New poly-thiourea derivative transfection lipid compounds, useful for transferring nucleic acids into cells, e.g. for gene therapy

摘要

New transfection compounds (I) comprise a polythiourea moiety bonded to a lipid via a spacer. Poly-thiourea derivatives of formula (I), including their isomers or isomer mixtures, are new; R' = -((CH2)p-NHCSNH)q-X; l = 0 or 1; n, q = 1-6; m, p = 2-4; R = H or R', provided that if n = 1 and l = 0, than at least one R group = R'; X = 1-8C saturated or unsaturated, linear or cyclic aliphatic group, -CHSH, -(CH2)x'-(CHOH)u or -(CH2CH2O)vH, provided that (I) does not contain more than one -(CH2)x'-(CHOH)u or -(CH2CH2O)vH group; x' = 0-10; u = 1-6; v = 1-3; Y = spacer; L = -NR1R2 or OR3; R1, R2 = H (but not both H), a fatty aliphatic chain or -(CH2)t-OZ; t = 11-15; Z = sugar, polyol or polyethylene glycol (PEG) residue; R3 = steroid derivative residue. Independent claims are also included for: (1) compositions (A) comprising (I) and a nucleic acid (II). (2) a method for transfecting (II) into cells, comprising contacting (I) with (II) then contacting the obtained complex with the cells; and (3) a transfection kit containing one or more (I).