Small Interfering RNA Targeting Amyloid‐Beta Precursor Protein Reduces Alzheimer’s Disease Pathology in 5xFAD Mice

Treatment with APP-lowering siRNA reduced AD pathology in the 5xFAD mouse model. Early intervention with the highest dose regimen prevented the emergence of behavioral deficits and substantially improved many aspects of disease phenotype. These results support the continued development of mivelsiran, an investigational, first-in-class APP-lowering RNAi therapeutic, in patients with AD (NCT05231785).