| 发明名称 |
COMPLEMENT COMPONENT C5 iRNA COMPOSITIONS AND METHODS OF USE THEREOF |
| 摘要 |
The invention relates to iRNA, e.g., double-stranded ribonucleic acid (dsRNA), compositions targeting the complement component C5 gene, and methods of using such iRNA, e.g., dsRNA, compositions to inhibit expression of C5 and to treat subjects having a complement component C5-associated disease, e.g., paroxysmal nocturnal hemoglobinuria. |
| 申请公布号 |
US2016108401(A1) |
申请公布日期 |
2016.04.21 |
| 申请号 |
US201514976261 |
申请日期 |
2015.12.21 |
| 申请人 |
Alnylam Pharmaceuticals, Inc. |
发明人 |
Fitzgerald Kevin;Butler James;Bettencourt Brian;Borodovsky Anna;Kuchimanchi Satyanarayana;Charisse Klaus;Manoharan Muthiah;Maier Martin;Rajeev Kallanthottathil G.;Foster Donald |
| 分类号 |
C12N15/113 |
主分类号 |
C12N15/113 |
| 代理机构 |
|
代理人 |
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| 主权项 |
1. A double stranded ribonucleic acid (dsRNA) agent for inhibiting expression of complement component C5, wherein said dsRNA comprises a sense strand and an antisense strand, wherein said sense strand comprises at least 15 contiguous nucleotides differing by no more than 3 nucleotides from the nucleotide sequence of SEQ ID NO:1 and said antisense strand comprises at least 15 contiguous nucleotides differing by no more than 3 nucleotides from the nucleotide sequence of SEQ ID NO:5. |
| 地址 |
Cambridge MA US |
