Biodegradable lipids for delivery of nucleic acids
AssigneeTranslate Bio, Inc.
InventorsFrank DeRosa, Michael Heartlein
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Abstract
The present invention provides, in part, a biodegradable compound of formula I, and sub-formulas thereof:Formula (I) or a pharmaceutically acceptable salt thereof, where each X independently is O or S, each Y independently is O or S, and each R1 independently is defined herein; and a liposome composition comprising the cationic lipid of formula I or a sub-formula thereof, and methods of delivering agents, such as nucleic acids including mRNA, in vivo, by administering to a subject the liposome comprising the cationic lipid of formula I or a sub-formula thereof, where the agent is encapsulated within the liposome.
Claims27
A lipid nanoparticle comprising:
- messenger RNA (mRNA) encoding a protein;
- one or more non-cationic lipids, one or more cholesterol-based lipids and/or one or more PEG-modified lipids;
- and
- a compound of formula III:
- or a pharmaceutically acceptable salt thereof.
A lipid nanoparticle comprising:
- messenger RNA (mRNA) encoding a protein;
- one or more non-cationic lipids, one or more cholesterol-based lipids and/or one or more PEG-modified lipids;
- and
- a compound of formula IV:
- or a pharmaceutically acceptable salt thereof.
A lipid nanoparticle comprising:
- messenger RNA (mRNA) encoding a protein;
- one or more non-cationic lipids, one or more cholesterol-based lipids and/or one or more PEG-modified lipids;
- and
- a compound of formula V:
- or a pharmaceutically acceptable salt thereof.
Dependencies are inferred from the claim text.