- 专利标题: Low molecular weight cationic lipids for oligonucleotide delivery
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申请号: US17848967申请日: 2022-06-24
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公开(公告)号: US11911475B2公开(公告)日: 2024-02-27
- 发明人: Matthew G. Stanton , Brian W. Budzik , Gregory L. Beutner , Hongbiao Liao
- 申请人: SIRNA THERAPEUTICS, INC.
- 申请人地址: US MA Cambridge
- 专利权人: SIRNA THERAPEUTICS, INC.
- 当前专利权人: SIRNA THERAPEUTICS, INC.
- 当前专利权人地址: US MA Cambridge
- 代理机构: NIXON PEABODY LLP
- 代理商 Ravinderjit Braich; David S. Resnick
- 主分类号: A61K47/18
- IPC分类号: A61K47/18 ; C07C211/08 ; C07C211/04 ; C07C211/17 ; C07C211/21 ; C12N15/88 ; C07D207/06 ; A61K48/00 ; A61K45/00
摘要:
The instant invention provides for novel cationic lipids that can be used in combination with other lipid components such as cholesterol and PEG-lipids to form lipid nanoparticles with oligonucleotides. It is an object of the instant invention to provide a cationic lipid scaffold that demonstrates enhanced efficacy along with lower liver toxicity as a result of lower lipid levels in the liver. The present invention employs low molecular weight cationic lipids with one short lipid chain to enhance the efficiency and tolerability of in vivo delivery of siRNA.
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