C - Chemistry – Metallurgy – 07 – J
Patent
C - Chemistry, Metallurgy
07
J
C07J 41/00 (2006.01) A61K 9/127 (2006.01) A61K 31/575 (2006.01) A61K 31/70 (2006.01) A61K 47/16 (2006.01) A61K 47/28 (2006.01) A61K 48/00 (2006.01) C12N 15/88 (2006.01)
Patent
CA 2248186
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.
La présente invention se rapporte à de nouveaux dérivés d'amidinium de formule (I) dans laquelle: R1 représente un dérivé du cholestérol ou un groupement alkyle amino-NR'R", R2 et R3 représentent indépendamment l'un de l'autre un atome d'hydrogène ou un groupement de formule (II) dans laquelle R4 et R5 représentent indépendamment l'un de l'autre un atome d'hydrogène ou un groupement de formule (III). La présente invention concerne en outre des compositions pharmaceutiques correspondantes utiles notamment en thérapie génique pour le transfert de gènes thérapeutiques dans des cellules.
Lehn Jean-Marie
Lehn Pierre
Vigneron Jean-Pierre
Centre National de La Recherche Scientifique
Robic
LandOfFree
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