申请人:Boylan John Frederick
公开号:US20130072543A1
公开(公告)日:2013-03-21
The invention relates to compositions containing compounds of formula I:
and pharmaceutically acceptable salts thereof, wherein R, R
1
, R
2
, R
3
, and n are defined in the detailed description and claims. In addition, the present invention relates to novel formulations containing compounds of formula I for improved delivery of nucleic acids such as siRNA to the cytoplasm of target cells. In particular embodiments these formulations comprise compounds of formula I, phospholipids, cholesterol, and pegylated lipids. The present invention also relates to methods of manufacturing and using such compounds and compositions.
本发明涉及含有公式I化合物的组合物及其药学上可接受的盐,其中R、R1、R2、R3和n在详细描述和权利要求中定义。此外,本发明涉及新的公式I化合物的制剂,用于改善核酸如siRNA输送至靶细胞的细胞质。在特定实施例中,这些制剂包括公式I化合物、磷脂、胆固醇和聚乙二醇化脂质。本发明还涉及制备和使用这些化合物和组合物的方法。