Biodegradable lipidoids and compositions and methods of use thereof for liver targeted delivery
AssigneeThe Trustees of the University of Pennsylvania
InventorsMichael Mitchell, Lulu Xue, Lili Wang, James M. Wilson, Claude Warzecha
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Abstract
Described are biodegradable lipid nanoparticles (LNPs) comprising biodegradable lipidoids and compositions thereof. In various embodiments, the LNP selectively targets a liver cell. In other aspects, the present invention relates to methods for in vivo delivery of therapeutic nucleic acids to the liver to prevent or treat diseases or disorders using the LNP compositions of the invention.
Claims25
A composition comprising biodegradable lipid nanoparticles (LNP) useful for delivering a nucleic acid to a liver cell, the LNP being formed from:
- (a) at least one ionizable lipid compound having the structure of Formula (LA), Formula (IB), or combinations thereof:
- or a racemate, an enantiomer, a diastereomer, a pharmaceutically acceptable salt, or a derivative thereof;
- wherein the total concentration of ionizable lipid(s) (a) in the LNP is present in a concentration range of about 1 mol % to about 99 mol %, based on the total amount of lipids in the LNP;
- (b) at least one neutral phospholipid, wherein the neutral phospholipid is present in a concentration range of about 10 mol % to about 45 mol % based on the total amount of lipids in the LNP;
- (c) at least one cholesterol lipid, wherein the total cholesterol lipid is in a concentration range of about 5 mol % to about 55 mol % based on the total amount of lipids in the LNP;
- and
- (d) at least one polyethylene glycol (PEG) lipid, wherein the total PEG-lipid is in a concentration range of about 0.5 mol % to about 12.5 mol % based on the total amount of lipids in the LNP;
- (e) at least one nucleic acid encapsulated in the LNP.
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Dependencies are inferred from the claim text.