{"id":4242,"date":"2025-07-29T10:23:25","date_gmt":"2025-07-29T07:23:25","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=4242"},"modified":"2025-07-30T13:54:29","modified_gmt":"2025-07-30T10:54:29","slug":"genethon-gnt0004-phase-3-pivotal-clinical-trial-begins","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/zh\/genethon-gnt0004-phase-3-pivotal-clinical-trial-begins\/","title":{"rendered":"Genethon \u5c06\u5728\u6b27\u6d32\u542f\u52a8 GNT0004 \u5173\u952e\u6027\u8bd5\u9a8c\uff0cGNT0004 \u662f\u4e00\u79cd\u7528\u4e8e\u6cbb\u7597\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684\u4f4e\u5242\u91cf\u5fae\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u57fa\u56e0\u7597\u6cd5"},"content":{"rendered":"<p class=\"wp-block-paragraph\"><strong>Genethon \u5df2\u83b7\u5f97\u76d1\u7ba1\u673a\u6784 MHRA \u548c EMA \u7684\u6279\u51c6\uff0c\u5c06\u5728\u6cd5\u56fd\u548c\u82f1\u56fd\u5f00\u59cb\u5176\u57fa\u56e0\u7597\u6cd5 GNT0004 \u6cbb\u7597\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD) \u7684\u5173\u952e\u6027 3 \u671f\u4e34\u5e8a\u8bd5\u9a8c\u3002<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Genethon\u9996\u5e2d\u6267\u884c\u5b98Frederic Revah\u8868\u793a\uff1a\u201c\u6211\u4eec\u5f88\u9ad8\u5174\u80fd\u591f\u7ee7\u7eed\u8fd9\u4e9b\u8bd5\u9a8c\uff0c\u5e76\u51b3\u5fc3\u5c06GNT0004\u63a8\u5411\u5e02\u573a\uff0c\u9020\u798f\u90a3\u4e9b\u6b63\u5728\u7b49\u5f85\u6cbb\u7597\u65b9\u6848\u7684\u5e74\u8f7b\u60a3\u8005\u53ca\u5176\u5bb6\u4eba\u3002\u8fd9\u6807\u5fd7\u7740\u6211\u4eec\u9488\u5bf9\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\uff08DMD\uff09\u7684\u57fa\u56e0\u6cbb\u7597\u9879\u76ee\u8fc8\u51fa\u4e86\u51b3\u5b9a\u6027\u7684\u4e00\u6b65\u3002\u8be5\u9879\u76ee\u4e8e2021\u5e74\u542f\u52a8\uff0c\u5e76\u5728\u6211\u4eec1\/2\/3\u671f\u4e34\u5e8a\u8bd5\u9a8c\u76841\/2\u671f\u90e8\u5206\u4e2d\uff0c\u5728\u9996\u6279\u63a5\u53d7\u6cbb\u7597\u7684\u513f\u7ae5\u8eab\u4e0a\u5c55\u73b0\u51fa\u4e86\u6781\u5176\u4ee4\u4eba\u6b23\u559c\u7684\u7ed3\u679c\u3002\u201d<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Genethon GNT0004 3\u671f\u5173\u952e\u8bd5\u9a8c<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u8fd9\u9879\u53cc\u76f2\u8bd5\u9a8c\u5c06\u4e8e 8 \u6708\u548c 9 \u6708\u5728\u82f1\u56fd\u548c\u6cd5\u56fd\u542f\u52a8\uff0c\u5e76\u5c06\u62db\u52df 64 \u540d 6 \u81f3 10 \u5c81\u60a3\u6709 DMD \u4f46\u4fdd\u7559\u4e86\u884c\u8d70\u80fd\u529b\u7684\u7537\u5b69\u3002<\/li>\n<\/ul>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u7b2c 3 \u9636\u6bb5\u8bd5\u9a8c\u7684\u6279\u51c6\u57fa\u4e8e\u7b2c 1\/2 \u9636\u6bb5\u7684\u7ed3\u679c\uff0c\u8be5\u7ed3\u679c\u8bc1\u660e\u4e86 3\u00d710\u00b9\u00b3 vg\/kg \u5242\u91cf\u5fae\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u5b89\u5168\u6027\u548c\u6709\u6548\u6027\uff0c\u8be5\u5242\u91cf\u4f4e\u4e8e\u5176\u4ed6\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD) \u57fa\u56e0\u7597\u6cd5\u4e2d\u4f7f\u7528\u7684\u5242\u91cf\u3002<\/li>\n<\/ul>\n\n\n\n<h2 class=\"wp-block-heading\">CPK \u663e\u8457\u6301\u7eed\u964d\u4f4e<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u6b27\u6d32\u76843\u671f\u4e34\u5e8a\u8bd5\u9a8c\u6388\u6743\u57fa\u4e8e1\/2\u671f\u4e34\u5e8a\u8bd5\u9a8c\u7684\u7ed3\u679c\uff0c\u8be5\u7814\u7a76\u663e\u793aGNT0004\u5177\u6709\u826f\u597d\u7684\u8010\u53d7\u6027\uff0c\u5e76\u5728\u5fae\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u8868\u8fbe\u3001\u808c\u9178\u78f7\u9178\u6fc0\u9176(CPK)\u964d\u4f4e\u548c\u8fd0\u52a8\u529f\u80fd\u65b9\u9762\u8868\u73b0\u51fa\u7597\u6548\u3002\u60a3\u8005\u8fd0\u52a8\u529f\u80fd\u5f97\u5230\u957f\u671f\u6539\u5584\u6216\u7a33\u5b9a\uff0c\u5e76\u4e14CPK\uff08\u808c\u8089\u635f\u4f24\u7684\u5173\u952e\u6807\u5fd7\u7269\uff09\u663e\u8457\u6301\u7eed\u964d\u4f4e\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u4e86\u89e3\u66f4\u591a\uff1a <a href=\"https:\/\/dmdwarrior.com\/zh\/genethon-reports-results-of-gnt0004-gene-therapy-clinical-trial-permanent-decrease-in-ck-levels\/\">CK\u6c34\u5e73\u6c38\u4e45\u6027\u964d\u4f4e\u8d85\u8fc775%<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u8fd9\u9879\u4e09\u671f\u53cc\u76f2\u8bd5\u9a8c\u5c06\u4e8e8\u6708\u548c9\u6708\u5728\u82f1\u56fd\u548c\u6cd5\u56fd\u542f\u52a8\uff0c\u91c7\u7528\u5355\u6b21\u9759\u8109\u6ce8\u5c04GNT0004\u3002\u8be5\u8bd5\u9a8c\u5305\u542b\u4f18\u5316\u7684hMD1\u8f6c\u57fa\u56e0\uff0c\u5373\u7f16\u7801\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u57fa\u56e0\uff08\u957f\u5ea6\u7f29\u77ed\uff083\u00d710\u00b9\u00b3 vg\/kg\u5fae\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\uff09\u4f46\u529f\u80fd\u6b63\u5e38\u7684\u7248\u672c\uff09\uff0c\u8be5\u57fa\u56e0\u4f4d\u4e8eAAV8\u8f7d\u4f53\u4e2d\uff0c\u4e0e\u77ac\u65f6\u514d\u75ab\u9884\u9632\u6cbb\u7597\u76f8\u5173\u3002\u8be5\u8f7d\u4f53\u8bbe\u8ba1\u7528\u4e8e\u5728\u808c\u8089\u7ec4\u7ec7\u548c\u5fc3\u810f\u4e2d\u8868\u8fbe\uff0c\u8fd9\u5f97\u76ca\u4e8e\u9488\u5bf9\u8fd9\u4e9b\u7ec4\u7ec7\u7279\u5f02\u7684Spc5-12\u542f\u52a8\u5b50\u5e8f\u5217\u3002\u8bd5\u9a8c\u5c06\u62db\u52df64\u540d6\u81f310\u5c81\u60a3\u6709\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\uff08DMD\uff09\u4e14\u4fdd\u7559\u4e86\u884c\u8d70\u80fd\u529b\u7684\u7537\u5b69\u3002<\/p>\n\n\n\n<figure class=\"wp-block-embed is-type-video is-provider-youtube wp-block-embed-youtube wp-embed-aspect-16-9 wp-has-aspect-ratio\"><div class=\"wp-block-embed__wrapper\">\n<div class=\"youtube-embed\" data-video_id=\"T0zZN7c2cSA\"><iframe loading=\"lazy\" title=\"\u675c\u6c0f\u808c\u75c5\uff1a\u4e00\u79cd\u72ec\u7279\u7684\u6cbb\u7597\u65b9\u6cd5| \u8bba\u6587AFM\u7535\u89c6\u9a6c\u62c9\u677e\" width=\"696\" height=\"392\" src=\"https:\/\/www.youtube.com\/embed\/T0zZN7c2cSA?feature=oembed&#038;enablejsapi=1\" frameborder=\"0\" allow=\"accelerometer; autoplay; clipboard-write; encrypted-media; gyroscope; picture-in-picture; web-share\" referrerpolicy=\"strict-origin-when-cross-origin\" allowfullscreen><\/iframe><\/div>\n<\/div><\/figure>\n\n\n\n<p class=\"wp-block-paragraph\">9\u5c81\u7684\u8428\u6c99\u662f\u63a5\u53d7G\u00e9n\u00e9thon\u516c\u53f8\u7814\u53d1\u7684\u5fae\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7597\u6cd5\u6cbb\u7597\u7684\u513f\u7ae5\u4e4b\u4e00\uff0c\u8be5\u7597\u6cd5\u662f\u8be5\u9879\u4e34\u5e8a\u8bd5\u9a8c\u7684\u7b2c\u4e00\u9636\u6bb5\u7684\u4e00\u90e8\u5206\u3002\u8be5\u7597\u6cd5\u65e8\u5728\u6062\u590d\u808c\u8089\u4e2d\u7f3a\u5931\u7684\u86cb\u767d\u8d28\u7684\u751f\u6210\u3002\u5982\u4eca\uff0c\u8428\u6c99\u53ef\u4ee5\u5954\u8dd1\u3001\u6500\u722c\u3001\u8df3\u8dc3\u2026\u2026\u5982\u679c\u6ca1\u6709\u6cbb\u7597\uff0c\u4ed6\u6839\u672c\u65e0\u6cd5\u5b8c\u6210\u8fd9\u4e9b\u52a8\u4f5c\uff01 <a href=\"https:\/\/www.afm-telethon.fr\/fr\/evenement-telethon\/le-combat-du-telethon\/les-familles-ambassadrices\/sacha-la-maladie-detruit-mes\" target=\"_blank\" rel=\"noopener\">\u9605\u8bfb\u66f4\u591a<\/a>.<\/p>","protected":false},"excerpt":{"rendered":"<p>Genethon has received approvals from regulatory authorities, MHRA and EMA, to begin pivotal Phase 3 clinical trials in France and the UK of its gene therapy, GNT0004, for Duchenne muscular dystrophy (DMD). Genethon CEO Frederic Revah observed, \u201cWe are delighted to be able to continue these trials and are determined to bring GNT0004 to market [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":4246,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[27],"tags":[137,342,105,106,220],"class_list":["post-4242","post","type-post","status-publish","format-standard","has-post-thumbnail","category-research","tag-clinical-trial","tag-frederic-revah","tag-genethon","tag-gnt-0004","tag-gnt0004"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/4242","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=4242"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/4242\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media\/4246"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media?parent=4242"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/categories?post=4242"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/tags?post=4242"}],"curies":[{"name":"\u53ef\u6e7f\u6027\u7c89\u5242","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}