Cationic lipids for transfection of negatively charged or...

A - Human Necessities – 01 – N

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A01N 37/18 (2006.01) A61K 9/127 (2006.01) A61K 38/17 (2006.01) A61K 48/00 (2006.01) C07C 217/08 (2006.01) C07C 229/16 (2006.01) C07C 237/10 (2006.01) C07C 255/24 (2006.01) C07C 271/12 (2006.01) C07C 279/12 (2006.01) C07F 9/10 (2006.01)

Patent

CA 2289078

A cationic lipid for transfection of nucleic acids comprising the mixture of a nucleic acid with a compound having structure (I), wherein Y = Cl-, Br-, I-, AcO-, or any pharmaceutically acceptable anion; X = O, S(O), CH2; R1, R1' = independently: C1-C18 linear hydrocarbon; m, n = 1, 2, 3; R2, R2' = independently: H; C1-C18 linear alkyl; cyanoethyl; aminopropyl; aminobutyl; C2- C4 alkyl guanidinium or amidinium; N,N,N- independently (C1-C18)aminopropyl or aminobutyl; C or N substituted spermine or spermidine; N,N-(C4-C18)alkyl-4- aminonobutyrylaminopropyl; p = 0, 1; i = 0, 1; R3, R3' = independently: C1-C6 linear alkyl, acetoxyethyl, CH2CO2CH2CH3; X ≠ CH2 when R2, R2' = C1-C6 linear alkyl; when p = 1, m and n ≠ 1.

Cette invention concerne un lipide cationique destiné à la transfection d'acides nucléiques et composé d'un mélange d'un acide nucléique et d'un composé représenté par la formule (I) dans laquelle Y est Cl-, Br-, I- et AcO- ou tout anion pharmaceutiquement acceptable; X est O, S(O), CH¿2?; R¿1? et R¿1'? sont indépendamment hydrocarbure linéaire C¿1?-C¿18?; m et n sont égaux à 1, 2 ou 3; R¿2? et R¿2'? sont indépendamment H; alkyle linéaire C¿1?-C¿18?, cyanoéthyle, aminopropyle, aminobutyle, guanidinium ou amidinium d'alkyle C¿2?-C¿4?, N,N,N-indépendamment aminobutyle ou aminopropyle (C¿1?-C¿18?), spermine ou spermidine à substitution C ou N,N,N-alkyl(C¿4?-C¿18?)-4-aminobutyrylaminopropyle; p = 0 ou 1; i = 0 ou 1; R¿3? et R¿3'? sont indépendamment alkyle linéaire C¿1?-C¿6?, acétoxyéthyle, CH¿2?CO¿2?CH¿2?CH¿3?; X étant différent de CH¿2? lorsque R¿2?, R¿2'? sont alkyle linéaire C¿1?-C¿6? et lorsque p = 1 et m et n sont différents de 1.

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