Abstract: The present invention relates to compounds of Formula (I) or pharmaceutically acceptable enantiomers, salts, solvates or prodrugs thereof. The invention further relates to the use of the compounds of Formula (I) for the treatment of cystic fibrosis. The invention also relates to a process for manufacturing compounds of Formula (I).

Abstract: The present invention relates to compounds of Formula (I) or pharmaceutically acceptable enantiomers, salts, solvates or prodrugs thereof. The invention further relates to the use of the compounds of Formula (I) for the treatment of cystic fibrosis. The invention also relates to a process for manufacturing compounds of Formula (I).

Abstract: The invention relates to a compound of general formula (I): where X=0 or 1, Z=O, S or CH2, R1 and R2 independently=OH or OY, where Y=a protecting group, OR3, where R3=a monosaccharide or disaccharide group, A1-CH2—N+(CH3)3, X?, where X=halogen, A1=an amide (NHC(O)) or ester (OC(C)) bond, OPO(OM)2, where M=an alkyli or alkyli earth metal, OP(O)O?—O(CH2)2—N+(CH3)3, A2-(PEGX1-A3)n-R4, where n=0 or 1, PEGX1=a polyethyleneglycol of molecular weight X1, where X1 being less than or equal to 5,000 daltons, A2 and A3 independently=a ether (O), ester (OC(O)), amide (NHC(O)), urea (NHC(O)NH), thiourea (NHC(S)NH), or thioether (S) bond R4=a directing agent.

Abstract: Transfecting compounds which include an aminoglycoside linked to a lipid via a spacer, and their polyguanidylated derivatives are provided. These compounds are useful for the in vitro, ex vivo, or in vivo transfection of nucleic acids into various cell types.

Abstract: The present invention relates to a composition comprising a siRNA and a transfecting compound consisting of an aminoglycoside of the class of 4,5-disubstituted 2-deoxystreptamine ring linked via a spacer molecule to a lipid moiety of formula —(R1)R2 wherein R1 and R2 represent, independently of one another, a hydrogen atom or a fatty aliphatic chain or R1 or R2 is absent, with the proviso that at least one of R1 and R2 represents a fatty aliphatic chain; or —OR3 or —NR3 wherein R3 represents a steroidal derivative. The invention also concerns in vitro and in vivo applications of these compositions.

Abstract: The invention relates to a compound of general formula (I): where =0 or 1, Z=O, S or CH1-2, R1 and R2 independently=OH or OY, where Y=a protecting group, OR3, where R3=a monosaccharide or disaccharide group, A1-CH2—N+(CH3)3, X?, where X=halogen, A1=an amide (NHC(O)) or ester (OC(O)) bond, OPO(OM)2, where M=an alkali or alkali earth metal, OP(O)O?—O(CH2)2—N+(CH3)3, A2-(PEGX1-A3)n-R4, where n=0 or 1, PEGx1=a polyethyleneglycol of molecular weight X1, where X1, ??5,000 daltons, A2 and A3 independently=a ether (O), ester (OC(O)), amide (NHC((O)), urea (NHC(O)NH), thiourea (NHC(S)NH), or thioether (S) bond and R4=a directing agent.

Abstract: Transfecting compounds which include an aminoglycoside linked to a lipid via a spacer, and their polyguanidylated derivatives are provided. These compounds are useful for the in vitro, ex vivo, or in vivo transfection of nucleic acids into various cell types.

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.

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.