Lipid complexes for transferring at least a therapeutically active substance, in particular a polynucleotide into a target cell and use in gene therapy
申请人:Transgene S.A.
公开号:US06291423B1
公开(公告)日:2001-09-18
The invention concerns a complex comprising at least a lipid and at least a therapeutically active substance useful for transferring said substance into a target cell, characterised in that said lipid is of formula (I): in which: n1, n2, identical or different are whole numbers between 0 and 1; R1, R2, identical or different are: a) selected among the group consisting of a hydrogen atom and alkyl radicals with 1 to 6 carbon atoms optionally substituted, independently of one another, by a hydroxyl radical; or b) in one particular case for which n1=n2=1, R1 and R2 can form together a divalent alkylene chain of 2 to 3 carbon atoms (C2-C3); R3, R4, identical or different are alkyl radicals of 1 to 6 carbon atoms or can together form a divalent alkylene chain of 2 to 3 carbon atoms (C2-C3); m, p, identical or different are whole numbers between 1 and 10; R5, R6, identical or different are selected in the group consisting of radicals of formula: 1) R7 C(═O)—X— in which: X=NH, O, S; R7 is an alkyl or alkenyl radical of 6 to 23 carbon atoms (C6-C23), linear or branched; 2) R8R9NC(═O)— in which: R8, R9, identical or different are selected among the group consisting of the hydrogen atom or alkyl or alkenyl radicals of 6 to 23 carbon atoms, linear or branched, provided that R8, R9, cannot simultaneously correspond to the hydrogen atom; 3) and one of the radicals R5, R6 can moreover correspond to the hydrogen atom. The invention also concerns the use of said complex in gene therapy.
该发明涉及至少包含一种脂质和至少一种有治疗活性的物质的复合物,用于将该物质转运到靶细胞中,其特征在于所述脂质的
化学式为(I):其中:n1、n2为0到1之间的整数;R1、R2相同或不同,选自由氢原子和烷基基团(含1到6个碳原子,可选地独立地被羟基基团取代)的群中;或在一个特定情况下,当n1=n2=1时,R1和R2可以共同形成2到3个碳原子(C2-C3)的二价烷基链;R3、R4相同或不同,为1到6个碳原子的烷基基团,或者共同形成2到3个碳原子(C2-C3)的二价烷基链;m、p相同或不同为1到10之间的整数;R5、R6相同或不同,选自下列基团的群中:1)R7C(═O)—X—其中:X=NH、O、S;R7为6到23个碳原子(C6-C23)的直链或支链烷基或烯基基团;2)R8R9NC(═O)—其中:R8、R9相同或不同,选自氢原子或6到23个碳原子的直链或支链烷基或烯基基团,前提是R8、R9不能同时对应于氢原子;3)并且其中一个基团R5、R6还可以对应于氢原子。该发明还涉及在
基因治疗中使用所述复合物。