{"id":8865,"date":"2026-10-14T15:15:01","date_gmt":"2026-10-14T12:15:01","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=8865"},"modified":"2026-10-14T15:28:15","modified_gmt":"2026-10-14T12:28:15","slug":"entr-601-45-dystrophin-production-duchenne","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/zh\/entr-601-45-dystrophin-production-duchenne\/","title":{"rendered":"Entrada Therapeutics ENTR-601-45\uff1a\u65e9\u671f\u5916\u663e\u5b5045\u8df3\u8dc3\u7814\u7a76\u8868\u660e\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u4ea7\u91cf\u589e\u52a0"},"content":{"rendered":"<p class=\"wp-block-paragraph\"><strong>Entrada Therapeutics \u516c\u53f8\u5ba3\u5e03\u5176\u5916\u663e\u5b50 45 \u8df3\u8dc3\u7814\u7a76 ELEVATE-45-201 \u53d6\u5f97\u4e86\u79ef\u6781\u7684\u521d\u6b65\u7ed3\u679c\uff0c\u8be5\u7814\u7a76\u8bc4\u4f30\u4e86 ENTR-601-45 \u5bf9\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (Duchenne) \u60a3\u8005\u7684\u7597\u6548\u3002.<\/strong> \u7b2c\u4e00\u6279\u53d7\u8bd5\u8005\u5728\u63a5\u53d7\u521d\u59cb\u5242\u91cf\u540e\u8868\u73b0\u51fa\u826f\u597d\u7684\u5b89\u5168\u6027\uff0c\u4e14\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u4ea7\u751f\u91cf\u7565\u6709\u589e\u52a0\u3002\u8fd9\u4e9b\u53d1\u73b0\u662f\u8bc4\u4f30\u8be5\u7597\u6cd5\u7684\u65e9\u671f\u6b65\u9aa4\uff0c\u540e\u7eed\u8ba1\u5212\u4f7f\u7528\u66f4\u9ad8\u5242\u91cf\uff0c\u4ee5\u8bc4\u4f30\u5176\u662f\u5426\u80fd\u4ea7\u751f\u66f4\u591a\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>1\/2 \u671f\u7814\u7a76\u6b63\u5728\u8bc4\u4f30 ENTR-601-45 \u5728\u53ef\u884c\u8d70\u7684\u3001\u5df2\u786e\u8bca\u60a3\u6709 DMD \u57fa\u56e0\u7a81\u53d8\u4e14\u9002\u5408\u8fdb\u884c\u5916\u663e\u5b50 45 \u8df3\u8dc3\u7684\u53c2\u4e0e\u8005\u4e2d\u7684\u7597\u6548\u3002.<\/strong><\/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=\"#what-did-the-first-cohort-show\">\u7b2c\u4e00\u6279\u5b66\u5458\u5c55\u73b0\u4e86\u4ec0\u4e48\uff1f<\/a><ul><li><a href=\"#dystrophin-production-increased-by-0-10\">\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u4ea7\u91cf\u589e\u52a0\u4e86 0.10%<\/a><\/li><\/ul><\/li><li><a href=\"#safety-and-tolerability-results\">\u5b89\u5168\u6027\u548c\u8010\u53d7\u6027\u7ed3\u679c<\/a><ul><li><a href=\"#drug-exposure-matched-expectations\">\u836f\u7269\u66b4\u9732\u91cf\u7b26\u5408\u9884\u671f<\/a><\/li><\/ul><\/li><li><a href=\"#what-comes-next-for-entr-601-45\">ENTR-601-45\u7684\u4e0b\u4e00\u6b65\u53d1\u5c55\u662f\u4ec0\u4e48\uff1f<\/a><\/li><li><a href=\"#a-potentially-less-frequent-dosing-schedule\">\u53ef\u80fd\u51cf\u5c11\u7ed9\u836f\u9891\u7387\u7684\u65b9\u6848<\/a><\/li><li><a href=\"#conclusion\">\u7ed3\u8bba<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<h2 id=\"what-did-the-first-cohort-show\" class=\"wp-block-heading\">\u7b2c\u4e00\u6279\u5b66\u5458\u5c55\u73b0\u4e86\u4ec0\u4e48\uff1f<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u7b2c\u4e00\u7ec4\u5305\u62ec\u516b\u540d\u53c2\u4e0e\u8005\uff1a\u516d\u540d\u63a5\u53d7\u4e86 ENTR-601-45\uff0c\u5242\u91cf\u4e3a 5 mg\/kg\uff0c\u800c\u4e24\u540d\u63a5\u53d7\u4e86\u5b89\u6170\u5242\u3002.<\/strong> \u5728\u7814\u7a76\u7684\u591a\u5242\u91cf\u9012\u589e\u9636\u6bb5\uff0c\u53c2\u4e0e\u8005\u63a5\u53d7\u4e86\u4e09\u5242\u836f\u7269\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u7814\u7a76\u7ed3\u679c\u8fbe\u5230\u4e86\u5b89\u5168\u6027\u548c\u8010\u53d7\u6027\u7684\u4e3b\u8981\u76ee\u6807\u3002\u6b21\u8981\u76ee\u6807\u4e5f\u663e\u793a\uff0c\u63a5\u53d7\u6cbb\u7597\u7684\u53d7\u8bd5\u8005\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u4ea7\u751f\u548c\u5916\u663e\u5b50\u8df3\u8dc3\u60c5\u51b5\u53d1\u751f\u4e86\u53d8\u5316\u3002.<\/p>\n\n\n\n<h3 id=\"dystrophin-production-increased-by-0-10\" class=\"wp-block-heading\">\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u4ea7\u91cf\u589e\u52a0\u4e86 0.10%<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u6700\u91cd\u8981\u7684\u53d1\u73b0\u662f\uff0c\u63a5\u53d7 ENTR-601-45 \u6cbb\u7597\u7684 6 \u540d\u53c2\u4e0e\u8005\u4e2d\uff0c\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u4ea7\u751f\u91cf\u6bd4\u57fa\u7ebf\u5e73\u5747\u589e\u52a0\u4e86 0.10%\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u76f8\u6bd4\u4e4b\u4e0b\uff0c\u5b89\u6170\u5242\u7ec4\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u5e73\u5747\u6bd4\u57fa\u7ebf\u6c34\u5e73\u4e0b\u964d\u4e86 0.02%\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u4e0e\u5b89\u6170\u5242\u7ec4\u5916\u663e\u5b50\u8df3\u8dc3\u5e73\u5747\u51cf\u5c11 0.04% \u76f8\u6bd4\uff0c\u6cbb\u7597\u7ec4\u5916\u663e\u5b50\u8df3\u8dc3\u5e73\u5747\u589e\u52a0 0.15%\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u8fd9\u4e9b\u53d1\u73b0\u521d\u6b65\u8868\u660e\uff0cENTR-601-45 \u80fd\u591f\u4ea7\u751f\u9884\u671f\u7684\u751f\u7269\u5b66\u6548\u5e94\u3002\u7136\u800c\uff0c\u5728\u521d\u59cb\u5242\u91cf\u4e0b\u89c2\u5bdf\u5230\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u4ea7\u91cf\u589e\u52a0\u5e45\u5ea6\u8f83\u5c0f\uff0c\u9700\u8981\u8fdb\u4e00\u6b65\u7814\u7a76\u4ee5\u4e86\u89e3\u66f4\u9ad8\u5242\u91cf\u4e0b\u7684\u5f71\u54cd\u3002.<\/p>\n\n\n\n<h2 id=\"safety-and-tolerability-results\" class=\"wp-block-heading\">\u5b89\u5168\u6027\u548c\u8010\u53d7\u6027\u7ed3\u679c<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">ENTR-601-45 \u5728\u521d\u59cb\u5242\u91cf 5 mg\/kg \u65f6\u8868\u73b0\u51fa\u826f\u597d\u7684\u5b89\u5168\u6027\u548c\u8010\u53d7\u6027\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u5934\u75db\u662f\u6700\u5e38\u89c1\u7684\u6cbb\u7597\u671f\u95f4\u51fa\u73b0\u7684\u4e0d\u826f\u4e8b\u4ef6\uff0c\u4e24\u540d\u53d7\u8bd5\u8005\u5171\u62a5\u544a\u4e86\u4e94\u6b21\u3002\u4e00\u540d\u53d7\u8bd5\u8005\u51fa\u73b0\u4e86\u4e24\u6b21\u4e25\u91cd\u7684\u6cbb\u7597\u671f\u95f4\u51fa\u73b0\u7684\u4e0d\u826f\u4e8b\u4ef6\uff1a\u5455\u5410\u548c\u53d1\u70ed\u3002\u8fd9\u4e24\u79cd\u4e0d\u826f\u4e8b\u4ef6\u968f\u540e\u5747\u5df2\u6d88\u9000\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u6ca1\u6709\u53c2\u4e0e\u8005\u9000\u51fa\u7814\u7a76\uff0c\u6240\u6709\u53c2\u4e0e\u8005\u90fd\u53c2\u52a0\u4e86\u5f00\u653e\u6807\u7b7e\u6269\u5c55\u7814\u7a76\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u8be5\u516c\u53f8\u8fd8\u62a5\u544a\u79f0\uff0c\u672a\u53d1\u73b0\u4e0e\u4f4e\u9541\u6c34\u5e73\u6216\u80be\u810f\u5b89\u5168\u6027\u76f8\u5173\u7684\u4efb\u4f55\u95ee\u9898\u3002\u63a5\u53d7\u6cbb\u7597\u7684\u53d7\u8bd5\u8005\u80be\u810f\u6307\u6807\uff0c\u5305\u62ec\u4f30\u7b97\u80be\u5c0f\u7403\u6ee4\u8fc7\u7387 (eGFR) \u548c\u80f1\u6291\u7d20 C\uff0c\u5747\u5728\u6b63\u5e38\u8303\u56f4\u5185\u3002\u9541\u6c34\u5e73\u4e5f\u5728\u6b63\u5e38\u8303\u56f4\u5185\u3002.<\/p>\n\n\n\n<h3 id=\"drug-exposure-matched-expectations\" class=\"wp-block-heading\">\u836f\u7269\u66b4\u9732\u91cf\u7b26\u5408\u9884\u671f<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u8840\u6db2\u4e2d ENTR-601-45 \u7684\u6d4b\u91cf\u6c34\u5e73\u4e0e\u57fa\u4e8e\u5148\u524d\u5bf9\u5e7c\u5e74\u975e\u4eba\u7075\u957f\u7c7b\u52a8\u7269\u7684\u7814\u7a76\u7684\u9884\u6d4b\u76f8\u4e00\u81f4\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u8be5\u516c\u53f8\u8868\u793a\uff0c\u8fd9\u4e9b\u7ed3\u679c\u652f\u6301\u4e86\u5176\u9884\u671f\uff0c\u5373\u66f4\u9ad8\u7684\u5242\u91cf\u53ef\u80fd\u4f1a\u4ea7\u751f\u66f4\u5927\u7684\u836f\u7269\u66b4\u9732\u91cf\u548c\u66f4\u591a\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u751f\u6210\u3002.<\/p>\n\n\n\n<h2 id=\"what-comes-next-for-entr-601-45\" class=\"wp-block-heading\">ENTR-601-45\u7684\u4e0b\u4e00\u6b65\u53d1\u5c55\u662f\u4ec0\u4e48\uff1f<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u8be5\u516c\u53f8\u5e0c\u671b\u901a\u8fc7\u66f4\u9ad8\u7684\u5242\u91cf\u6765\u786e\u5b9a ENTR-601-45 \u662f\u5426\u80fd\u66f4\u663e\u8457\u5730\u589e\u52a0\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u4ea7\u91cf\uff0c\u5e76\u6709\u53ef\u80fd\u5e26\u6765\u529f\u80fd\u6027\u76ca\u5904\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u7b2c\u4e8c\u7ec4\u53d7\u8bd5\u8005\u63a5\u53d710 mg\/kg\u7684\u5242\u91cf\u6cbb\u7597\uff0c\u76ee\u524d\u5df2\u5b8c\u6210\u5165\u7ec4\u3002Entrada Therapeutics\u9884\u8ba1\u5c06\u4e8e2027\u5e74\u4e0a\u534a\u5e74\u516c\u5e03\u8be5\u7ec4\u53d7\u8bd5\u8005\u7684\u6570\u636e\u3002.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u7b2c\u4e09\u7ec4\u8bd5\u9a8c\u8ba1\u5212\u8bc4\u4f30\u5242\u91cf\u9ad8\u8fbe 15 mg\/kg \u7684\u60c5\u51b5\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u7b2c\u4e00\u6279\u53c2\u4e0e\u8005\u4e5f\u5df2\u8fdb\u5165\u8be5\u7814\u7a76\u7684\u5f00\u653e\u6807\u7b7e 2 \u671f\u90e8\u5206\uff0c\u4ed6\u4eec\u5c06\u63a5\u53d7 6 \u5242 ENTR-601-45\uff0c\u5242\u91cf\u4e3a 5 mg\/kg\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u5728\u7b2c\u4e00\u7ec4\u53d7\u8bd5\u8005\u4e2d\uff0c\u4e09\u6b21\u7ed9\u836f\u540e\u672a\u89c2\u5bdf\u5230\u6216\u62a5\u544a\u4efb\u4f55\u53ef\u89c2\u5bdf\u5230\u7684\u529f\u80fd\u83b7\u76ca\u3002\u672a\u6765\u5c06\u5728\u66f4\u9ad8\u5242\u91cf\u548c\u66f4\u957f\u7597\u7a0b\u4e0b\uff0c\u901a\u8fc7\u540e\u7eed\u7684\u53d7\u8bd5\u8005\u961f\u5217\u7814\u7a76\u548c\u5f00\u653e\u6807\u7b7e\u7814\u7a76\u6765\u8bc4\u4f30\u8be5\u7597\u6cd5\u7684\u529f\u80fd\u5f71\u54cd\u3002\u4e86\u89e3\u66f4\u591a\uff1a <a href=\"https:\/\/dmdwarrior.com\/zh\/duchenne-exon-deletion-tool\/\" target=\"_blank\" rel=\"noopener\">DMDWarrior \u5916\u663e\u5b50\u5220\u9664\u5de5\u5177<\/a><\/p>\n\n\n\n<h2 id=\"a-potentially-less-frequent-dosing-schedule\" class=\"wp-block-heading\">\u53ef\u80fd\u51cf\u5c11\u7ed9\u836f\u9891\u7387\u7684\u65b9\u6848<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">ENTR-601-45\u7684\u8bbe\u8ba1\u7ed9\u836f\u65b9\u6848\u4e3a\u6bcf\u516d\u5468\u4e00\u6b21\u3002\u8fd9\u79cd\u7ed9\u836f\u65b9\u6848\u610f\u5473\u7740\u6bcf\u5e74\u5927\u7ea6\u9700\u8981\u7ed9\u836f\u4e5d\u6b21\uff0c\u800c\u6bcf\u5468\u4e00\u6b21\u7684\u7ed9\u836f\u65b9\u6848\u5219\u9700\u8981\u7ed9\u836f\u4e94\u5341\u591a\u6b21\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u51cf\u5c11\u7ed9\u836f\u9891\u7387\u7684\u53ef\u80fd\u6027\u662f\u8be5\u7814\u7a76\u7597\u6cd5\u7684\u4e00\u4e2a\u91cd\u8981\u65b9\u9762\u3002\u7136\u800c\uff0c\u5176\u957f\u671f\u5b89\u5168\u6027\u3001\u6709\u6548\u6027\u548c\u529f\u80fd\u6027\u83b7\u76ca\u4ecd\u9700\u901a\u8fc7\u8fdb\u4e00\u6b65\u7684\u4e34\u5e8a\u7814\u7a76\u6765\u786e\u5b9a\u3002.<\/p>\n\n\n\n<h2 id=\"conclusion\" class=\"wp-block-heading\">\u7ed3\u8bba<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">ELEVATE-45-201 \u7814\u7a76\u7684\u7b2c\u4e00\u6279\u53d7\u8bd5\u8005\u8fbe\u5230\u4e86\u5176\u521d\u6b65\u7684\u5b89\u5168\u6027\u548c\u8010\u53d7\u6027\u76ee\u6807\uff0c\u7ed3\u679c\u663e\u793a\uff0c\u5728 5 mg\/kg \u5242\u91cf\u4e0b\uff0cENTR-601-45 \u53ef\u4f7f\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u4ea7\u751f\u5e73\u5747\u589e\u52a0 0.10%\u3002\u5916\u663e\u5b50\u8df3\u8dc3\u4e5f\u6bd4\u57fa\u7ebf\u6c34\u5e73\u6709\u6240\u589e\u52a0\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u8fd9\u4e9b\u662f\u65e9\u671f\u751f\u7269\u5b66\u53d1\u73b0\uff0c\u5e76\u975e\u5df2\u786e\u7acb\u7684\u4e34\u5e8a\u6216\u529f\u80fd\u83b7\u76ca\u7684\u8bc1\u636e\u3002\u4e0b\u4e00\u4e2a\u91cd\u8981\u7684\u91cc\u7a0b\u7891\u5c06\u662f\u66f4\u9ad8\u5242\u91cf\u7ec4\u7684\u7ed3\u679c\uff0c\u9996\u5148\u662f10 mg\/kg\u5242\u91cf\u7ec4\uff0c\u9884\u8ba1\u5c06\u4e8e2027\u5e74\u4e0a\u534a\u5e74\u516c\u5e03\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u4e86\u89e3\u66f4\u591a<\/strong>:&nbsp;<a href=\"https:\/\/dmdwarrior.com\/clinical-trials\/map\/\" target=\"_blank\" rel=\"noopener\">\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u4e34\u5e8a\u8bd5\u9a8c\u5730\u70b9\u5730\u56fe<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>Entrada Therapeutics has announced positive initial results from its Exon 45 Skipping Study, ELEVATE-45-201, evaluating ENTR-601-45 in people with Duchenne muscular dystrophy (Duchenne). The first group of participants showed a favorable safety profile and a small increase in dystrophin production after receiving the initial dose. The findings are an early step in evaluating the treatment, [&hellip;]<\/p>\n","protected":false},"author":6,"featured_media":8867,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[320,321,233,234,317,318,319],"class_list":["post-8865","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-elevate-45-201","tag-entr-601-45","tag-entrada","tag-entrada-therapeutics","tag-exon-45","tag-exon-45-skipping","tag-exon-45-skipping-therapy"],"subtitle":"Entrada Therapeutics\u62a5\u544a\u4e86ENTR-601-45\u5728\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u4e2d\u7684\u65e9\u671f\u7ed3\u679c\u3002ELEVATE-45-201\u7814\u7a76\u663e\u793a\uff0c\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u4ea7\u751f\u5e73\u5747\u589e\u52a0\u4e860.10%\uff0c\u8ba1\u5212\u4f7f\u7528\u66f4\u9ad8\u5242\u91cf\u4ee5\u63a2\u7d22\u66f4\u5927\u7684\u7597\u6548\u3002.","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/8865","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\/6"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/comments?post=8865"}],"version-history":[{"count":3,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/8865\/revisions"}],"predecessor-version":[{"id":8870,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/8865\/revisions\/8870"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media\/8867"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media?parent=8865"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/categories?post=8865"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/tags?post=8865"}],"curies":[{"name":"\u53ef\u6e7f\u6027\u7c89\u5242","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}