Lipid complexes for transferring at least a therapeutically active substance, in particular a polynucleotide into a target cell and use in gene therapy

Abstract

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: n,, n,, identical or different are whole numbers between 0 and 1; R,, 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 n,=n,=1, R,and R,can form together a divalent alkylene chain of 2 to 3 carbon atoms (C,-C,); R,, R,, 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 (C,-C,); m, p, identical or different are whole numbers between 1 and 10; R,, R,, identical or different are selected in the group consisting of radicals of formula: 1) R,C(═O)—X— in which: X=NH, O, S; R,is an alkyl or alkenyl radical of 6 to 23 carbon atoms (C,-C,), linear or branched; 2) R,R,NC(═O)— in which: R,, R,, 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 R,, R,, cannot simultaneously correspond to the hydrogen atom; 3) and one of the radicals R,, R,can moreover correspond to the hydrogen atom. The invention also concerns the use of said complex in gene therapy.