{"id":4065,"date":"2025-07-10T11:42:12","date_gmt":"2025-07-10T08:42:12","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=4065"},"modified":"2025-07-10T11:43:34","modified_gmt":"2025-07-10T08:43:34","slug":"jcr-pharmaceuticals-proprietary-just-aav-capsids-may-be-used-in-the-development-of-duchenne-cures","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/zh\/jcr-pharmaceuticals-proprietary-just-aav-capsids-may-be-used-in-the-development-of-duchenne-cures\/","title":{"rendered":"JCR Pharmaceuticals \u4e13\u6709 JUST-AAV \u8863\u58f3\u53ef\u7528\u4e8e\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684\u6cbb\u7597"},"content":{"rendered":"<p class=\"wp-block-paragraph\">AstraZeneca \u65d7\u4e0b\u7684\u7f55\u89c1\u75c5\u5b50\u516c\u53f8 Alexion \u6b63\u5728\u4ece\u65e5\u672c\u516c\u53f8 JCR Pharmaceuticals \u83b7\u5f97\u4e13\u95e8\u7684\u57fa\u56e0\u6cbb\u7597\u8f7d\u4f53\u7684\u8bb8\u53ef\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u8be5\u4ea4\u6613\u4e8e 2025 \u5e74 6 \u6708 8 \u65e5\u5ba3\u5e03\uff0c\u5141\u8bb8 Alexion \u4f7f\u7528 JCR \u7684 JUST-AAV \u8863\u58f3\uff0c\u4ee5\u7528\u4e8e Alexion \u7684\u6700\u591a\u4e94\u4e2a\u57fa\u56e0\u7ec4\u836f\u7269\u9879\u76ee\u3002<\/strong> \u4f5c\u4e3a\u4ea4\u6362\uff0cAlexion \u5c06\u5411 JCR \u652f\u4ed8\u4e00\u7b14\u672a\u62ab\u9732\u7684\u9884\u4ed8\u8d39\u7528\uff0c\u4ee5\u53ca\u9ad8\u8fbe 2.25 \u4ebf\u7f8e\u5143\u7684\u7814\u53d1\u91cc\u7a0b\u7891\u4ed8\u6b3e\uff0c\u4ee5\u53ca\u989d\u5916\u7684 6 \u4ebf\u7f8e\u5143\u9500\u552e\u91cc\u7a0b\u7891\u4ed8\u6b3e\u3002JCR \u8fd8\u5c06\u83b7\u5f97\u4e0e\u9500\u552e\u76f8\u5173\u7684\u5206\u7ea7\u7279\u8bb8\u6743\u4f7f\u7528\u8d39\uff0c\u4f46\u5177\u4f53\u7ec6\u8282\u5c1a\u672a\u62ab\u9732\u3002<\/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=\"#whar-is-just-aav-capsids\">JUST-AAV \u8863\u58f3\u662f\u4ec0\u4e48\uff1f<\/a><\/li><li><a href=\"#just-aav-capsids-may-be-used-for-duchenne-cures\">JUST-AAV\u8863\u58f3\u6216\u53ef\u7528\u4e8e\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684\u6cbb\u7597<\/a><\/li><li><a href=\"#pre-clinical-trials-of-just-aav-capsids\">JUST-AAV\u8863\u58f3\u7684\u4e34\u5e8a\u8bd5\u9a8c\u524d<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"whar-is-just-aav-capsids\">JUST-AAV \u8863\u58f3\u662f\u4ec0\u4e48\uff1f<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>JUST-AAV \u662f\u4e00\u79cd\u5229\u7528\u6539\u826f\u7684\u817a\u76f8\u5173\u75c5\u6bd2 (AAV) \u8f7d\u4f53\u7684\u4e13\u6709\u5e73\u53f0\u6280\u672f\u3002<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u8be5\u6280\u672f\u9700\u8981\u5c06\u9488\u5bf9\u9009\u5b9a\u7ec4\u7ec7\u3001\u5668\u5b98\u6216\u8840\u8111\u5c4f\u969c\u4e0a\u7684\u53d7\u4f53\u7684\u5fae\u578b\u6297\u4f53\u63d2\u5165\u8863\u58f3\u8868\u9762\uff0c\u4ece\u800c\u589e\u5f3a\u5bf9\u8fd9\u4e9b\u7ec4\u7ec7\u548c\u5668\u5b98\u7684\u9776\u5411\u8f93\u9001\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u8fdb\u4e00\u6b65\u7684\u8863\u58f3\u4fee\u9970\u53ef\u6700\u5927\u9650\u5ea6\u5730\u51cf\u5c11\u8131\u9776\u6548\u5e94\u5e76\u63d0\u9ad8\u5b89\u5168\u6027\u3002\u8be5\u540d\u79f0\u6e90\u4e8e\u201cJCR\u201d\uff08\u201c\u6700\u7ec8\u5668\u5b98\u76ee\u6807\u201d\uff09\u3001\u201c\u9632\u6b62\u8131\u9776\u9012\u9001\u201d\u548c\u201c\u8f6c\u5316\u6280\u672f\u201d\uff0c\u53cd\u6620\u4e86\u5176\u5728\u591a\u79cd\u75be\u75c5\u9886\u57df\u5e7f\u6cdb\u5e94\u7528\u7684\u6f5c\u529b\u3002<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"just-aav-capsids-may-be-used-for-duchenne-cures\">JUST-AAV\u8863\u58f3\u6216\u53ef\u7528\u4e8e\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684\u6cbb\u7597<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Alexion \u5c06\u4f7f\u7528\u8fd9\u4e9b\u8863\u58f3\u6765\u6cbb\u7597\u54ea\u4e9b\u9002\u5e94\u75c7\u4e5f\u5c1a\u672a\u660e\u786e\u3002AstraZeneca \u7684\u7f55\u89c1\u75c5\u7814\u53d1\u7ba1\u7ebf\u5145\u6ee1\u4e86\u91cd\u75c7\u808c\u65e0\u529b\u548c\u72fc\u75ae\u6027\u80be\u708e\u7b49\u75be\u75c5\u7684\u4e2d\u540e\u671f\u8d44\u4ea7\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>JCR \u5ba3\u79f0\u5176\u4e13\u6709\u7684 JUST-AAV \u8863\u58f3\u6709\u591a\u79cd\u7c7b\u578b\uff0c\u7279\u522b\u662f\u201c\u4fdd\u809d\u201d\u578b\u3001\u808c\u8089\u9776\u5411\u578b\u548c\u8111\u9776\u5411\u578b\u3002<\/strong> \u7531\u4e8e\u4eca\u5e74\u65e9\u4e9b\u65f6\u5019\u4e24\u540d\u60a3\u8005\u5728\u63a5\u53d7 Sarepta \u7684\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 Elevidys \u57fa\u56e0\u6cbb\u7597\u540e\u56e0\u809d\u635f\u4f24\u800c\u6b7b\u4ea1\uff0c\u57fa\u56e0\u6cbb\u7597\u5bf9\u809d\u810f\u7684\u635f\u4f24\u6700\u8fd1\u53d7\u5230\u4e86\u5bc6\u5207\u5173\u6ce8\u3002\u2014\u2014\u9605\u8bfb\u66f4\u591a\uff1a <a href=\"https:\/\/dmdwarrior.com\/zh\/sarepta-reported-elevidys-gene-therapy-for-duchenne-muscular-dystrophy-will-not-be-used-in-non-ambulatory-patients\/\">Elevidys \u57fa\u56e0\u7597\u6cd5\u4e0d\u9002\u7528\u4e8e\u65e0\u6cd5\u884c\u8d70\u7684\u60a3\u8005<\/a> \u2013<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"pre-clinical-trials-of-just-aav-capsids\">JUST-AAV\u8863\u58f3\u7684\u4e34\u5e8a\u8bd5\u9a8c\u524d<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u7814\u7a76\u4eba\u5458\u901a\u8fc7\u5c06\u4e00\u79cd\u4e0e\u8f6c\u94c1\u86cb\u767d\u53d7\u4f53\u7ed3\u5408\u7684\u5fae\u578b\u6297\u4f53\u6574\u5408\u5230AAV\u8863\u58f3\u4e2d\uff0c\u6210\u529f\u6784\u5efa\u4e86\u4e00\u79cd\u9776\u5411\u8111\u7684AAV\u8f7d\u4f53\uff08\u9776\u5411\u8111\u7684JUST-AAV\uff09\u3002\u6b64\u5916\uff0c\u5bf9AAV\u8863\u58f3\u5e8f\u5217\u7684\u72ec\u7279\u4fee\u9970\u663e\u8457\u964d\u4f4e\u4e86AAV\u8f7d\u4f53\u5728\u809d\u810f\u4e2d\u7684\u84c4\u79ef\uff0c\u800c\u809d\u810f\u662f\u5df2\u77e5\u7684\u4e0d\u826f\u53cd\u5e94\u6765\u6e90\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u5728\u5c0f\u9f20\u7814\u7a76\u4e2d\uff0cJUST-AAV\u8f7d\u4f53\u5728\u8111\u4e2d\u7684\u7eff\u8272\u8367\u5149\u86cb\u767d\uff08GFP\uff09\u8868\u8fbe\u91cf\u6bd4AAV9\u9ad8\u51fa77\u500d\uff0c\u540c\u65f6\u964d\u4f4e\u4e8699%\u8f7d\u4f53\u5bf9\u809d\u810f\u7684\u8d8b\u5411\u6027\u3002\u4e3a\u4e86\u8fdb\u4e00\u6b65\u589e\u5f3a\u8111\u9776\u5411\u6027\u548c\u8840\u8111\u5c4f\u969c\uff08BBB\uff09\u901a\u900f\u6027\uff0c\u7814\u7a76\u4eba\u5458\u5728AAV\u8863\u58f3\u4e2d\u52a0\u5165\u4e86\u4e0e\u8f6c\u94c1\u86cb\u767d\u53d7\u4f53\u4ee5\u5916\u7684\u53d7\u4f53\u7ed3\u5408\u7684\u5176\u4ed6\u5206\u5b50\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u5728\u7334\u5b50\u7814\u7a76\u4e2d\uff0c\u8fd9\u79cd\u53cc\u7279\u5f02\u6027\u8f7d\u4f53\u4e0e AAV9 \u76f8\u6bd4\uff0c\u5c06\u57fa\u56e0\u4f20\u9012\u5230\u5927\u8111\u7684\u6548\u7387\u63d0\u9ad8\u4e86\u51e0\u4e2a\u6570\u91cf\u7ea7\uff0c\u540c\u65f6\u5c06\u809d\u810f\u548c\u80cc\u6839\u795e\u7ecf\u8282\u7b49\u6f5c\u5728\u95ee\u9898\u7ec4\u7ec7\u7684\u611f\u67d3\u51cf\u5c11\u4e86 90% \u4ee5\u4e0a\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u5c06JUST-AAV\u5e94\u7528\u4e8e\u795e\u7ecf\u5143\u8721\u6837\u8102\u8910\u8d28\u6c89\u79ef\u75c7\u5c0f\u9f20\u6a21\u578b\uff0c\u53ef\u4f7f\u766b\u75eb\u7b49\u75c7\u72b6\u6d88\u5931\uff0c\u5bff\u547d\u5ef6\u957f\uff0c\u8fbe\u5230\u529f\u80fd\u6027\u6cbb\u6108\u7684\u7a0b\u5ea6\u3002\u8fd9\u4e9b\u7ed3\u679c\u8868\u660e\uff0c\u65b0\u5f00\u53d1\u7684JUST-AAV\u6280\u672f\u6709\u671b\u5b9e\u73b0\u6bd4\u4f20\u7edf\u57fa\u56e0\u9012\u9001\u8f7d\u4f53\u66f4\u5b89\u5168\u3001\u66f4\u9ad8\u6548\u7684\u57fa\u56e0\u6cbb\u7597\u3002\u2014\u2014\u4e86\u89e3\u66f4\u591a\uff1a <a href=\"https:\/\/jcrpharm.com\/updatedjcr-pharmaceuticals-presents-preclinical-gene-therapy-data-at-asgct-28th-annual-meeting\/\" target=\"_blank\" rel=\"noreferrer noopener\">JCR Pharmaceuticals \u5c55\u793a\u4e34\u5e8a\u524d\u57fa\u56e0\u6cbb\u7597\u6570\u636e<\/a> \u2013<\/p>","protected":false},"excerpt":{"rendered":"<p>Alexion, AstraZeneca\u2019s rare disease subsidiary, is licensing specialized gene therapy vehicles from the Japanese firm JCR Pharmaceuticals. The deal, announced on 8th June 2025, gives Alexion access to JCR\u2019s JUST-AAV capsids to use in up to five of Alexion\u2019s genomic medicines programs. In exchange, Alexion will pay JCR an undisclosed upfront fee plus up to [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":4069,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[386,387,388,385],"class_list":["post-4065","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-alexion","tag-astrazeneca","tag-genomic-medicines","tag-jcr-pharmaceuticals"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/4065","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=4065"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/4065\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media\/4069"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media?parent=4065"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/categories?post=4065"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/tags?post=4065"}],"curies":[{"name":"\u53ef\u6e7f\u6027\u7c89\u5242","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}