Abstract: The description is directed to ionizable lipids useful for enhancing the delivery of therapeutic agents in liposomes.

Abstract: Here described is a composition comprising a compound of formula I: wherein R1 and R2 is independently selected from a group consisting of C10 to C18 alkyl, C12 to C18 alkenyl, and oleoyl group; R3 and R4 are independently selected from a group consisting of C1 to C6 alkyl, and C2 to C6 alkanol; X is selected from a group consisting of —CH2—, —S—, and —O— or absent; Y is selected from —(CH2)n, —S(CH2)n, —O(CH2)n—, thiophene, —SO2(CH2)n—, and ester; n=1-4; a=1-4; b=1-4; c=1-4; and Z? is a counterion.

Abstract: Here described is a composition comprising a compound of formula I: wherein R1 and R2 is independently selected from a group consisting of C10 to C18 alkyl, C12 to C18 alkenyl, and oleoyl group; R3 and R4 are independently selected from a group consisting of C1 to C6 alkyl, and C2 to C6 alkanol; X is selected from a group consisting of —CH2—, —S—, and —O— or absent; Y is selected from —(CH2)n, —S(CH2)n, —O(CH2)n—, thiophene, —SO2(CH2)n—, and ester; n=1-4; a=1-4; b=1-4; c=1-4; and Z? is a counterion.

Abstract: Here described is a composition comprising a compound of formula I: wherein R1 and R2 is independently selected from a group consisting of C10 to C18 alkyl, C12 to C18 alkenyl, and oleoyl group; R3 and R4 are independently selected from a group consisting of C1 to C6 alkyl, and C2 to C6 alkanol; X is selected from a group consisting of —CH2—, —S—, and —O— or absent; Y is selected from —(CH2)n, —S(CH2)n, —O(CH2)n—, thiophene, —SO2(CH2)n—, and ester; n=1-4; a=1-4; b=1-4; c=1-4; and Z? is a counterion.

Abstract: Here described are compounds consisting of the structure (targeting molecule)m-linker-(targeting molecule)n, wherein the targeting molecule is a retinoid or a fat soluble vitamin having a specific receptor on the target cell; wherein m and n are independently 0, 1, 2 or 3; and wherein the linker comprises a polyethylene glycol (PEG) or PEG-like molecule, as well as compositions and pharmaceutical formulations including these compounds which are useful for the targeting and delivery of therapeutic agents; and methods of using these compositions and pharmaceutical formulations.

Abstract: The description is directed to ionizable lipids useful for enhancing the delivery of therapeutic agents in liposomes.

Abstract: Here described is a method of delivery of a drug to a stellate cell using a composition comprising a compound of formula I: wherein R1 and R2 are independently selected from C10 to C18 alkyl, C12 to C18 alkenyl, and oleoyl; R3 and R4 are independently selected from C1 to C6 alkyl and C2 to C6 alkanol; X is selected from —CH2—, —S—, and —O—, or X is absent; Y is selected from —(CH2)n, —S(CH2)n—, —O(CH2)n—, -thiophene-, —SO2(CH2)n—, and ester; n?1-4; a?1-4; b?1-4; c?1-4; and Z? is a counterion.

Abstract: The description is directed to ionizable lipids useful for enhancing the delivery of therapeutic agents in liposomes.

Abstract: What is described is a method for treating a fibrotic disease by administering a pharmaceutical composition comprising a drug carrier, which comprises a lipid and a retinoid, and a double-stranded nucleic acid molecule, which comprises an antisense sequence to mRNA encoding human hsp47.

Abstract: What is described is a pharmaceutical composition for treating a fibrotic disease comprising a drug carrier, which comprises a lipid and a retinoid, and a double-stranded nucleic acid molecule, which comprises an antisense sequence to mRNA encoding human hsp47.

Abstract: What is described is a method for preparing a liposome that efficiently encapsulates a negatively charged therapeutic polymer, e.g., siRNA. The process involves preparing a lipid mixture comprising a cationic lipid in a water miscible organic solvent, such as ethanol, at a concentration of 2.3 mg/ml, and adding this solution to the polymer dissolved in water to a final concentration of 35% ethanol in water. The final charge ratio of drug:lipid is 1:2.5. The resulting nanoparticles have a mean size of 50 to 150 nm.

Abstract: Here described are compounds consisting of the structure (targeting molecule)m-linker-(targeting molecule)n, wherein the targeting molecule is a retinoid or a fat soluble vitamin having a specific receptor on the target cell; wherein m and n are independently 0, 1, 2 or 3; and wherein the linker comprises a polyethylene glycol (PEG) or PEG-like molecule, as well as compositions and pharmaceutical formulations including these compounds which are useful for the targeting and delivery of therapeutic agents; and methods of using these compositions and pharmaceutical formulations.

Abstract: Here described are compounds consisting of the structure (targeting molecule)m-linker-(targeting molecule)n, wherein the targeting molecule is a retinoid or a fat soluble vitamin having a specific receptor on the target cell; wherein m and n are independently 0, 1, 2 or 3; and wherein the linker comprises a polyethylene glycol (PEG) or PEG-like molecule, as well as compositions and pharmaceutical formulations including these compounds which are useful for the targeting and delivery of therapeutic agents; and methods of using these compositions and pharmaceutical formulations.

Abstract: Here described is a method of delivery of a drug to a stellate cell using a composition comprising a compound of formula I: wherein R1 and R2 are independently selected from C10 to C18 alkyl, C12 to C18 alkenyl, and oleoyl; R3 and R4 are independently selected from C1 to C6 alkyl and C2 to C6 alkanol; X is selected from —CH2—, —S—, and —O—, or X is absent; Y is selected from —(CH2)n, —S(CH2)n—, —O(CH2)n—, -thiophene-, -SO2(CH2)n—, and ester; n=1-4; a=1-4; b=1-4; c=1-4; and Z? is a counterion.

Abstract: Here described are compounds consisting of the structure (targeting molecule)m-linker-(targeting molecule)n, wherein the targeting molecule is a retinoid or a fat soluble vitamin having a specific receptor on the target cell; wherein m and n are independently 0, 1, 2 or 3; and wherein the linker comprises a polyethylene glycol (PEG) or PEG-like molecule, as well as compositions and pharmaceutical formulations including these compounds which are useful for the targeting and delivery of therapeutic agents; and methods of using these compositions and pharmaceutical formulations.

Abstract: Here described are compounds of formula I: wherein R1 and R2 are independently selected from C10 to C18 alkyl, C12 to C18 alkenyl, and oleoyl; R3 and R4 are independently selected from C1 to C6 alkyl and C2 to C6 alkanol; X is selected from —CH2—, —S—, and —O—, or X is absent; Y is selected from —(CH2)n, —S(CH2)n—, —O(CH2)n—, -thiophene-, —SO2(CH2)n—, and ester; n=1-4; a=1-4; b=1-4; c=1-4; and Z? is a counterion. Also described herein are compositions and pharmaceutical formulations including compounds of formula I which are useful for the delivery of therapeutic agents, and methods of using these compositions and formulations.

Abstract: What is described is a method for treating a fibrotic disease by administering a pharmaceutical composition comprising a drug carrier, which comprises a lipid and a retinoid, and a double-stranded nucleic acid molecule, which comprises an antisense sequence to mRNA encoding human hsp47.

Abstract: What is described is a method for preparing a liposome that efficiently encapsulates a negatively charged therapeutic polymer, e.g., siRNA. The process involves preparing a lipid mixture comprising a cationic lipid in a water miscible organic solvent, such as ethanol, at a concentration of 2.3 mg/ml, and adding this solution to the polymer dissolved in water to a final concentration of 35% ethanol in water. The final charge ratio of drug:lipid is 1:2.5. The resulting nanoparticles have a mean size of 50 to 150 nm.

Abstract: What is described is a pharmaceutical composition for treating a fibrotic disease comprising a drug carrier, which comprises a lipid and a retinoid, and a double-stranded nucleic acid molecule, which comprises an antisense sequence to mRNA encoding human hsp47.

Abstract: What is described is a method for preparing a liposome that efficiently encapsulates a negatively charged therapeutic polymer, e.g., siRNA. The process involves preparing a lipid mixture comprising a cationic lipid in a water miscible organic solvent, such as ethanol, at a concentration of 2.3 mg/ml, and adding this solution to the polymer dissolved in water to a final concentration of 35% ethanol in water. The final charge ratio of drug:lipid is 1:2.5. The resulting nanoparticles have a mean size of 50 to 150 nm.