发明授权
US09587240B2 Compositions and methods for inhibiting expression of a target gene
有权
用于抑制靶基因表达的组合物和方法
- 专利标题: Compositions and methods for inhibiting expression of a target gene
- 专利标题(中): 用于抑制靶基因表达的组合物和方法
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申请号: US14737304申请日: 2015-06-11
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公开(公告)号: US09587240B2公开(公告)日: 2017-03-07
- 发明人: Roland Kreutzer , Stefan Limmer , Sylvia Limmer , Philipp Hadwiger
- 申请人: Alnylam Pharmaceuticals, Inc.
- 申请人地址: US MA Cambridge
- 专利权人: Alnylam Pharmaceuticals, Inc.
- 当前专利权人: Alnylam Pharmaceuticals, Inc.
- 当前专利权人地址: US MA Cambridge
- 代理机构: Fenwick & West LLP
- 优先权: DE10100586 20010109; DE10155280 20011026; DE10158411 20011129; DE10160151 20011207
- 主分类号: A61K31/713
- IPC分类号: A61K31/713 ; A61K31/7115 ; C12N15/113 ; C12N15/63 ; C12N15/11 ; A61K38/00
摘要:
The present invention relates to a double-stranded ribonucleic acid (dsRNA) having a nucleotide sequence which is substantially identical to at least a part of a target gene and which is no more than 49, preferably less than 25, nucleotides in length, and which comprises a complementary (antisense) RNA strand having a 1 to 4 nucleotide overhang at the 3′-end and a blunt 5′-end. The invention further relates to a pharmaceutical composition comprising the dsRNA and a pharmaceutically acceptable carrier. The pharmaceutical compositions are useful for inhibiting the expression of a target gene, as well as for treating diseases caused by expression of the target gene, at low dosages (i.e., less than 5 milligrams, preferably less than 25 micrograms, per kg body weight per day). The invention also relates to methods for inhibiting the expression of a target gene, as well as methods for treating diseases caused by the expression of the gene.
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