RN-0361: An Investigational APOC3-Targeted siRNA Therapeutic for Hypertriglyceridemia

I. Executive Summary

RN-0361 is an investigational therapeutic agent currently under development by Rona Therapeutics Inc. It belongs to the class of small interfering RNA (siRNA) therapeutics, designed to specifically target Apolipoprotein C-III (APOC3) mRNA.[1] By employing the mechanism of RNA interference (RNAi), RN-0361 aims to reduce the hepatic production of APOC3 protein, a key regulator of triglyceride metabolism, thereby addressing conditions characterized by elevated triglyceride levels.[2] The primary indications being pursued include hypertriglyceridemia (HTG), severe hypertriglyceridemia (SHTG), mixed dyslipidemia, familial chylomicronemia syndrome (FCS), and hyperlipoproteinemia Type I.[1]

RN-0361 utilizes Rona Therapeutics' proprietary GAIA platform, an siRNA conjugate and oligo chemistry technology engineered to enhance stability and enable robust, durable, hepatocyte-specific gene silencing following subcutaneous administration.[1] This platform aims to optimize the therapeutic profile of the siRNA molecule.