Targeting Endosomal GPCR (eGPCR) Signaling Platforms for the Treatment of Chronic Pain

National Center For Advancing Translational Sciences

Investigators

Abstract

The small interfering RNA (siRNA) is carefully encapsulated within polymeric nanoparticles, which have been demonstrated to successfully deliver the therapeutic agent to endosomes specifically in spinal cord dorsal horn neurons following intrathecal administration. The proof-of-concept efficacy studies of this innovative polymeric nanoparticle formulation have shown that it provides significantly prolonged efficacy when compared to traditional lipid nanoparticle formulations (LNP). Presently, the lead siRNAs were selected, and the pilot-scale production of the nanoparticle drug product is underway. The formulation characterization and confirmatory efficacy study are being conducted to produce key supporting data for further preclinical development of this novel therapeutic approach.