In cystic fibrosis, inhaled CFTR mRNA (ARCT-032) advanced to phase 2 after early phase 1 data demonstrated safety and tolerability.<h4>Discussion</h4>Evidence for non-viral nanoparticle-mediated mRNA therapeutics is strong in preclinical research and increasingly promising in clinical applications beyond vaccinology. The gene discussed is CFTR; the disease is cystic fibrosis.