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AssigneeCLSN Laboratories, Inc.
InventorsMajed MATAR, Jason Fewell, Danny H. Lewis, Khursheed Anwer
Estimating the term…
Compositions, methods, and applications that increase the efficiency of nucleic acid transfection are provided. In one aspect, a pharmaceutical composition may include at least about 0.5 mg/ml concentration of a nucleic acid condensed with a cationic lipopolymer suspended in an isotonic solution, where the cationic lipopolymer includes a cationic polymer backbone having cholesterol and polyethylene glycol covalently attached thereto, and wherein the molar ratio of cholesterol to cationic polymer backbone is within a range of from about 0.1 to about 10, and the molar ratio of polyethylene glycol to cationic polymer backbone is within a range of from about 0.1 to about 10. The composition further may include a filler excipient.
1-26. (canceled)
A method of transfecting a mammalian cell, comprising:
A method of transfecting a targeted tissue of a subject, comprising administering to the subject a reconstituted composition comprising a nucleic acid, a cationic lipopolymer, and a filler excipient in an aqueous medium, wherein the cationic lipopolymer consists of polyethyleneimine (PEI) covalently linked independently to cholesterol and/or polyethylene glycol (PEG) groups, wherein the average PEG:PEI:cholesterol molar ratio in the cationic lipopolymer is within the range of 1-5 PEG:1 PEI:0.4-1.5 cholesterol, and wherein the nucleic acid and cationic lipopolymer form a complex, wherein the complex of the reconstituted composition enters the targeted tissue and elicits biological activity of the nucleic acid.
31-52. (canceled)
A method for treating a cancer in a subject comprising administering to a subject in need thereof a reconstituted composition comprising a nucleic acid, a cationic lipopolymer, and a filler excipient in an aqueous medium, wherein the cationic lipopolymer consists of polyethyleneimine (PEI) covalently linked independently to cholesterol and/or polyethylene glycol (PEG) groups, wherein the average PEG:PEI:cholesterol molar ratio in the cationic lipopolymer is within the range of 1-5 PEG:1 PEI:0.4-1.5 cholesterol, and wherein the nucleic acid and cationic lipopolymer form a complex, wherein the complex of the reconstituted composition enters cancer cells of the subject and elicits biological activity of the nucleic acid.
Dependencies are inferred from the claim text.