Patent ID: 8193246
Filing Date: 2012-06-05
Classification: A61K,A61P,C07C,C12N,Y02P

Abstract:
1. A formulation comprising a composition of at least one lipid moiety covalently linked to one or more transfection enhancer elements (TEE's), wherein the at least one lipid moiety comprises a sterol and wherein the one or more TEE's are defined according to the formula: wherein the pH sensitive hydrophilic moiety of each of the one or more TEE's is a weak acid having a pKa of between 2 and 6 and is located distally from the link between the lipid moiety and the TEE, and wherein the hydrophobic moiety of each of the one or more TEE's is independently an alkyl carbon chain comprising 12 to 20 carbon atoms; wherein the composition comprises the general formula (XIV) wherein Xa is bound at the 3′ position of the sterol and is —OPO wherein R Ya is absent or a non-branched, branched or cyclic substituted or unsubstituted alkyl, alkenyl, alkylene, alkynyl, or an aryl group with 1 to 8 carbon atoms, wherein Ya is substituted with one or more groups are selected from —OH; —NH Za is absent or selected from —O—; —S—; —N(H)C(O)—; —C(O)N(H)—; —C(O)O—; —OC(O)N(H)—; —C(O)—; —C(O)N(H)—; —N(H)C(O)O—; —NH—; —N(R wherein the sterol is selected from cholesterol, sitosterol, campesterol, desmosterol, fucosterol, 22-ketosterol, 20-hydroxysterol, stigmasterol, 22-hydroxycholesterol, 25-hydroxycholesterol, lanosterol, 7-dehydrocholesterol, dihydrocholesterol, 19-hydroxycholesterol, 5α-cholest-7-en3β-ol, 7-hydroxycholesterol, epicholesterol, ergosterol, and/or dehydroergosterol; and wherein the dotted lines represent possible graft positions of the one or more TEE's.