Cationic lipid compounds and use thereof for transferring...

A - Human Necessities – 61 – K

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A61K 47/48 (2006.01) A61K 9/127 (2006.01) A61K 31/575 (2006.01) A61K 33/18 (2006.01) A61K 33/20 (2006.01) A61K 48/00 (2006.01) C07J 9/00 (2006.01) C07J 41/00 (2006.01) C12N 15/87 (2006.01) C12N 15/88 (2006.01)

Patent

CA 2381224

The invention concerns the use of a monocationic lipid compound of general formula I(A): R¿1?O-CO-NH-(CH¿2?)¿n?-N?+¿(C¿2?H¿5?)¿3?, I?-¿ wherein: R¿1? represents the cholesteryl radical; n represents an integer equal to 1, 2 or 3 and in particular 3-.beta.[N-(N',N',N'-triethylaminopropane iodide)-carbamoyl] cholesterol (TEAPC-Chol) and/or a monocationic lipid compound of general formula I(B): R¿2?-CO-NH-(CH¿2?)¿n?-N?+¿(R)¿3?,X?-¿ wherein: R¿2? represents in particular the abietic acid radical, or the radical of a derivative of cholanic (cholic, deoxycholic, dehydrocholic, lithocholic) acid; R represents in particular a methyl or ethyl radical; n represents an integer equal to 2 or 3; X represents in particular a chlorine or iodine atom; and/or a dicationic lipid compound of general formula II(A): R¿1?O-CO-N[CH¿2?- CH¿2?-N?+¿(R)¿3?,X?-¿]¿2?; and/or a dicationic lipid compound of general formula II(B): R¿1?O-CO-CH¿2?-CH¿2?-CO-N[CH¿2?-CH¿2?-N?+¿(R)¿3?, X?-¿]¿2?; and/or a dicationic lipid compound of formula II(C): R¿2?-CO-N[CH¿2?-CH¿2?-N?+¿(R)¿3?,X?-¿]¿2? wherein R¿1? represents the cholesteryl radical; R¿2? represents in particular the abietic acid radical or the radical of a derivative of a cholanic (cholic, deoxycholic, dehydrocholic, lithocholic) acid; R represents in particular a hydrogen atom, a methyl or ethyl radical; X represents in particular a chlorine or iodine atom. Said compounds are useful for transfecting living organisms in vivo, organs in vivo, tumours in vivo, or cells in vitro or ex vivo.

La présente invention concerne l'utilisation d'un composé lipidique monocationique répondant à la formule générale I(A): R1O-CO-NH-(CH2)n- N+(C2H5)3, I- dans laquelle R1 représente le radical cholestéryle, n représente un entier égal à 1, 2 ou 3 et notamment le 3-.beta.[N-(N',N',N'- triéthylaminopropane iodure)-carbamoyle] cholestérol (TEAPC-Chol) et/ou d'un composé lipidique monocationique de formule générale I(B) R2-CO-NH-(CH2)n- N+(R)3,X- dans laquelle R2 représente notamment le radical de l'acide abiétique, ou le radical d'un dérivé de l'acide cholanique (cholique, déoxycholique, déhydrocholique, lithocholique), R représente notamment un radical méthyle ou éthyle, n représente un nombre entier égal à 2 ou 3, X représente notamment un atome de chlore ou d'iode, et/ou d'un composé lipidique dicationique de formule générale II(A) R1O-CO-N[CH2-CH2-N+(R)3,X-]2 et/ou d'un composé lipidique dicationique de formule générale II(B) R1O-CO-CH2- CH2-CO-N[CH2-CH2-N+(R)3,X-]2 et/ou d'un composé lipidique dicationique de formule générale (II)(C) R2-co-N[CH2-CH2-N+(R)3,X-]2; dans lesquelles R1 représente le radical cholestéryle, R2 représente notamment le radical de l'acide abiétique, ou le radical d'un dérivé de l'acide cholanique (cholique, déoxycholique, déhydrocholique, lithocholique, R représente notamment un atome d'hydrogène, un radical méthyle ou éthyle, X représente notamment un atome de chlore ou d'iode, pour la transfection d'organismes vivants in vivo, d'organes in vivo, de tumeurs in vivo, ou de cellules in vitro ou ex vivo.

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