Lipid nanoparticle compositions for delivering antisense oligonucleotides

摘要

A lipid nanoparticle composition comprising a macromolecule conjugated to a polymer and a targeting agent is described. The composition can include a therapeutic agent. The therapeutic agent can be an antisense oligonucleotide (ASO). Exemplary ASOs target a portion of the nucleic acid encoding Akt-1 and modulate the expression of Akt-1 or target a portion of the nucleic acid encoding HIF-1 to express HIF-1 Adjust. And targeting a macromolecule bound to the polymer and a portion of the nucleic acid encoding Akt-1, targeting a portion of the nucleic acid encoding ASO that regulates the expression of Akt-1, or HIF-1. Also described are lipid nanoparticle compositions comprising a therapeutic agent that is an ASO such as ASO that modulates the expression of HIF-1. Also disclosed are pharmaceutical formulations, methods of making lipid nanoparticles, and methods of using lipid nanoparticles, eg, to treat cancer.