Novel lipid complexes for transferring at least a...

A - Human Necessities – 61 – K

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A61K 47/18 (2006.01) A61K 31/70 (2006.01) A61K 47/22 (2006.01) A61K 47/48 (2006.01) A61K 48/00 (2006.01) C07C 237/06 (2006.01) C07D 295/13 (2006.01) C07D 295/15 (2006.01)

Patent

CA 2263367

The invention concerns a complex comprising at least a lipid and at least a therapeutically active substance useful for transferring said substance into a target cell, characterised in that said lipid is of formula (I): in which: n1, n2, identical or different are whole numbers between 0 and 1; R1, R2, identical or different are: a) selected among the group consisting of a hydrogen atom and alkyl radicals with 1 to 6 carbon atoms optionally substituted, independently of one another, by a hydroxyl radical; or b) in one particular case for which n1 = n2 = 1, R1 and R2 can form together a divalent alkylene chain of 2 to 3 carbon atoms (C2-C3); R3, R4, identical or different are alkyl radicals of 1 to 6 carbon atoms or can together form a divalent alkylene chain of 2 to 3 carbon atoms (C2-C3); m, p, identical or different are whole numbers between 1 and 10; R5, R6, identical or different are selected in the group consisting of radicals of formula: 1) R7 C(=0)-X- in which: X = NH, 0, S; R7 is an alkyl or alkenyl radical of 6 to 23 carbon atoms (C6-C23), linear or branched; 2) R8R9NC(=0)- in which: R8, R9, identical or different are selected among the group consisting of the hydrogen atom or alkyl or alkenyl radicals of 6 to 23 carbon atoms, linear or branched, provided that R8, R9, cannot simultaneously correspond to the hydrogen atom; 3) and one of the radicals R5, R6 can moreover correspond to the hydrogen atom. The invention also concerns the use of said complex in gene therapy.

L'invention concerne un complexe comprenant au moins un lipide et au moins une substance thérapeutiquement active utilisable pour le transfert de ladite substance à l'intérieur d'une cellule cible, caractérisé en ce que ledit lipide est de formule (I), dans laquelle: n1, n2 identiques ou différents sont des nombres entiers choisis parmi 0 ou 1; R1, R2 identiques ou différents, sont: a) choisis parmi le groupe constitué par un atome d'hydrogène et des radicaux alkyle de 1 à 6 atomes de carbone éventuellement substitués, indépendamment les uns des autres, par un radical hydroxyle; ou b) selon un cas particulier pour lequel n1 = n2 = 1, R1 et R2 peuvent former ensemble une chaîne divalente alkylène de 2 à 3 atomes de carbone (C2-C3); R3, R4, identiques ou différents sont des radicaux alkyle de 1 à 6 atomes de carbone ou peuvent former ensemble une chaîne divalente alkylène de 2 à 3 atomes de carbone (C2-C3); m, p identiques ou différents sont des nombres entiers compris entre 1 et 10; R5, R6, identiques ou différents sont choisis dans le groupe constitué par les radicaux de formule: I) R7 C(=0)-X- dans laquelle: X = NH, 0, S; R7 est un radical de 6 à 23 atomes de carbone (C6-C23) alkyle ou alcényle, linéaire ou ramifié; 2) R8R9NC(=0)- dans laquelle: R8, R9, identiques ou différents sont choisis parmi le groupe constitué par l'atome d'hydrogène ou des radicaux de 6 à 23 atomes de carbone alkyle ou alcényle, linéaires ou ramifiés, à la condition que R8, R9 ne puissent correspondre simultanément à l'atome d'hydrogène; 3) et l'un des radicaux R5, R6 pouvant en outre correspondre à l'atome d'hydrogène et son utilisation en thérapie génique.

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