- 专利标题: CARBOHYDRATE CONJUGATES AS DELIVERY AGENTS FOR OLIGONUCLEOTIDES
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申请号: US17533559申请日: 2021-11-23
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公开(公告)号: US20220143189A1公开(公告)日: 2022-05-12
- 发明人: Muthiah MANOHARAN , Kallanthottathil G. RAJEEV , Jayaprakash K. NAIR , Martin MAIER
- 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
- 主分类号: A61K47/28
- IPC分类号: A61K47/28 ; A61K31/7004 ; A61K31/7052 ; A61K31/70 ; A61K47/60 ; A61K47/54 ; C07H21/02 ; A61K31/7088 ; A61K31/713 ; A61K47/16 ; A61K47/22
摘要:
The present invention provides a modified RNAi agent comprising a sense strand and an antisense strand, wherein the antisense strand targets an HBV mRNA; all of the nucleotide sugars of the RNAi agent are 2′-modified; and at least one of the sense and antisense strands is conjugated to at least one targeting ligand that comprises two or more building blocks connected to a biantennary or triantennary branched linker, optionally via one or more linking groups.
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