0\u4e34\u5e8a\u524d\u6570\u636e\u663e\u793a\uff0c\u4e3b\u8981\u808c\u8089\u7c7b\u578b\u7684\u6297\u808c\u840e\u7f29\u86cb\u767d\u9633\u6027\u808c\u8089\u7ec6\u80de\u6709\u6240\u589e\u52a0\uff0c\u8fd9\u53ef\u80fd\u662f\u7531\u808c\u8089\u536b\u661f\u7ec6\u80de\u7684\u7f16\u8f91\u6240\u81f4\u3002<\/li>\n<\/ul>\n\n\n\n<ul class=\"wp-block-list\">\n<li>PBGENE-DMD \u662f\u4e00\u79cd\u4e00\u6d41\u7684\u4f53\u5185\u57fa\u56e0\u7f16\u8f91\u65b9\u6cd5\uff0c\u9002\u7528\u4e8e\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u60a3\u8005\uff0c\u6700\u9ad8\u53ef\u8fbe 60%\uff0c\u7279\u522b\u662f\u90a3\u4e9b\u5728\u5916\u663e\u5b50 45-55 \u4e4b\u95f4\u7684\u201c\u70ed\u70b9\u201d\u533a\u57df\u53d7\u5230\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7a81\u53d8\u5f71\u54cd\u7684\u60a3\u8005 -<\/li>\n<\/ul>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u7cbe\u51c6\u5b9a\u4f4d\uff0c\u4e8e 2025 \u5e74\u63d0\u4ea4 PBGENE-DMD \u7684\u65b0\u836f\u4e34\u5e8a\u8bd5\u9a8c\u7533\u8bf7 (IND) \u548c\/\u6216\u4e34\u5e8a\u8bd5\u9a8c\u7533\u8bf7 (CTA)\uff0c\u9884\u8ba1 2026 \u5e74\u83b7\u5f97\u4e34\u5e8a\u6570\u636e \u2013<\/li>\n<\/ul>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"pbgene-dmd-is-the-first-in-vivo-gene-editing-program\">PBGENE-DMD\u662f\u7b2c\u4e00\u4e2a\u4f53\u5185\u57fa\u56e0\u7f16\u8f91\u7a0b\u5e8f<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><br>\u201c\u5728\u5bf9\u60a3\u6709 DMD \u7684\u5c0f\u9f20\u6a21\u578b\u8fdb\u884c\u7684\u957f\u671f\u8010\u4e45\u6027\u7814\u7a76\u4e2d\uff0c\u6211\u4eec\u89c2\u5bdf\u5230\u5728\u4e09\u5230\u4e5d\u4e2a\u6708\u7684\u65f6\u95f4\u91cc\uff0c\u80a1\u56db\u5934\u808c\u3001\u8153\u80a0\u808c\uff08\u5c0f\u817f\uff09\u3001\u5fc3\u810f\u548c\u6a2a\u8188\u819c\u4e2d\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u9633\u6027\u808c\u8089\u7ec6\u80de\u589e\u52a0\u4e86\u4e09\u500d\u3002 <strong>\u5728\u8153\u80a0\u808c\u4e2d\uff0c\u9ad8\u8fbe 85% \u7684\u7ec6\u80de\u5448\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u9633\u6027\uff0c\u201dPrecision BioSciences \u9996\u5e2d\u79d1\u5b66\u5b98 Cassie Gorsuch \u535a\u58eb\u8bf4\u3002<\/strong> \u8fd9\u4e9b\u65b0\u6570\u636e\u5efa\u7acb\u57282025\u5e745\u6708ASGCT\u5927\u4f1a\u4e0a\u5206\u4eab\u7684\u4e34\u5e8a\u524d\u6570\u636e\u4e4b\u4e0a\uff0c\u8868\u660ePBGENE-DMD\u6cbb\u7597\u5728\u76f8\u540c\u76843\u4e2a\u6708\u548c9\u4e2a\u6708\u65f6\u95f4\u70b9\u663e\u8457\u4e14\u6301\u7eed\u5730\u63d0\u9ad8\u4e86\u6700\u5927\u529b\u91cf\u8f93\u51fa\u3002\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u9633\u6027\u7ec6\u80de\u7684\u5e7f\u6cdb\u589e\u52a0\uff0c\u4ee5\u53ca\u7ec4\u7ec7\u4e2d\u68c0\u6d4b\u5230\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u589e\u52a0\uff0c\u8fdb\u4e00\u6b65\u8bc1\u5b9e\u4e86\u968f\u65f6\u95f4\u63a8\u79fb\u89c2\u5bdf\u5230\u7684\u808c\u8089\u529f\u80fd\u6539\u5584\uff0c\u8fd9\u53ef\u80fd\u5f52\u56e0\u4e8e\u7f16\u8f91\u540e\u7684\u536b\u661f\u7ec6\u80de\uff0c\u672c\u7814\u7a76\u4e2d\u4e5f\u89c2\u5bdf\u5230\u4e86\u8fd9\u4e9b\u536b\u661f\u7ec6\u80de\u3002\u6211\u4eec\u76f8\u4fe1\uff0c\u8fd9\u4e9b\u7ed3\u679c\u8bc1\u660e\u4e86PBGENE-DMD\u57fa\u56e0\u7f16\u8f91\u65b9\u6cd5\u7684\u72ec\u7279\u6f5c\u529b\uff0c\u5b83\u65e0\u9700AAV\u6301\u7eed\u5b58\u5728\u5373\u53ef\u4ea7\u751f\u6301\u7eed\u7684\u529f\u80fd\u76ca\u5904\uff0c\u800cAAV\u6301\u7eed\u5b58\u5728\u662f\u5fae\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u6cbb\u7597\u65b9\u6cd5\u6240\u5fc5\u9700\u7684\u3002\u6211\u4eec\u671f\u5f85\u5728\u672a\u6765\u7684\u79d1\u5b66\u4f1a\u8bae\u4e0a\u5c55\u793a\u5b8c\u6574\u7684\u6570\u636e\u96c6\u3002\u9274\u4e8e\u4e34\u5e8a\u524d\u8bc1\u636e\u7684\u5b8c\u6574\u6027\uff0c\u6211\u4eec\u4ecd\u7136\u5145\u6ee1\u671f\u5f85\uff0c\u5e76\u575a\u5b9a\u4e0d\u79fb\u5730\u63a8\u8fdb\u8be5\u9879\u76ee\u8d70\u5411\u4e34\u5e8a\u3002<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"pbgene-dmd-between-exons-45-55\">PBGENE-DMD\uff1a\u5916\u663e\u5b50 45-55 \u4e4b\u95f4<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u76ee\u524d\uff0c\u5c1a\u65e0\u5df2\u83b7\u6279\u51c6\u6216\u6b63\u5728\u7814\u53d1\u7684\u6cbb\u7597\u65b9\u6cd5\u80fd\u591f\u968f\u7740\u65f6\u95f4\u7684\u63a8\u79fb\u663e\u8457\u6539\u5584\u808c\u8089\u529f\u80fd\uff0c\u4ece\u800c\u6709\u76ca\u5730\u6539\u53d8 DMD \u7684\u957f\u671f\u9884\u540e\u3002 <strong>PBGENE-DMD \u662f\u7b2c\u4e00\u4e2a\u4f53\u5185\u57fa\u56e0\u7f16\u8f91\u7a0b\u5e8f\uff0c\u5b83\u6709\u53ef\u80fd\u6539\u53d8\u6cbb\u7597\u6a21\u5f0f\u5e76\u4e3a\u5927\u591a\u6570\u60a3\u8005\u5e26\u6765\u6301\u4e45\u7684\u529f\u80fd\u6539\u5584\uff0c\u56e0\u4e3a\u591a\u8fbe 60% \u7684\u60a3\u8005\u5728\u5916\u663e\u5b50 45-55 \u4e4b\u95f4\u7684\u201c\u70ed\u70b9\u201d\u533a\u57df\u643a\u5e26\u7a81\u53d8\u3002<\/strong> Precision \u7684\u65b9\u6cd5\u65e8\u5728\u6c38\u4e45\u7f16\u8f91\u60a3\u8005\u81ea\u8eab\u7684 DNA \u5e8f\u5217\uff0c\u4ece\u800c\u81ea\u7136\u751f\u6210\u8fd1\u4e4e\u5168\u957f\u7684\u3001\u5df2\u77e5\u5728\u4eba\u4f53\u4e2d\u5177\u6709\u529f\u80fd\u7684\u6297\u808c\u840e\u7f29\u86cb\u767d\u3002\u6700\u7ec8\u7684 IND \u6bd2\u7406\u5b66\u7814\u7a76\u6b63\u5728\u8fdb\u884c\u4e2d\uff0c\u76ee\u6807\u662f\u5728 2025 \u5e74\u63d0\u4ea4 IND \u548c\/\u6216 CTA \u7533\u8bf7\uff0c\u5e76\u9884\u8ba1\u5728 2026 \u5e74\u83b7\u5f97\u521d\u6b65\u4e34\u5e8a\u6570\u636e\u3002\u516c\u53f8\u76f8\u4fe1\uff0c\u5176\u76ee\u524d\u7684\u73b0\u91d1\u6d41\u8db3\u4ee5\u652f\u6301\u5176\u76ee\u524d\u5904\u4e8e 1 \u671f\u4e34\u5e8a\u9636\u6bb5\u7684 PBGENE-HBV \u548c PBGENE-DMD \u7684\u8fdb\u5c55\uff0c\u76f4\u81f3 1 \u671f\u4e34\u5e8a\u7ed3\u679c\u51fa\u7089\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u9605\u8bfb\u66f4\u591a<\/strong>: <a href=\"https:\/\/dmdwarrior.com\/zh\/precision-biosciences-pbgene-dmd-restores-production-of-nearly-full-length-dystrophin-protein\/\">Precision BioSciences PBGENE-DMD \u6062\u590d\u8fd1\u5168\u957f\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u4ea7\u751f<\/a><\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"what-is-pbgene-dmd\">\u4ec0\u4e48\u662f PBGENE-DMD\uff1f<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">PBGENE-DMD\u662fPrecision\u9488\u5bf9DMD\u6cbb\u7597\u5f00\u53d1\u7684\u65b9\u6848\u3002 <strong>\u8be5\u65b9\u6cd5\u4f7f\u7528\u4e24\u79cd\u4e92\u8865\u7684 ARCUS \u6838\u9178\u9176\uff0c\u901a\u8fc7\u4e00\u6b21\u6027\u7ed9\u836f\u5728\u5355\u4e2a AAV \u4e2d\u9012\u9001\uff0c\u5207\u9664\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u57fa\u56e0\u7684\u5916\u663e\u5b50 45-55\uff0c\u76ee\u7684\u662f\u6062\u590d\u4f53\u5185\u63a5\u8fd1\u5168\u957f\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\uff0c\u4ece\u800c\u6539\u5584\u529f\u80fd\u7ed3\u679c\u3002<\/strong> PBGENE-DMD \u65e8\u5728\u89e3\u51b3\u8d85\u8fc7 60% \u7684 DMD \u60a3\u8005\u7fa4\u4f53\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u5728\u4e34\u5e8a\u524d\u7814\u7a76\u4e2d\uff0cPBGENE-DMD \u663e\u793a\u51fa\u9776\u5411\u4f5c\u7528\u4e8e\u4e0e\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD) \u8fdb\u5c55\u76f8\u5173\u7684\u5173\u952e\u808c\u8089\u7c7b\u578b\u7684\u80fd\u529b\uff0c\u5e76\u5728\u4eba\u6e90\u5316 DMD \u5c0f\u9f20\u6a21\u578b\u4e2d\u4ea7\u751f\u4e86\u663e\u8457\u4e14\u6301\u4e45\u7684\u529f\u80fd\u6539\u5584\u3002PBGENE-DMD \u6062\u590d\u4e86\u4eba\u4f53\u5728\u591a\u79cd\u808c\u8089\uff08\u5305\u62ec\u5fc3\u810f\u7ec4\u7ec7\u548c\u5404\u79cd\u5173\u952e\u9aa8\u9abc\u808c\u7fa4\uff09\u4e2d\u4ea7\u751f\u63a5\u8fd1\u5168\u957f\u529f\u80fd\u6027\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u80fd\u529b\u3002\u6b64\u5916\uff0cPBGENE-DMD \u8fd8\u7f16\u8f91\u4e86\u536b\u661f\u808c\u8089\u5e72\u7ec6\u80de\uff0c\u8fd9\u88ab\u8ba4\u4e3a\u5bf9\u957f\u671f\u6301\u4e45\u6027\u548c\u6301\u7eed\u7684\u529f\u80fd\u6539\u5584\u81f3\u5173\u91cd\u8981\u3002<\/p>","protected":false},"excerpt":{"rendered":"<p>Precision BioSciences a clinical stage gene editing company utilizing its novel proprietary ARCUS\u00ae platform to develop in vivo gene editing therapies for diseases with high unmet need, today announced updated additional preclinical data further validating the scientific rationale supporting the rapid development of PBGENE-DMD, the Company\u2019s first-in-class in vivo gene editing approach for Duchenne muscular [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":4134,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[359,72,314,313],"class_list":["post-4132","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-full-length-dystrophin","tag-gene-editing","tag-pbgene-dmd","tag-precision-biosciences"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/4132","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=4132"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/4132\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media\/4134"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media?parent=4132"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/categories?post=4132"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/tags?post=4132"}],"curies":[{"name":"\u53ef\u6e7f\u6027\u7c89\u5242","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}