| 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 |
US2016076040(A1) |
申请公布日期 |
2016.03.17 |
| 申请号 |
US201514943612 |
申请日期 |
2015.11.17 |
| 申请人 |
Alnylam Pharmaceuticals, Inc. |
发明人 |
MANOHARAN Muthiah;RAJEEV Kallanthottathil G.;BUMCROT David |
| 分类号 |
C12N15/113 |
主分类号 |
C12N15/113 |
| 代理机构 |
|
代理人 |
|
| 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 |
