{"id":4166,"date":"2025-07-23T16:48:01","date_gmt":"2025-07-23T13:48:01","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=4166"},"modified":"2025-07-23T16:50:07","modified_gmt":"2025-07-23T13:50:07","slug":"the-fda-designates-pbgene-dmd-as-an-orphan-drug-by-precision-biosciences-to-treat-duchenne-muscular-dystrophy","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/zh\/the-fda-designates-pbgene-dmd-as-an-orphan-drug-by-precision-biosciences-to-treat-duchenne-muscular-dystrophy\/","title":{"rendered":"FDA \u5c06 PBGENE-DMD \u6307\u5b9a\u4e3a\u6cbb\u7597\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684\u5b64\u513f\u836f"},"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 \u5b64\u513f\u836f\u79f0\u53f7\uff0c\u7528\u4e8e\u6cbb\u7597\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD)\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Precision BioSciences\u9996\u5e2d\u5f00\u53d1\u548c\u4e1a\u52a1\u5b98Cindy Atwell\u8868\u793a\uff1a\u201cFDA\u4e3aPBGENE-DMD\u6388\u4e88\u5b64\u513f\u836f\u8d44\u683c\u8ba4\u5b9a\uff0c\u51f8\u663e\u4e86\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u60a3\u513f\u4e9f\u5f85\u6ee1\u8db3\u7684\u5de8\u5927\u9700\u6c42\uff0c\u4ee5\u53ca\u63d0\u4f9b\u5b89\u5168\u6cbb\u7597\u65b9\u6848\u3001\u663e\u8457\u6539\u5584\u808c\u8089\u529f\u80fd\u7684\u7d27\u8feb\u6027\u3002\u201d \u201c\u8fd9\u4e00\u76d1\u7ba1\u91cc\u7a0b\u7891\u5efa\u7acb\u5728\u6211\u4eec\u8fd1\u671f\u83b7\u5f97\u513f\u79d1\u7f55\u89c1\u75c5\u8d44\u683c\u8ba4\u5b9a\u7684\u57fa\u7840\u4e0a\uff0c\u52a0\u4e0a\u6211\u4eec\u4e30\u5bcc\u7684\u4e34\u5e8a\u524d\u8bc1\u636e\uff0c\u8ba9\u6211\u4eec\u5bf9\u5c06\u8be5\u9879\u76ee\u63a8\u5411\u4e34\u5e8a\u5145\u6ee1\u4fe1\u5fc3\u3002\u5c55\u671b\u672a\u6765\uff0c\u6211\u4eec\u5c06\u7ee7\u7eed\u4e0eFDA\u4fdd\u6301\u79ef\u6781\u6c9f\u901a\uff0c\u63a8\u52a8PBGENE-DMD\u671d\u7740\u76d1\u7ba1\u91cc\u7a0b\u7891\u8fc8\u8fdb\uff0c\u9884\u8ba12026\u5e74\u5c06\u83b7\u5f97\u4e34\u5e8a\u6570\u636e\u3002\u201d<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u9605\u8bfb\u66f4\u591a\uff1a<\/strong> <a href=\"https:\/\/dmdwarrior.com\/zh\/precision-biosciences-highlights-new-preclinical-data-pbgene-dmd\/\">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<\/a><\/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\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 that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for PBGENE-DMD for the treatment of Duchenne muscular dystrophy (DMD). \u201cReceipt of Orphan [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":4169,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[88,195,314,313],"class_list":["post-4166","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-fda","tag-orphan-drug","tag-pbgene-dmd","tag-precision-biosciences"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/4166","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=4166"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/4166\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media\/4169"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media?parent=4166"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/categories?post=4166"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/tags?post=4166"}],"curies":[{"name":"\u53ef\u6e7f\u6027\u7c89\u5242","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}