发明名称 Therapeutic compositions
摘要 This application relates to therapeutic siRNA agents and methods of making and using the agents.
申请公布号 US8809516(B2) 申请公布日期 2014.08.19
申请号 US201213626196 申请日期 2012.09.25
申请人 Alnylam Pharmaceuticals, Inc. 发明人 Manoharan Muthiah;Rajeev Kallanthottathil G.;Bumcrot David
分类号 C07H21/02;C12N15/113 主分类号 C07H21/02
代理机构 LeClairRyan 代理人 Townes Jeffrey N.;LeClairRyan
主权项 1. An iRNA agent, comprising: a sense strand sequence having 4-12 asymmetrical 2′-O-alkyl modifications, at least 4 of which occur within the 6 terminal nucleotides of the 5′ end; and an antisense sequence having at least 4 asymmetrical phosphorothioate modifications.
地址 Cambridge MA US