siSOD1-047M3-AC1VP

siSOD1-047M3-AC1VP is a small interfering RNA (siRNA) therapeutic candidate designed to target the superoxide dismutase 1 (SOD1) gene. SOD1 mutations are associated with a subset of familial amyotrophic lateral sclerosis (ALS), known as SOD1 ALS. The drug’s mechanism involves RNA interference, leading to the degradation of mutant SOD1 mRNA and subsequent reduction in toxic SOD1 protein levels. This approach aims to slow or halt disease progression in patients carrying pathogenic SOD1 mutations. While other therapies such as tofersen have been developed for this indication, there is no direct evidence that siSOD1-047M3-AC1VP has reached advanced clinical development or approval stages as of June 2025.