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US 6,670,332 B1Granted
Complex cationic lipids having quarternary nitrogens therein
AssigneeVical Incorporated
InventorCarl J. Wheeler
Life of this patent
Estimating the term…
Abstract
Cationic lipids having a derivatized quaternary ammonium head group that provide improved cell targeting ability and enhanced transfective efficacy for introducing molecules into cells are provided.
Claims27
A compound of the formula:
- R1 and R2 are independently H;
- linear or branched, unsubstituted or substituted C1-23 alkyl, acyl, alkenyl, or heteroalkyl group having from 0 to 6 sites of unsaturation;
- or a cyclic or aryl group, said heteroalkyl, cyclic, and aryl groups comprising from 0 to 5 heteroatoms wherein said heteroatoms are not the first atoms in said groups, wherein the substituent groups are selected from the group consisting of —O—(CH2)k—CH3, —S—(CH2)k—CH3, and X—(CH2)k—, wherein X is a halide, and k is 0 to 4;
- R3 and R4 are independently H;
- linear or branched, unsubstituted or substituted C1-23 alkyl, acyl, alkenyl, or heteroalkyl group having from 0 to 6 sites of unsaturation;
- or a cyclic or aryl group, said heteroalkyl, cyclic, and aryl groups comprising from 0 to 5 heteroatoms wherein said heteroatoms are not the first atoms in said groups, wherein the substituent groups are selected from the group consisting of —O—(CH2)k—CH3, —S—(CH2)k—CH3, and X—(CH2)k—, wherein X is a halide, and k is 0 to 4;
- R5 has the structure
- wherein
- Z is selected from the group consisting of O, S, NR1, NH, Se, and CR7R8;
- R6 is selected from the group consisting of H, R1, R2, R3, and R4, and, when Z is O, NH, NR1, or S, R6 can further be an amino acid, peptide, polypeptide, protein, mono-, di- or polysaccharide, or other bioactive or pharmaceutical agent, wherein Z is an atom of said amino acid, peptide, polypeptide, protein, mono-, di- or polysaccharide, or other bioactive or pharmaceutical agent;
- n is 1 to 6;
- m is 1 to 10;
- Y is a pharmaceutically acceptable anion;
- and
- R7 and R8 independently or in combination are H or alkyl groups as defined for R1 and R2;
- wherein if Z is O, n is 1, and m is 3, then R6 is selected from the group defined for R3 and R4 and wherein R1 and R2 are not both H.
13 more elements
A compound of the formula:
- wherein
- R1 and R2 are saturated or unsaturated C10-C18 alkyl groups;
- R3 and R4 are independently H;
- linear or branched, unsubstituted or substituted C1-23 alkyl, acyl, alkenyl, or heteroalkyl group having from 0 to 6 sites of unsaturation;
- or a cyclic or aryl group, said heteroalkyl, cyclic, and aryl groups comprising from 0 to 5 heteroatoms wherein said heteroatoms are not the first atoms in said groups, wherein the substituent groups are selected from the group consisting of —O—(CH2)k—CH3, —S—(CH2)k—CH3, and X—(CH2)k—, wherein X is a halide, and k is 0 to 4;
- R5 has the structure:
- R7 and R8 are independently selected from the group defined for R1, R2, R3 and R4, and one of R7 and R8 can further be an amino acid, peptide, polypeptide, protein, mono-, di- or polysaccharide, or other bioactive or pharmaceutical agent, wherein an amino nitrogen of said amino acid, peptide, polypeptide, protein, mono-, di- or polysaccharide, or other bioactive or pharmaceutical agent is the N to which R7 or R8 is attached;
- n is 1 to 6;
- m is 1 to 10;
- and
- Y is a pharmaceutically acceptable anion.
8 more elements
A compound of the formula:
- wherein
- R1 and R2 are saturated or unsaturated C10-C18 alkyl groups;
- R3 and R4 are independently H;
- linear or branched, unsubstituted or substituted C1-23 alkyl, acyl, alkenyl or heteroalkyl group having from 0 to 6 sites of unsaturation;
- or a cyclic or aryl group, said heteroalkyl, cyclic, and aryl groups comprising from 0 to 5 heteroatoms wherein said heteroatoms are not the first atoms in said groups, wherein the substituent groups are selected from the group consisting of —O—(CH2)k—CH3, —S—(CH2)k—CH3, and X—(CH2)k—, wherein X is a halide, and k is 0to 4;
- wherein R5 has the structure
- wherein
- R6 and R7 are independently selected from the group defined for R1, R2, R3 and R4 and one of R6 and R7 can further be an amino acid, peptide, polypeptide, protein, mono-, di- or polysaccharide, or other bioactive or pharmaceutical agent, wherein an amino nitrogen of said amino acid, peptide, polypeptide, protein, mono-, di- or polysaccharide, or other bioactive or pharmaceutical agent is the N to which R6 or R7 is attached;
- W is O, NR8, NH, S, or Se;
- R8 is an alkyl group as defined for R1 and R2;
- n is 1 to 6;
- m is 1 to 10;
- and
- Y is a pharmaceutically acceptable anion.
11 more elements
A compound of the formula:
- wherein
- R1 and R2 are saturated or unsaturated C10-C18 alkyl groups;
- R3 and R4 are independently H;
- linear or branched, unsubstituted or substituted C1-23 alkyl, acyl, alkenyl, or heteroalkyl group having from 0 to 6 sites of unsaturation;
- or a cyclic or aryl group, said heteroalkyl, cyclic, and aryl groups comprising from 0 to 5 heteroatoms wherein said heteroatoms are not the first atoms in said groups, wherein the substituent groups are selected from the group consisting of —O—(CH2)k—CH3, —S—(CH2)k—CH3, and X—(CH2)k—, wherein X is a halide, and k is 0 to 4;
- wherein R5 has the structure
- wherein
- R6 and R7 are independently selected from the group defined for R1, R2, R3 and R4 and one of R6 and R7 can further be an amino acid, peptide, polypeptide, protein, mono-, di- or polysaccharide, or other bioactive or pharmaceutical agent, wherein a hydroxy oxygen of said amino acid, peptide, polypeptide, protein, mono-, di- or polysaccharide, or other bioactive or pharmaceutical agent is the O to which R6 is attached;
- W is O, NR8, NH, S, or Se;
- R8 is an alkyl group as defined for R1 and R2;
- n is 1 to 6;
- m is 1 to 10;
- and
- Y is a pharmaceutically acceptable anion.
11 more elements
Dependencies are inferred from the claim text.