Formulation for the Delivery of Nucleotide Sequences That Can Modulate Endogenous Interfering RNA Mechanisms

摘要

The present invention concerns a formulation in nanoemulsion form comprising a continuous aqueous phase and at least one dispersed phase, useful as system for delivering nucleotide sequences able to modulate endogenous mechanisms of RNA interference or as transfecting agent.

主权项

1. A formulation in nanoemulsion form comprising a continuous aqueous phase and a least one dispersed phase, and comprising:
at least 5 mole % of amphiphilic lipid; 15 to 70 mole % of at least one cationic surfactant comprising:
at least one lipophilic group selected from among:
an R or R—(C═O)— group, where R is a linear hydrocarbon chain having 11 to 23 carbon atoms,an ester or amide of fatty acids having 12 to 24 carbon atoms and phosphatidylethanolamine, anda poly(propylene oxide), andat least one hydrophilic group comprising at least one cationic group selected from among:
a linear or branched alkyl group having 1 to 12 carbon atoms and interrupted and/or substituted by at least one cationic group; anda hydrophilic polymeric group comprising at least one cationic group; and 10% to 55 mole % of a co-surfactant comprising at least one poly(ethylene oxide) chain comprising at least 25 ethylene oxide units; a solubilising lipid, optionally a helper lipid,where the mole percentages of amphiphilic lipid, cationic surfactant and co-surfactant are relative to the whole (amphiphilic lipid/cationic surfactant/co-surfactant/optional helper lipid).