ARO-SNCA

ARO-SNCA is a **preclinical, subcutaneously administered small interfering RNA (siRNA) drug** developed using Arrowhead Pharmaceuticals’ TRiM platform, designed to silence the SNCA gene encoding **alpha-synuclein**[1][2][3][4][5]. By inhibiting expression of alpha-synuclein, a protein known to misfold and aggregate in neurological diseases such as **Parkinson’s disease and other synucleinopathies**, it aims to arrest disease progression upstream of existing pathology[1][6][7][8]. The approach of RNA interference distinctly reduces the production of pathogenic protein instead of clearing existing aggregates[1][3][5][6]. Novartis acquired the exclusive rights to worldwide development, manufacturing, and commercialization through a licensing agreement with Arrowhead[1][3][4][5][6][8]. The drug has shown **promising preclinical results, including effective delivery to central nervous system and deep brain regions via subcutaneous injection**[1][3][5][6]. No human clinical data has been reported yet.