Development of an Antisense Oligonucleotide (ASO) Delivery Platform for Rare Diseases

National Center For Advancing Translational Sciences

Investigators

Abstract

Cell-based assays were established to measure the efficiency and ability of the LNPs to deliver ASOs into neuronal and non-neuronal cell types, as well as their toxicity. The ability of LNPs to encapsulate ASOs was confirmed, and the delivery efficiency of several formulations were compared by knockdown experiments. A promising lead formulation has been identified that shows a 1-to-2 log increase of knockdown efficiency, compared to unencapsulated ASO. We are in the process of further examining how LNP encapsulation can improve acute ASO toxicity using the assays we developed internally to evaluate the safety and toxicity of the ASO scaffolds.