| 发明名称 |
SERPINA1 iRNA COMPOSITIONS AND METHODS OF USE THEREOF |
| 摘要 |
The invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the Serpina1 gene, and methods of using such RNAi agents to inhibit expression of Serpina1 and methods of treating subjects having a Serpina1 associated disease, such as a liver disorder. |
| 申请公布号 |
EP2999785(A1) |
申请公布日期 |
2016.03.30 |
| 申请号 |
EP20140730051 |
申请日期 |
2014.05.22 |
| 申请人 |
ALNYLAM PHARMACEUTICALS, INC. |
发明人 |
SEHGAL, ALFICA;CHARISSE, KLAUS;BETTENCOURT, BRIAN;MAIER, MARTIN;RAJEEV, KALLANTHOTTATHIL G.;HINKLE, GREGORY;MANOHARAN, MUTHIAH |
| 分类号 |
C12N15/113;A61K31/712;A61K31/713;A61P1/16;A61P11/00;A61P35/00 |
主分类号 |
C12N15/113 |
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