COMPOUNDS FOR TARGETING DRUG DELIVERY AND ENHANCING siRNA ACTIVITY

摘要

Here described are compounds consisting of the structure
(targeting molecule)m-linker-(targeting molecule)n,

wherein the targeting molecule is a retinoid or a fat soluble vitamin having a specific receptor on the target cell; wherein m and n are independently 0, 1, 2 or 3; and wherein the linker comprises a polyethylene glycol (PEG) or PEG-like molecule, as well as compositions and pharmaceutical formulations including these compounds which are useful for the targeting and delivery of therapeutic agents; and methods of using these compositions and pharmaceutical formulations.

主权项

1. A compound, consisting of the structure (retinoid)m-linker-(retinoid)n, wherein m and n are independently 1, 2, or 3; wherein m+n=4-6; and wherein the linker comprises one or more elements selected from a polyethylene glycol (PEG), glutarate (Glu), hexyl, Gly3, and 4-aminobutanoate (GluNH).