| 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 | US9222091(B2) | 申请公布日期 | 2015.12.29 |
| 申请号 | US201414282769 | 申请日期 | 2014.05.20 |
| 申请人 | ALNYLAM PHARMACEUTICALS, INC. | 发明人 | Manoharan Muthiah;Rajeev Kallanthottathil G.;Bumcrot David |
| 分类号 | C12N15/113;A01K67/027;C07K14/775;C12N15/11;C07H21/02 | 主分类号 | C12N15/113 |
| 代理机构 | LeClairRyan | 代理人 | Townes Jeffrey N.;LeClairRyan |
| 主权项 | 1. An RNA agent comprising: a sense 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, wherein the RNA agent comprises a multivalent galactose or a multivalent N-acetyl-galactosamine, and wherein the antisense sequence targets a human gene sequence. | ||
| 地址 | Cambridge MA US | ||