Compounds related to the amidinium family, pharmaceutical compositions containing same, and uses thereof
AssigneeAventis Pharma S.A.
InventorsJean-Marie Lehn, Pierre Lehn, Jean-Pierre Vigneron
Life of this patent
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Abstract
Novel amidinium derivatives of formula (I), wherein R1 is a cholesterol derivative or an alkylamino-NR'R" grouping, and each of R2 and R3 is independently a hydrogen atom or a grouping of formula (II), wherein each of R4 and R5 is independently a hydrogen atom or a grouping of formula (III), are disclosed. The corresponding pharmaceutical compositions, which are particularly useful in gene therapy for transferring therapeutic genes into cells, are also disclosed. ##STR1##
Claims32
A composition comprising a neutral lipid, a nucleic acid, and a compound of general formula I or a salt thereof ##STR7## in which:
- R1 represents a cholesterol derivative or an alkylamino --NR'R" group with R' and R", independently representing a saturated or unsaturated, linear or branched C.sub.12 to C.sub.22 aliphatic radical,
- R2 and R3 independently represent a hydrogen or a group of general formula II with at least one of R2 or R3 being different from a hydrogen, ##STR8## n and m represent, independently of each other and distinctly between the groups R2 and R3, an integer between 0 an 4,
- R4 and R5 independently represent a hydrogen or a group of formula III ##STR9## in which p and q independently represent an integer between 0 and 4 and r is equal to 0 or 1, with when r is equal to 1
- X representing an NH group and x then being equal to 1 or
- X representing a nitrogen and x then being equal to 2, and
- it being possible for p, q and r to vary independently between the group R4 and R5 and at least one of R.sub.4 or R.sub.5 is different from hydrogen.
A compound or a salt thereof selected from the group consisting of:
- (H.sub.2 N).sub.2.sup.+ C--NH--(CH.sub.2 (.sub.2 --NH--C))--R1 VI
- [(H.sub.2 N).sub.2.sup.+ C--NH--(CH.sub.2).sub.3 ][(H.sub.2 N).sub.2.sup.+ C--NH--(CH.sub.2).sub.4 ]--N --COO--R1 VII
- [(H.sub.2 N).sub.2.sup.+ C--NH--(CH.sub.2).sub.2 --N--(CH.sub.2).sub.2 --NH--COO--R1 VIII
- [(H.sub.2 N).sub.2.sup.+ C--(CH.sub.2).sub.2 ].sub.2 N--COO--R1 IX and
- [[(H.sub.2 N).sub.2.sup.+ C--(CH.sub.2 .sub.2 ].sub.2 --N--(CH.sub.2).sub.2 ].sub.2 N--COO--R1 X
- in which R1represents a cholesterol derivative or an alkylamino --NR'R" group with R' and R", independently representing a saturated or unsaturated, linear or branched C.sub.12 to C.sub.22 aliphatic radical.
Dependencies are inferred from the claim text.