Novel lipid compounds and compositions containing same used for the transfer of at least an active substance, in particular a polynucleotide, in a target cell and therapeutic use

摘要

The invention relates to new lipid compounds of formula:R-HN-[-(CH2)m-NR-]n-1-(CH2)m-NH-R Iin which: the R residues are, independently of each other, a hydrogen atom or a group of formula II: for which: R1 and R2 are, independently of each other, C6-C23 alkyl or alkenyl radicals, which are linear or branched, or radicals -C(=O)-(C6-C23) alkyl or -C(=O)-(C6-C23) alkenyl, which are linear or branched, aryl radicals, cycloalkyl radicals, fluoroalkyl radicals, polyethylene glycol groups, oxyethylene or oxymethylene groups which are optionally repeated, linear or branched, optionally substituted, p is a positive integer from 1 to 4, n is a positive integer from 1 to 6, m is a positive integer from 1 to 6 which may be different for each motif -(CH2)m, and more particularly for each motif -(CH2)m-NR- when n>1, the number of R groups of formula II being between 1 and 4 said compounds being optionally in a cationic form and being combined with one or more biologically acceptable anions. It also relates to new complexes comprising at least one said cationic compound and an active substance comprising negative charges allowing the introduction of said active substances into cells. It relates in particular to new complexes, in which the active substance consists of one or more nucleic acids, useful for transfecting cells.