Dianhydrohexitol based ionizable lipids for nucleic acid delivery

Abstract

The present invention provides, in part, dianhydrohexitol-based cationic lipids of Formula (I), and sub-formulas thereof: (I), or a pharmaceutically acceptable salt thereof. The present invention also provides, in part, dianhydrohexitol-based cationic lipids of Formulae (IIA), and sub-formulas thereof: (IIA), or a pharmaceutically acceptable salt thereof. The present invention also provides, in part, dianhydrohexitol-based cationic lipids of Formulae (IIB), and sub-formulas thereof: (IIB), or a pharmaceutically acceptable salt thereof. The compounds provided herein can be useful for delivery and expression of mRNA and encoded protein, e.g., as a component of liposomal delivery vehicle, and accordingly can be useful for treating various diseases, disorders and conditions, such as those associated with deficiency of one or more proteins.

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