Artificially synthesized sphingosine derivative lipoid monomer and use of same for delivering nucleic acid
AssigneeBeijing Baishihekang Pharmaceutical Technology (Bsjpharma) Co., Ltd
InventorsChengyu JIANG, Yuhao QIN, Xiaoyun LI, Cong ZHANG
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Abstract
The invention provides an artificially synthesized single sphingosine lipid and use of delivering a nucleic acid thereof. More particularly, the invention provides Use or method for delivering a nucleic acid to a cell or a subject using a compound of Formula (I), a stereoisomer or a pharmaceutical acceptable salt thereof, or a combination comprising a compound of Formula (I), a stereoisomer or a pharmaceutical acceptable salt thereof,
Claims20
A method for delivering a nucleic acid to a cell or a subject, comprising:
- mixing a lipid composition with the nucleic acid to obtain a lipid-nucleic acid mixture, and
- delivering the lipid-nucleic acid mixture so that the nucleic acid is delivered to the cell or the subject,
- wherein the lipid composition comprises one or more compounds of Formula (I), a stereoisomer or a pharmaceutical acceptable salt thereof:
- wherein:
- R1 is selected from C1-20 alkyl or C2-20 alkenyl, which is optionally substituted by one to three hydroxyl groups;
- R2 is hydrogen and R3 is hydroxyl;
- or
- R2 and R3 together form oxo (═O);
- R4 and R5 are independently selected from the group consisting of hydrogen, C1-20 alkyl, C1-30 alkylacyl and C1-30 alkenylacyl, said C1-30 alkylacyl and C1-30 alkenylacyl are optionally substituted by one to three groups selected from biotin acyl or hydroxyl;
- or —NR4R5 group is a quaternary ammonium cation;
- R6 is selected from the group consisting of hydrogen, hydroxyl, phosphate ester group, —O-glycosyl, Ganglioside, and aminoethoxyphosphonate ester group (NH2—CH2—CH2—O—P(O)OH—).
A pharmaceutical composition, comprising a lipid composition and a nucleic acid, wherein the lipid composition comprises one or more compounds of Formula (I), a stereoisomer or a pharmaceutical acceptable salt thereof:
- wherein:
- R1 is selected from C1-20 alkyl or C2-20 alkenyl, which is optionally substituted by one to three hydroxyl groups;
- R2 is hydrogen and R3 is hydroxyl;
- or
- R2 and R3 together form oxo (═O);
- R4 and R5 are independently selected from the group consisting of hydrogen, C1-20 alkyl, C1-30 alkylacyl and C1-30 alkenylacyl, said C1-30 alkylacyl and C1-30 alkenylacyl are optionally substituted by one to three groups selected from biotin acyl or hydroxyl;
- or —NR4R5 group is a quaternary ammonium cation;
- R6 is selected from the group consisting of hydrogen, hydroxyl, phosphate ester group, —O-glycosyl, Ganglioside, and aminoethoxyphosphonate ester group (NH2—CH2—CH2—O—P(O)OH—).
A kit comprising a lipid composition,
- the lipid composition comprising one or more compounds of Formula (I), a stereoisomer or a pharmaceutical acceptable salt thereof:
- wherein:
- R1 is selected from C1-20 alkyl or C2-20 alkenyl, which is optionally substituted by one to three hydroxyl groups;
- R2 is hydrogen and R3 is hydroxyl;
- or
- R2 and R3 together form oxo (═O);
- R4 and R5 are independently selected from the group consisting of hydrogen, C1-20 alkyl, C1-30 alkylacyl and C1-30 alkenylacyl, said C1-30 alkylacyl and C1-30 alkenylacyl are optionally substituted by one to three groups selected from biotin acyl or hydroxyl;
- or —NR4R5 group is a quaternary ammonium cation;
- R6 is selected from the group consisting of hydrogen, hydroxyl, phosphate ester group, —O-glycosyl, Ganglioside, and aminoethoxyphosphonate ester group (NH2—CH2—CH2—O—P(O)OH—).
Dependencies are inferred from the claim text.