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1. 发明申请

WO2023001323A1 CYCLOHEXANE LIPIDOIDS FOR NUCLEIC ACID TRANSFECTION AND USE THEREOF 审中-公开
公开(公告)号：WO2023001323A1
公开(公告)日：2023-01-26
申请号：PCT/CZ2022/050065
申请日：2022-07-15
申请人： USTAV ORGANICKE CHEMIE A BIOCHEMIE AV CR V.V.I.
发明人： CIGLER, Petr , GRANTZ SASKOVA, Klara , VANEK, Vaclav , HEJDANKOVA, Zuzana , LOUKOTOVA, Lenka , SVEC, Pavel , PRISS, Anastasiia , PETREZSELYOVA, Silvia
IPC分类号： C07C233/62 , C07C237/10 , C07D295/185 , C07D313/20 , A61K47/00
摘要： The present invention relates to a lipidoid of general formula I, wherein X is selected from -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(=S)O-, -CH2C(=S)S-, -CH2C(=O)NHNH-, -N=CH-, -CH=N-, -NH-N=CH-, and -CH=N-NH-; Y is alkylene C2-C10 chain; R1 is selected from alkyl C1-C46, alkenyl C2-C46, alkynyl C2-C46; Z is selected from H, -OH, -CH3, -CH2OH, -NH2, -C(=O)NH2, -CONH(CH2)2OH, -CON[(CH2)2OH]2, -CONHCH(CH2OH)2, -CONHCH2CH(-OH)CH2OH, -CONH(CH2)2C(=O)NH2, -CON[CH2C(=O)NH2]2, -CONH(CH2)2NHC(=O)NH2, -CONH(CH2)3-N+(CH3)2-(CH2)2-SO3–, -CONH(CH2)3-N+(CH3)2-(CH2)2-COO–, -COO(CH2)2-O-P(=O)(O–)-O(CH2)2-N+(CH3)3, -N+(CH3)2-(CH2)3-SO3–, -N+(CH3)2-(CH2)2-COO-, formula (II), and formula (III), wherein R2 is independently selected from hydrogen and -CH3; E is independently selected from O and S atoms; n is an integer within the range of from 1 to 5; and T is selected from -X-Y-N(R1)2, -C(=O)O(C1-C3 alkyl), -C(=O)OCH2CH2OH, formula (IV), formula (V), formula (VI), -C(=O)OH, -CONH(CH2)2OH, -CON[(CH2)2OH]2, -CONHCH(CH2OH)2, CONH(CH2)2C(=O)NH2, -CON[CH2C(=O)NH2]2, -CONHCH[C(=O)NH2]2, -CONH(CH2)2NHC(=O)NH2, -C(=O)NH2, -CONH(CH2)3-N+(CH3)2-(CH2)2-SO3–, -CONH(CH2)3-N+(CH3)2-(CH2)2-COO–, -NH2, -NHC(=O)CH3, -COO(CH2)2-O-P(=O)(O–)-O(CH2)2-N+(CH3)3, -OH, -O(C1-C3 alkyl), -NHC(=O)NH(CH3), -NHC(=S)N(CH3)2, -NHC(=S)NH(CH3), -NHC(=N-CN)NH2, -NHC(=N-CN)NH(CH3), -NHC(=N-CN)N(CH3)2, -NHC[=N-S(=O)2NH2]NH2, -N+(CH3)2-(CH2)3-SO3–, -N+(CH3)2-(CH2)2-COO–, wherein R2, E and n are as defined above; and/or if Z is -OH or -CH2OH, and T is -C(=O)OH, then Z together with T and three carbon atoms between them may form a cyclic lactone containing 4 to 5 carbon atoms; and pharmaceutically acceptable salts, addition salts and solvates thereof. This lipidoid is useful as a transfection agent. The invention further describes transfection reagents, transfection particles containing this lipidoid, and their use.

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