[EN] CATIONIC LIPIDS AND COMPOSITIONS THEREOF<br/>[FR] LIPIDES CATIONIQUES ET COMPOSITIONS DE CEUX-CI
申请人:GENERATION BIO CO
公开号:WO2022266032A1
公开(公告)日:2022-12-22
Provided herein are cationic lipids having the Formula I or la: and pharmaceutically acceptable salts thereof, wherein R', R1, R2, R3, R4, R5, R6a, R6b, X, and n are as defined herein. Also provided herein are lipid nanoparticle (LNP) compositions comprising a cationic lipid having the Formula I or la and a capsid-free, non- viral vector (e.g., ceDNA). In one aspect of any of the aspects or embodiments herein, these LNPs can be used to deliver a capsid-free, non-viral DNA vector to a target site of interest (e.g., cell, tissue, organ, and the like).
本文提供了具有公式I或la的阳离子脂质及其药学上可接受的盐,其中R'、R1、R2、R3、R4、R5、R6a、R6b、X和n的定义如本文所述。本文还提供了脂质纳米粒(LNP)组合物,包括具有公式I或la的阳离子脂质和无外壳、非病毒载体(例如ceDNA)。在本文的任何方面或实施例中的一个方面中,这些LNP可以用于将无外壳、非病毒DNA载体输送到感兴趣的靶位点(例如细胞、组织、器官等)。