- 专利标题: Extrahepatic delivery
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申请号: US18311484申请日: 2023-05-03
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公开(公告)号: US12005074B2公开(公告)日: 2024-06-11
- 发明人: Jayaprakash K. Nair , Martin Maier , Vasant Jadhav , Stuart Milstein , Kirk Brown , Rubina G. Parmar , Kallanthottathil G. Rajeev , Muthiah Manoharan , Alexander V. Kel'In , Muthusamy Jayaraman , Klaus Charisse , Adam Castoreno , Christopher S. Theile , Kevin Fitzgerald
- 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: Cozen O'Connor
- 代理商 Jeffrey N. Townes
- 主分类号: A61K31/713
- IPC分类号: A61K31/713 ; A61K9/00 ; A61K47/54 ; C12N15/113
摘要:
One aspect of the present invention relates to a double stranded iRNA agent comprising an antisense strand which is complementary to a target gene; a sense strand which is complementary to said antisense strand; and one or more lipophilic moieties conjugated to one or more internal positions on at least one strand, optionally via a linker or carrier. Another aspect of the invention relates to a method of gene silencing, comprising administering to a cell or a subject in need thereof a therapeutically effective amount of the lipophilic moieties-conjugated double-stranded iRNAs.
公开/授权文献
- US20240009225A1 EXTRAHEPATIC DELIVERY 公开/授权日:2024-01-11
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