{"id":8838,"date":"2026-10-03T11:28:47","date_gmt":"2026-10-03T08:28:47","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=8838"},"modified":"2026-10-03T12:06:54","modified_gmt":"2026-10-03T09:06:54","slug":"wms-2026-entr-601-44-exon-44-skipping","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/zh\/wms-2026-entr-601-44-exon-44-skipping\/","title":{"rendered":"Entrada Therapeutics&#039; ENTR-601-44 \u5728\u65e9\u671f\u836f\u4ee3\u52a8\u529b\u5b66\/\u836f\u6548\u5b66\u6570\u636e\u4e2d\u663e\u793a\u51fa\u5242\u91cf\u4f9d\u8d56\u6027\u7684\u5916\u663e\u5b50 44 \u8df3\u8dc3"},"content":{"rendered":"<p class=\"wp-block-paragraph\"><strong>Entrada Therapeutics&#039; ENTR-601-44 \u662f\u4e00\u79cd\u7814\u7a76\u6027\u7597\u6cd5\uff0c\u65e8\u5728\u6cbb\u7597\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD) \u60a3\u8005\uff0c\u5176\u7a81\u53d8\u9002\u5408\u8fdb\u884c\u5916\u663e\u5b50 44 \u8df3\u8dc3\u6cbb\u7597\u3002.<\/strong> \u5728\u65e5\u672c\u5e7f\u5c9b\u4e3e\u884c\u7684\u7b2c 31 \u5c4a\u4e16\u754c\u808c\u8089\u534f\u4f1a\u5e74\u4f1a\u4e0a\u516c\u5e03\u7684\u65b0\u6570\u636e\uff0c\u7814\u7a76\u4e86 ENTR-601-44 \u5728\u4f53\u5185\u7684\u884c\u4e3a\u65b9\u5f0f\u53ca\u5176\u66b4\u9732\u4e0e\u5916\u663e\u5b50 44 \u8df3\u8dc3\u7684\u5173\u7cfb\u3002.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">ENTR-601-44\u662f\u4ec0\u4e48\uff1f<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>ENTR-601-44 \u662f\u5185\u4f53\u9003\u9038\u8f7d\u4f53-\u78f7\u9170\u4e8c\u80fa\u5417\u5549\u5be1\u805a\u4f53 (EEV-PMO) \u7f00\u5408\u7269\u3002.<\/strong> \u5b83\u65e8\u5728\u6539\u5584 PMO \u5411\u808c\u8089\u7ec6\u80de\u7684\u8f93\u9001\uff0c\u5e76\u6700\u7ec8\u5230\u8fbe\u7ec6\u80de\u6838\uff0c\u5728\u90a3\u91cc\u9776\u5411\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u524d\u4f53\u4fe1\u4f7f RNA\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u8be5\u7597\u6cd5\u7684\u76ee\u7684\u662f\u4f7f\u7b2c 44 \u53f7\u5916\u663e\u5b50\u8df3\u8dc3\uff0c\u4ece\u800c\u4ea7\u751f\u63a5\u8fd1\u5168\u957f\u4e14\u53ef\u80fd\u5177\u6709\u529f\u80fd\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u3002.<\/strong> \u65e9\u671f\u7684\u975e\u4e34\u5e8a\u7814\u7a76\u8868\u660e\uff0c\u60a3\u8005\u6765\u6e90\u7684\u7ec6\u80de\u4e2d\u5b58\u5728\u5916\u663e\u5b50 44 \u8df3\u8dc3\uff0cDMD \u5c0f\u9f20\u6a21\u578b\u4e2d\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u4ea7\u91cf\u589e\u52a0\uff0c\u4ee5\u53ca\u975e\u4eba\u7075\u957f\u7c7b\u52a8\u7269\u4e2d\u5b58\u5728\u5916\u663e\u5b50 44 \u8df3\u8dc3\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">WMS 2026 \u6d77\u62a5\u91cd\u70b9\u5173\u6ce8\u6765\u81ea\u975e\u4eba\u7075\u957f\u7c7b\u52a8\u7269\u3001\u5065\u5eb7\u6210\u5e74\u5fd7\u613f\u8005\u548c\u60a3\u6709\u5916\u663e\u5b50 44 \u8df3\u8dc3\u6cbb\u7597\u7684 DMD \u7684\u513f\u79d1\u53c2\u4e0e\u8005\u7684\u836f\u4ee3\u52a8\u529b\u5b66\u548c\u836f\u6548\u5b66\u6570\u636e\u3002. <strong>\u4e86\u89e3\u66f4\u591a<\/strong>:&nbsp;<a href=\"https:\/\/dmdwarrior.com\/zh\/mutations-and-deletions-amenable-to-exon-44-skipping-therapies-dmd\/\" target=\"_blank\" rel=\"noopener\">\u9002\u5408\u5916\u663e\u5b5044\u8df3\u8dc3\u7597\u6cd5\u7684\u7a81\u53d8\u548c\u7f3a\u5931<\/a><\/p>\n\n\n\n<h2 class=\"wp-block-heading\">ENTR-601-44\u66b4\u9732\u91cf\u968f\u5242\u91cf\u589e\u52a0\u800c\u589e\u52a0<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u6570\u636e\u663e\u793a\uff0c\u5728\u975e\u4eba\u7075\u957f\u7c7b\u52a8\u7269\u548c\u5065\u5eb7\u5fd7\u613f\u8005\u4e2d\uff0c\u968f\u7740\u5242\u91cf\u7684\u589e\u52a0\uff0c\u8840\u6d46\u4e2d ENTR-601-44 \u7684\u66b4\u9732\u91cf\u4e5f\u968f\u4e4b\u589e\u52a0\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u5728\u6309\u4f53\u91cd\u8c03\u6574\u540e\u7684\u5242\u91cf\u4e0b\uff0c\u6210\u5e74\u5065\u5eb7\u5fd7\u613f\u8005\u7684\u66b4\u9732\u91cf\u4f3c\u4e4e\u6bd4\u6210\u5e74\u975e\u4eba\u7075\u957f\u7c7b\u52a8\u7269\u9ad8\u51fa\u7ea6\u4e24\u5230\u4e09\u500d\u3002.<\/strong> \u5bf9\u60a3\u6709 DMD \u7684\u53c2\u4e0e\u8005\u8fdb\u884c\u91cd\u590d\u7ed9\u836f\uff0c\u672a\u53d1\u73b0\u8840\u6d46\u4e2d ENTR-601-44 \u79ef\u7d2f\u7684\u8bc1\u636e\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u8be5\u836f\u7269\u672c\u8eab\u4ece\u8840\u6d46\u4e2d\u6e05\u9664\u901f\u5ea6\u76f8\u5bf9\u8f83\u5feb\uff0c\u636e\u62a5\u9053\u5176\u534a\u8870\u671f\u7ea6\u4e3a2-4\u5c0f\u65f6\u3002\u7136\u800c\uff0c\u6700\u7ec8\u7684\u542bPMO\u7684\u4ee3\u8c22\u7269\u5728\u8840\u6d46\u4e2d\u505c\u7559\u7684\u65f6\u95f4\u8981\u957f\u5f97\u591a\uff0c\u534a\u8870\u671f\u8d85\u8fc7100\u5c0f\u65f6\u3002.<\/strong><\/p>\n\n\n\n<h2 class=\"wp-block-heading\">\u513f\u7ae5\u53c2\u4e0e\u8005\u4e0e\u6210\u4eba\u53c2\u4e0e\u8005\u4e4b\u95f4\u7684\u5dee\u5f02<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u8be5\u6d77\u62a5\u8fd8\u6307\u51fa\uff0c\u5728\u7ed9\u4e88 6 mg\/kg \u5242\u91cf\u540e\uff0c\u60a3\u6709 DMD \u7684\u513f\u79d1\u53c2\u4e0e\u8005\u4e0e\u5065\u5eb7\u6210\u5e74\u5fd7\u613f\u8005\u4e4b\u95f4\u5b58\u5728\u5dee\u5f02\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u5728\u513f\u79d1\u53d7\u8bd5\u8005\u4e2d\uff0c\u8840\u6d46\u4e2d\u836f\u7269\u7684\u6700\u5927\u6d53\u5ea6\u6bd4\u6210\u5e74\u5065\u5eb7\u5fd7\u613f\u8005\u4f4e\u7ea6 43%\uff0c\u800c\u53cd\u6620\u836f\u7269\u968f\u65f6\u95f4\u603b\u4f53\u66b4\u9732\u91cf\u7684\u6d53\u5ea6-\u65f6\u95f4\u66f2\u7ebf\u4e0b\u9762\u79ef\u6bd4\u6210\u5e74\u5065\u5eb7\u5fd7\u613f\u8005\u4f4e\u7ea6 58%\u3002.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u5728\u975e\u4eba\u7075\u957f\u7c7b\u52a8\u7269\u7814\u7a76\u4e2d\u4e5f\u89c2\u5bdf\u5230\u4e86\u5e7c\u5e74\u52a8\u7269\u548c\u6210\u5e74\u52a8\u7269\u4e4b\u95f4\u7684\u7c7b\u4f3c\u5dee\u5f02\u3002.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">ENTR-601-44 \u5230\u8fbe\u9aa8\u9abc\u808c<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u5176\u4e2d\u4e00\u9879\u5173\u952e\u53d1\u73b0\u662f\u5728\u9aa8\u9abc\u808c\u4e2d\u68c0\u6d4b\u5230\u4e86\u6700\u7ec8\u7684\u542b PMO \u7684\u4ee3\u8c22\u7269\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u5728\u5065\u5eb7\u5fd7\u613f\u8005\u548c\u975e\u4eba\u7075\u957f\u7c7b\u52a8\u7269\u4e2d\u5747\u89c2\u5bdf\u5230\u4e86\u5242\u91cf\u4f9d\u8d56\u6027\u6d53\u5ea6\u3002. <strong>\u5728\u5242\u91cf\u76f8\u5f53\u7684\u60c5\u51b5\u4e0b\uff0c\u5065\u5eb7\u5fd7\u613f\u8005\u7684\u808c\u8089\u66b4\u9732\u91cf\u4f3c\u4e4e\u6bd4\u975e\u4eba\u7075\u957f\u7c7b\u52a8\u7269\u9ad8\u51fa\u7ea6\u4e09\u500d\u3002.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u91cd\u8981\u7684\u662f\uff0c\u5728\u60a3\u6709 DMD \u7684\u53c2\u4e0e\u8005\u4e2d\uff0c\u7b2c\u4e09\u6b21\u7ed9\u836f\u540e\u516d\u5468\uff0c\u808c\u8089\u7ec4\u7ec7\u4e2d\u4ecd\u7136\u53ef\u4ee5\u68c0\u6d4b\u5230\u6700\u7ec8\u7684\u542b PMO \u7684\u4ee3\u8c22\u7269\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u5728\u5065\u5eb7\u5fd7\u613f\u8005\u548c\u975e\u4eba\u7075\u957f\u7c7b\u52a8\u7269\u7684\u5c3f\u6db2\u4e2d\u4e5f\u68c0\u6d4b\u5230\u4e86\u8be5\u4ee3\u8c22\u7269\u3002\u5728 6 mg\/kg \u5242\u91cf\u4e0b\uff0c\u5065\u5eb7\u5fd7\u613f\u8005\u6216 DMD \u60a3\u8005\u5747\u672a\u62a5\u544a\u4e0e\u80be\u529f\u80fd\u76f8\u5173\u7684\u5177\u6709\u4e34\u5e8a\u610f\u4e49\u7684\u5f02\u5e38\u7ed3\u679c\u3002. <strong>\u4e86\u89e3\u66f4\u591a<\/strong>: <a href=\"https:\/\/dmdwarrior.com\/zh\/upcoming-exon-44-skipping-therapies-for-dmd\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u5373\u5c06\u63a8\u51fa\u7684\u7528\u4e8e\u6cbb\u7597\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684Exon 44\u8df3\u8dc3\u7597\u6cd5<\/a><\/p>\n\n\n\n<h2 class=\"wp-block-heading\">\u66f4\u9ad8\u7684\u66b4\u9732\u6c34\u5e73\u53ef\u80fd\u5bfc\u81f4\u66f4\u591a\u7684\u5916\u663e\u5b50\u8df3\u8dc3<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u5728\u5e7c\u5e74\u975e\u4eba\u7075\u957f\u7c7b\u52a8\u7269\u4e2d\uff0c\u5916\u663e\u5b5044\u8df3\u8dc3\u7684\u53d1\u751f\u7387\u968fENTR-601-44\u66b4\u9732\u91cf\u7684\u589e\u52a0\u800c\u589e\u52a0\u3002\u8fd9\u79cd\u589e\u52a0\u5e76\u975e\u7ebf\u6027\u5173\u7cfb\uff0c\u5728\u6700\u9ad8\u6d4b\u8bd5\u5242\u91cf\u4e0b\u89c2\u5bdf\u5230\u5c24\u4e3a\u663e\u8457\u7684\u5f71\u54cd\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u6839\u636e\u8fd9\u4e9b\u53d1\u73b0\uff0c\u7814\u7a76\u4eba\u5458\u8ba4\u4e3a\uff0c\u5728\u6b63\u5728\u8fdb\u884c\u7684 ELEVATE-44-201 \u7814\u7a76\u7684\u540e\u7eed\u961f\u5217\u4e2d\uff0c\u5242\u91cf\u4e3a 12 mg\/kg \u6216\u66f4\u9ad8\u65f6\u9884\u671f\u7684\u8f83\u9ad8\u66b4\u9732\u91cf\u53ef\u80fd\u4f1a\u4ea7\u751f\u66f4\u5927\u7684\u836f\u6548\u5b66\u6548\u5e94\u3002.<\/strong><\/p>\n\n\n\n<h2 class=\"wp-block-heading\">\u6570\u636e\u663e\u793a\u4ec0\u4e48<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">WMS 2026 \u6d77\u62a5\u63d0\u4f9b\u4e86\u65e9\u671f\u836f\u4ee3\u52a8\u529b\u5b66\u548c\u836f\u6548\u5b66\u8bc1\u636e\uff0c\u8868\u660e ENTR-601-44 \u53ef\u4ea7\u751f\u5242\u91cf\u4f9d\u8d56\u6027\u66b4\u9732\uff0c\u5230\u8fbe\u9aa8\u9abc\u808c\uff0c\u5e76\u751f\u6210\u542b\u6709 PMO \u7684\u4ee3\u8c22\u7269\uff0c\u8be5\u4ee3\u8c22\u7269\u53ef\u5728\u808c\u8089\u4e2d\u505c\u7559\u8f83\u957f\u65f6\u95f4\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u6570\u636e\u8fd8\u663e\u793a\u513f\u7ae5\u548c\u6210\u4eba\u53c2\u4e0e\u8005\u4e4b\u95f4\u5b58\u5728\u5dee\u5f02\uff0c\u5e76\u8868\u660e\u5728\u5e7c\u5e74\u975e\u4eba\u7075\u957f\u7c7b\u52a8\u7269\u4e2d\uff0c\u8f83\u9ad8\u66b4\u9732\u91cf\u4f1a\u5bfc\u81f4\u66f4\u5f3a\u7684\u5916\u663e\u5b50\u8df3\u8dc3\u6548\u5e94\u3002. <strong>\u6b63\u5728\u8fdb\u884c\u7684 1\/2b \u671f ELEVATE-44-201 \u7814\u7a76\u6b63\u5728\u8bc4\u4f30 ENTR-601-44 \u5bf9\u9002\u5408\u5916\u663e\u5b50 44 \u8df3\u8dc3\u6cbb\u7597\u7684 DMD \u60a3\u8005\u7684\u7597\u6548\uff0c\u5176\u4e2d\u5305\u62ec\u9ad8\u5242\u91cf\u7ec4\u3002.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u91cd\u8981\u7684\u662f\uff0c\u8fd9\u4e9b\u7ed3\u679c\u662f\u65e9\u671f\u7684\u836f\u4ee3\u52a8\u529b\u5b66\/\u836f\u6548\u5b66\u53d1\u73b0\u3002.<\/strong> \u8be5\u6d77\u62a5\u5e76\u672a\u63d0\u4f9b\u4e34\u5e8a\u529f\u80fd\u83b7\u76ca\u7684\u8bc1\u636e\uff0c\u4e5f\u672a\u8bc1\u5b9eENTR-601-44\u80fd\u5ef6\u7f13DMD\u7684\u8fdb\u5c55\u3002\u6d77\u62a5\u62a5\u544a\u79f0\uff0c\u5728\u6240\u63cf\u8ff0\u7684\u4e34\u5e8a\u7814\u7a76\u4e2d\uff0c\u8fc4\u4eca\u4e3a\u6b62\u5c1a\u672a\u89c2\u5bdf\u5230\u4e25\u91cd\u4e0d\u826f\u4e8b\u4ef6\u6216\u5177\u6709\u4e34\u5e8a\u610f\u4e49\u7684\u80be\u529f\u80fd\u53d8\u5316\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>\n\n\n\n<p class=\"wp-block-paragraph\"><\/p>","protected":false},"excerpt":{"rendered":"<p>Entrada Therapeutics\u2019 ENTR-601-44 is an investigational therapy designed for patients with Duchenne muscular dystrophy (DMD) whose mutations are amenable to exon 44 skipping. New data presented at the 31st Annual Congress of the World Muscle Society in Hiroshima, Japan, examined how ENTR-601-44 behaves in the body and how its exposure relates to exon 44 skipping. 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