Ionizable lipids and nanoparticle compositions thereof

Provided herein are ionizable lipids represented by Formula (I): or a pharmaceutically acceptable salt thereof, wherein R1, R2, R3, R4, R5, R6, R1 ', R2', R3 ', R4', R5 ', R6', m, and n are as defined herein. Also provided herein are lipid nanoparticle (LNP) composition...

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Bibliographische Detailangaben
Hauptverfasser: LEBLANC MICHAEL, CHATTERTON, JON, E, STANTON MATTHEW G, NOLTING, BJU RN, FEINSTEIN, GUY
Format: Patent
Sprache:chi ; eng
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Zusammenfassung:Provided herein are ionizable lipids represented by Formula (I): or a pharmaceutically acceptable salt thereof, wherein R1, R2, R3, R4, R5, R6, R1 ', R2', R3 ', R4', R5 ', R6', m, and n are as defined herein. Also provided herein are lipid nanoparticle (LNP) compositions comprising an ionizable lipid of the invention and a capsid-free non-viral vector (e.g., ceDNA). These LNPs can be used to deliver capsid-free non-viral DNA vectors to target sites of interest (e.g., cells, tissues, organs, etc.). 本文提供了由式(I)表示的可电离脂质:或其药学上可接受的盐,其中R1、R2、R3、R4、R5、R6、R1'、R2'、R3'、R4'、R5'、R6'、m和n如本文所定义。本文还提供了脂质纳米颗粒(LNP)组合物,其包含本发明的可电离脂质和无衣壳的非病毒载体(例如ceDNA)。这些LNP可用于将无衣壳的非病毒DNA载体递送至所关注的靶位点(例如,细胞、组织、器官等)。