{"id":3883,"date":"2025-05-29T11:06:14","date_gmt":"2025-05-29T08:06:14","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=3883"},"modified":"2025-11-03T20:23:45","modified_gmt":"2025-11-03T17:23:45","slug":"pepgen-announces-it-will-halt-clinical-trials-of-exon-51-skipping-study","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/zh\/pepgen-announces-it-will-halt-clinical-trials-of-exon-51-skipping-study\/","title":{"rendered":"PepGen \u5ba3\u5e03\u5c06\u505c\u6b62\u5916\u663e\u5b50 51 \u8df3\u8dc3\u7814\u7a76\u7684\u4e34\u5e8a\u8bd5\u9a8c"},"content":{"rendered":"<p class=\"wp-block-paragraph\">PepGen\u662f\u4e00\u5bb6\u81f4\u529b\u4e8e\u5f00\u53d1\u4e0b\u4e00\u4ee3\u5be1\u6838\u82f7\u9178\u7597\u6cd5\u7684\u4e34\u5e8a\u9636\u6bb5\u751f\u7269\u6280\u672f\u516c\u53f8\uff0c\u8be5\u516c\u53f8\u4eca\u65e5\u5ba3\u5e03\uff0c\u57fa\u4e8e\u5176CONNECT1-EDO51\u7814\u7a76\u4e2d10\u6beb\u514b\/\u5343\u514b\u5242\u91cf\u7ec4\u4eba\u7fa4\u4e2d\u6d4b\u5f97\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u6c34\u5e73\uff0c\u8be5\u7814\u7a76\u65e8\u5728\u7814\u7a76PGN-EDO51\u57fa\u56e0\u5728\u53ef\u8fdb\u884c51\u53f7\u5916\u663e\u5b50\u8df3\u8dc3\u7597\u6cd5\u6cbb\u7597\u7684\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7(DMD)\u60a3\u8005\u4e2d\u7684\u4f5c\u7528\uff0c\u516c\u53f8\u5c06\u4e13\u6ce8\u4e8e\u63a8\u8fdb\u5176\u76ee\u524d\u5904\u4e8eII\u671f\u4e34\u5e8a\u5f00\u53d1\u7684\u3001\u524d\u666f\u5e7f\u9614\u76841\u578b\u5f3a\u76f4\u6027\u808c\u8425\u517b\u4e0d\u826f\u75c7(DM1)\u9879\u76ee\u3002\u516c\u53f8\u81ea\u613f\u7ec8\u6b62PGN-EDO51\u57fa\u56e0\u7684\u5f00\u53d1\uff0c\u5e76\u8ba1\u5212\u9010\u6b65\u505c\u6b62\u6240\u6709DMD\u76f8\u5173\u7684\u7814\u53d1\u6d3b\u52a8\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u5728CONNECT1\u7814\u7a76\u768410 mg\/kg\u7ec4\uff08n=4\uff09\u4e2d\uff0cPGN-EDO51\u4f7f\u5916\u663e\u5b5051\u8df3\u8dc3\u8f6c\u5f55\u672c\u589e\u52a0\u81f34.26%\uff08\u5e73\u5747\u589e\u52a03.5%\uff09\uff1b\u7136\u800c\uff0c\u603b\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u4ec5\u589e\u52a0\u81f3\u6b63\u5e38\u6c34\u5e73\u76840.59%\uff08\u5e73\u5747\u589e\u52a00.36%\uff09\u3002PGN-EDO51\u7684\u5b89\u5168\u6027\u603b\u4f53\u4e0a\u4ecd\u7136\u826f\u597d\uff0c\u6240\u6709\u6cbb\u7597\u76f8\u5173\u4e0d\u826f\u4e8b\u4ef6\u5747\u4e3a\u8f7b\u5fae\u3002\u7814\u7a76\u4e2d\u672a\u62a5\u544a\u4e25\u91cd\u4e0d\u826f\u4e8b\u4ef6\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u9605\u8bfb\u66f4\u591a\uff1a<\/strong> <a href=\"https:\/\/dmdwarrior.com\/zh\/next-generation-exon-skipping-therapies-for-duchenne-muscular-dystrophy\/\">\u4e3a\u6cbb\u7597\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u800c\u5f00\u53d1\u7684\u4e0b\u4e00\u4ee3\u5916\u663e\u5b50\u8df3\u8dc3\u7597\u6cd5<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">PepGen \u603b\u88c1\u517c\u9996\u5e2d\u6267\u884c\u5b98 James McArthur \u535a\u58eb\u8868\u793a\uff1a\u201c\u6211\u4eec\u5bf9 CONNECT1 10 mg\/kg \u5242\u91cf\u7ec4\u89c2\u5bdf\u5230\u7684\u6297\u808c\u840e\u7f29\u86cb\u767d\u7ed3\u679c\u611f\u5230\u5931\u671b\uff0c\u56e0\u4e3a\u6211\u4eec\u539f\u672c\u5e0c\u671b\u80fd\u591f\u66f4\u6df1\u5165\u5730\u6539\u8fdb\u73b0\u6709\u7684\u60a3\u8005\u7597\u6cd5\u3002\u503c\u6b64 DMD \u9879\u76ee\u5373\u5c06\u7ed3\u675f\u4e4b\u9645\uff0c\u6211\u4eec\u8981\u611f\u8c22\u60a3\u8005\u3001\u5bb6\u5c5e\u3001\u62a4\u7406\u4eba\u5458\u3001\u7814\u7a76\u4eba\u5458\u548c\u7814\u7a76\u4eba\u5458\u5bf9\u672c\u7814\u7a76\u7684\u652f\u6301\u548c\u53c2\u4e0e\u3002\u6211\u8fd8\u8981\u611f\u8c22\u6211\u4eec\u56e2\u961f\u7684\u8f9b\u52e4\u5de5\u4f5c\u548c\u81f4\u529b\u4e8e\u4e3a DMD \u60a3\u8005\u5f00\u53d1\u65b0\u7684\u6f5c\u5728\u7597\u6cd5\u7684\u627f\u8bfa\u3002\u201d<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u9605\u8bfb\u66f4\u591a<\/strong>:\u00a0<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>PepGen, a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies, today announced that based on the levels of dystrophin protein measured in the 10 mg\/kg cohort of its CONNECT1-EDO51 study investigating PGN-EDO51 in Duchenne muscular dystrophy (DMD) patients amenable to exon 51 skipping, the Company will focus on advancing its promising myotonic dystrophy [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":3889,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[242,275,122,193,194,371,273,274],"class_list":["post-3883","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-clinical-trials","tag-connect1-edo51","tag-exon-51","tag-exon-51-skipping","tag-exon-51-treatment","tag-freedom-dm1","tag-pepgen","tag-pgn-edo51"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/3883","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=3883"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/3883\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media\/3889"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media?parent=3883"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/categories?post=3883"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/tags?post=3883"}],"curies":[{"name":"\u53ef\u6e7f\u6027\u7c89\u5242","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}