Cationic lipids and compositions thereof

Provided herein are lipids having Formula I or Formula Ia: # imgabs0 # and pharmaceutically acceptable salts thereof, wherein R ', R1, R2, R3, R4, R5, R6a, R6b, X1, X2, and n are as defined herein for Formula I and Formula Ia, respectively. Also provided herein are lipid nanoparticle (LNP) comp...

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Bibliographische Detailangaben
Hauptverfasser: STANTON MATTHEW G, NOLTING, BJU RN, MYRSTED ANDERS
Format: Patent
Sprache:chi ; eng
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Zusammenfassung:Provided herein are lipids having Formula I or Formula Ia: # imgabs0 # and pharmaceutically acceptable salts thereof, wherein R ', R1, R2, R3, R4, R5, R6a, R6b, X1, X2, and n are as defined herein for Formula I and Formula Ia, respectively. Also provided herein are lipid nanoparticle (LNP) compositions comprising a lipid having Formula I or Ia and an uncapsid non-viral vector (e.g., ceDNA). In one aspect of any of aspects or embodiments herein, these LNPs may be used to deliver an uncapsid non-viral DNA vector to a target site of interest (e.g., a cell, tissue, organ, etc. 本文提供了具有式I或式Ia的脂质:#imgabs0#以及其药学上可接受的盐,其中R'、R1、R2、R3、R4、R5、R6a、R6b、X1、X2和n如本文分别对于式I和式Ia所定义的。本文还提供了脂质纳米颗粒(LNP)组合物,其包含具有式I或Ia的脂质和无衣壳非病毒载体(例如,ceDNA)。在本文的方面或实施方案中的任一者的一个方面,这些LNP可用于将无衣壳非病毒DNA载体递送至所关注的靶位点(例如,细胞、组织、器官等)。