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US 2026/0176231 A1Published application
Lipidoids for nucleic acid transfection and use thereof
AssigneeUstav Organicke Chemie A Biochemie Av Cr, V.V.I.
InventorsPetr Cígler, Klara GRANTZ SASKOVA, Vaclav VANEK, Zuzana KRUZIKOVA, Frantisek SEDLAK
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Abstract
The subject of the present invention is a lipidoid of general formula I wherein X, Y, Z and R are as defined in the claims. This lipidoid is useful as a transfection agent. The invention further describes transfection agents, transfection particles containing this lipidoid, and their use.
Claims14
A lipidoid of general formula I
- wherein X is selected from a group consisting of —C(═O)NH—, —C(═O)O—, —C(═S)O—, —C(═O)S—, —C(═S)S—, —C(═O)NHNH—, —CH2—, —O—, —OC(═O)—, —S—, —SC(═O)—, —NH—, —NHNH—, —NHC(═O)—, —NHNHC(═O)—, —C≡C—, —CH═CH—, a five-membered heterocycle containing at least 2 nitrogen atoms, —CH2C(═O)NH—, —CH2C(═O)O—, —CH2C(═S)O—, —CH2C(═S)S—, —CH2C(═O)NHNH—, —N═CH—, and —CH═N—;
- Y is independently selected from a group consisting of alkylene chains C2-C10, wherein in the said alkylene chain, one or more —CH2— groups may optionally be replaced with one or more O or S atoms;
- Z is selected from the group consisting of hydrogen, —OH, —CH2OH, —NH2, —N+(CH3)2—(CH2)3—SO3, —N+(CH3)2—(CH2)2—COO—, and —NHCH3, —N(CH3)2, —N+(CH3)3, —OCH3, —OCH2CH3, —C(═O)R1,
- wherein R1 is selected from —NH2, —NH(CH2)nOH, —N[(CH2)nOH]2, —NHCH(CH2OH)2, —NHCH2CH(—OH)CH2OH, —NH(CH2)nC(═O)NH2, —N[CH2C(═O)NH2]2, —NHCH[C(═O)NH2]2, —NH(CH2)2NHC(═O)NH2,
- wherein n is an integer within the range from 2 to 5;
- and R are the same or different from each other, each R being independently selected from the group consisting of alkyl C8-C20, alkenyl C8-C20, and alkynyl C8-C20, wherein in the said alkyl, alkenyl or alkynyl, one or more —CH2— groups may optionally be replaced with one or more groups selected from —CH(OH)—, —OC(═O)—, —C(═O)O—, —S—S—, —C(═O)NH—, —NHC(═O)—, —O—, and —S—;
- and pharmaceutically acceptable salts, addition salts and solvates thereof.
4 more elements
Dependencies are inferred from the claim text.