ARO-ENaC

ARO-ENaC is an investigational inhaled RNA interference (RNAi) therapeutic developed by Arrowhead Pharmaceuticals for the treatment of cystic fibrosis (CF). It is designed to selectively reduce production of the epithelial sodium channel alpha subunit (αENaC) in the airways of the lung. Overactivity of ENaC in CF patients leads to airway dehydration and impaired mucociliary clearance, contributing to persistent lung infections and progressive loss of pulmonary function. By delivering a small interfering RNA (siRNA) molecule via inhalation using Arrowhead’s proprietary Targeted RNAi Molecule (TRiM) platform, ARO-ENaC targets αENaC mRNA for degradation, thereby reducing protein expression specifically in the lungs while sparing other organs such as the kidney. This approach aims to overcome limitations seen with previous small molecule ENaC inhibitors related to renal toxicity and short duration of action[2][4][5][6][8].