Novel cationic lipopolymer as a biocompatible gene delivery agent
AssigneeExpression Genetics, Inc.
InventorsRam I. Mahato, Sang-Oh Han, Darin Y. Furgeson, Khursheed Anwer
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Abstract
A biodegradable cationic lipopolymer comprising a polyethylenimine (PEI), a lipid, and a biocompatible hydrophilic polymer, wherein 1) the lipid and the biocompatible hydrophilic polymer are directly linked to the PEI backbone or 2) the lipid is linked to the PEI backbone through the biocompatible hydrophilic polymer. The cationic lipopolymers of the present invention can be used for delivery of a nucleic acid or any anionic bioactive agent to various organs and tissues after local or systemic administration.
Claims27
A biocompatible cationic lipopolymer comprising a polyethylenimine (PEI), a lipid, and a biocompatible hydrophilic polymer spacer, wherein the lipid is attached to the PEI back bone via the biocompatible hydrophilic polymer spacer by a covalent bond.
8 The cationic lipopolymer of claim 1 further comprises a targeting moiety which is covalently attached to the PEI back bone directly or through a hydrophilic spacer.
The cationic lipopolymer of claim 8, wherein the targeting moiety is selected from the group consisting of transferrin, asialoglycoprotein, antibodies, antibody fragments, low density lipoproteins, interleukins, GM-CSF, G-CSF, M-CSF, stem cell factors, erythropoietin, epidermal growth factor (EGF), insulin, asialoorosomucoid, mannose-6-phosphate, mannose, LewisX and sialyl LewisX, N-acetyllactosamine, folate, galactose, lactose, and thrombomodulin, fusogenic agents, lysosomotrophic agents, and nucleus localization signals (NLS).
The cationic lipopolymer of claim 8, wherein the covalent bond is an ester, amide, urethane, or dithiol bond.
The cationic lipopolymer of claim 8, wherein the molar ratio of the cationic lipopolymer and the targeting moiety is within a range of 1:0.1 to 1:100.
A cationic lipopolymer comprising a polyethylenimine (PEI), a lipid, and a biocompatible hydrophilic polymer, wherein the lipid and the biocompatible hydrophilic polymer are directly and independently attached to the PEI backbone by a covalent bond.
A complex formed between a nucleic acid and a cationic lipopolymer of claim 8 in a N/P (nitrogen atoms on polymer/phosphate atoms on DNA) ratio within a range of 0.1/1 to 500/1.
A liposome comprising a biocompatible cationic lipopolymer of claim of 1 and a helper lipid in a molar ratio within a range of 1:0.1 to 1:500.
Dependencies are inferred from the claim text.