{"id":4000,"date":"2025-06-25T14:58:50","date_gmt":"2025-06-25T11:58:50","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=4000"},"modified":"2025-06-25T15:02:05","modified_gmt":"2025-06-25T12:02:05","slug":"precision-biosciences-receives-fda-rare-pediatric-disease-designation-for-pbgene-dmd-for-the-treatment-of-duchenne-muscular-dystrophy","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/zh\/precision-biosciences-receives-fda-rare-pediatric-disease-designation-for-pbgene-dmd-for-the-treatment-of-duchenne-muscular-dystrophy\/","title":{"rendered":"Precision BioSciences \u83b7\u5f97 FDA \u7f55\u89c1\u513f\u79d1\u75be\u75c5\u8ba4\u5b9a\uff0cPBGENE-DMD \u7528\u4e8e\u6cbb\u7597\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7"},"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\u8be5\u516c\u53f8\u4eca\u5929\u5ba3\u5e03\uff0c\u7f8e\u56fd\u98df\u54c1\u836f\u54c1\u76d1\u7763\u7ba1\u7406\u5c40 (FDA) \u5df2\u6388\u4e88 PBGENE-DMD \u7f55\u89c1\u513f\u79d1\u75be\u75c5\u8ba4\u5b9a\uff0c\u7528\u4e8e\u6cbb\u7597\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD)\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=\"#near-full-length-dystrophin-protein\">\u63a5\u8fd1\u5168\u957f\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d<\/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=\"near-full-length-dystrophin-protein\">\u63a5\u8fd1\u5168\u957f\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Precision BioSciences \u9996\u5e2d\u5f00\u53d1\u548c\u4e1a\u52a1\u5b98 Cindy Atwell \u8868\u793a\uff1a\u201c\u83b7\u5f97\u7f55\u89c1\u513f\u79d1\u75be\u75c5\u8ba4\u5b9a\u51f8\u663e\u4e86\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD) \u60a3\u513f\u4e9f\u9700\u65b0\u7684\u6cbb\u7597\u65b9\u6848\uff0c\u8fd9\u4e00\u5de8\u5927\u672a\u6ee1\u8db3\u9700\u6c42\u4e9f\u5f85\u6ee1\u8db3\u3002\u201d \u201c\u6211\u4eec\u9996\u521b\u7684 PBGENE-DMD \u9879\u76ee\u5229\u7528\u6211\u4eec ARCUS \u57fa\u56e0\u7f16\u8f91\u5e73\u53f0\u7684\u5dee\u5f02\u5316\u529f\u80fd\uff0c\u5207\u9664\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u57fa\u56e0\u7684 45-55 \u53f7\u5916\u663e\u5b50\uff0c\u6709\u671b\u4e3a\u591a\u8fbe 60% \u7684\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD) \u60a3\u8005\u5e26\u6765\u663e\u8457\u7684\u6539\u5584\u548c\u6301\u4e45\u7684\u529f\u80fd\u83b7\u76ca\u3002\u6211\u4eec\u7684\u65b9\u6cd5\u65e8\u5728\u6c38\u4e45\u7f16\u8f91\u60a3\u8005\u81ea\u8eab\u7684 DNA \u5e8f\u5217\uff0c\u4ece\u800c\u751f\u6210\u5929\u7136\u4ea7\u751f\u7684\u3001\u63a5\u8fd1\u5168\u957f\u7684\u3001\u5df2\u77e5\u5728\u4eba\u4f53\u4e2d\u5177\u6709\u529f\u80fd\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u3002\u6211\u4eec\u671f\u5f85\u5b8c\u6210\u76ee\u524d\u6b63\u5728\u8fdb\u884c\u7684\u6700\u7ec8 IND \u7533\u62a5\u6bd2\u7406\u5b66\u7814\u7a76\uff0c\u5e76\u751f\u6210\u521d\u6b65\u4e34\u5e8a\u6570\u636e\uff0c\u9884\u8ba1\u5c06\u4e8e 2026 \u5e74\u5b8c\u6210\u3002\u201d<\/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 \u662f Precision \u516c\u53f8\u7528\u4e8e\u6cbb\u7597\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD) \u7684\u5f00\u53d1\u9879\u76ee\u3002\u8be5\u9879\u76ee\u4f7f\u7528\u4e24\u79cd\u4e92\u8865\u7684 ARCUS \u6838\u9178\u9176\uff0c\u901a\u8fc7\u4e00\u6b21\u6027\u7ed9\u836f\uff0c\u4ee5\u5355\u4e2a\u817a\u76f8\u5173\u75c5\u6bd2 (AAV) \u7684\u5f62\u5f0f\u9012\u9001\uff0c\u5207\u9664\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u57fa\u56e0\u7684 45-55 \u4e2a\u5916\u663e\u5b50\uff0c\u65e8\u5728\u6062\u590d\u4f53\u5185\u63a5\u8fd1\u5168\u957f\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\uff0c\u4ece\u800c\u6539\u5584\u529f\u80fd\u9884\u540e\u3002PBGENE-DMD \u65e8\u5728\u6cbb\u7597\u8d85\u8fc7 60% \u7684\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (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>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u4e86\u89e3\u66f4\u591a\uff1a<\/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>","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 that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation for PBGENE-DMD for the treatment of Duchenne muscular dystrophy (DMD). Near Full-Length [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":4001,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[359,72,314,313],"class_list":["post-4000","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"],"subtitle":"\u4eca\u5929\uff0cPrecision BioSciences \u5ba3\u5e03 FDA \u5df2\u6388\u4e88 PBGENE-DMD \u7f55\u89c1\u513f\u79d1\u75be\u75c5\u8ba4\u5b9a\uff0c\u7528\u4e8e\u6cbb\u7597\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD)\u3002","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/4000","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=4000"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/4000\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media\/4001"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media?parent=4000"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/categories?post=4000"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/tags?post=4000"}],"curies":[{"name":"\u53ef\u6e7f\u6027\u7c89\u5242","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}