Abstract: The present invention provides, in part, dianhydrohexitol-based cationic lipids of Formula (I), and sub-formulas thereof: or a pharmaceutically acceptable saft thereof. The present invention also provides, in part, dianhydrohexitol-based cationic lipids of Formula (II), and sub-formulas thereof: or a pharmaceutically acceptable salt thereof. The compounds provided herein 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.

Abstract: The present invention provides, in part, bis-ester and amide cationic lipid compounds of Formula (I): or a pharmaceutically acceptable salt thereof, bis-ester and amide cationic lipid compounds of Formula (II): or a pharmaceutically acceptable salt thereof, bis-ester and amide cationic lipid compounds of Formula (III): or a pharmaceutically acceptable salt thereof, and bis-ester and amide cationic lipid compounds of Formula (IV): 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.

Abstract: The present invention provides, in part, second generation “good” buffer-based cationic lipids of Formula (I), and subformulas thereof: Formula (I), (t), 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.

Abstract: The present invention provides, among other things, a novel class of cationic lipid compounds (e.g., cationic lipids having a structure according to Formula (I)) for in vivo delivery of therapeutic agents, such as nucleic acids. It is contemplated that these compounds are capable of highly effective in vivo delivery while maintaining a favorable toxicity profile.

Abstract: The present invention provides, in part, piperazine-based lipid compounds of Formula (I), and sub-formulas thereof: 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.

Abstract: The present disclosure relates generally to derivatives of polygodial and methods of use thereof. In some aspects, the present disclosure relates to using polygodial derivatives to treat cancer or other hyperproliferative diseases.