| 发明名称 | Dual targeting siRNA agents | ||
| 摘要 | The invention relates to dual targeting siRNA agents targeting a PCSK9 gene and a second gene, and methods of using dual targeting siRNA agents to inhibit expression of PCSK.9 and to treat PCSK.9 related disorders, e.g., hyperlipidemia. | ||
| 申请公布号 | US9187746(B2) | 申请公布日期 | 2015.11.17 |
| 申请号 | US201013497226 | 申请日期 | 2010.09.22 |
| 申请人 | Alnylam Pharmaceuticals, Inc. | 发明人 | Fitzgerald Kevin;Frank-Kamenetsky Maria;Charisse Klaus |
| 分类号 | A61K31/70;C07H21/02;C07H21/04;C12N15/113;A61K31/713;C12Q1/68 | 主分类号 | A61K31/70 |
| 代理机构 | Fenwick & West LLP | 代理人 | Fenwick & West LLP |
| 主权项 | 1. A dual targeting siRNA agent comprising a first dsRNA targeting a PCSK9 gene, the first dsRNA comprising a first sense strand comprising the nucleotide sequence of SEQ ID NO:4148 and a first antisense strand comprising the nucleotide sequence of SEQ ID NO:4150, and a second dsRNA targeting a XBP-1 gene, the second dsRNA comprising a second sense strand comprising the nucleotide sequence of SEQ ID NO:4154 and a second strand comprising the nucleotide sequence of SEQ ID NO:4156 wherein the first dsRNA and the second dsRNA are linked with a covalent linker. | ||
| 地址 | Cambridge MA US | ||