Parkinson’s disease (PD) is the second-fastest growing neurological disease in the world and in Norway, affecting 0.2% of Norwegian population, and costing the taxpayers ~0.5% of GDP in 2024. The causes and the precise onset mechanisms of PD remain unknown, and all the available therapeutics on the market remain purely symptomatic. This makes PD one of the largest problems in neurology.
Previously, our partners at Oslo University Hospital (OUS) and us have identified a novel gene, mechanistically linked to development of PD. Within SEP reASOn, we have designed, screened, and improved several therapeutic antisense oligonucleotide candidates (ASOs) that effectively downregulate expression of this target gene by 70%. We have selected and validated the best three candidates in human cell line models. We have detected a very low general toxicity of ASOs for human cells in vitro. Finally, we have demonstrated that our ASOs can rescue human cells from death in the presence of a PD-inducing drug, demonstrating therapeutic potential of our ASO candidates.
We will continue our ASO research together with our OUS partners. Recently, we have obtained a Qualification grant from the RCN where we will develop a strategy for therapeutic development of our ASO technology towards clinic.