ARCT-032

ARCT-032 is an investigational inhaled mRNA therapy developed to treat cystic fibrosis (CF). It uses a lipid-mediated aerosolized delivery platform (LUNAR technology) to deliver healthy copies of cystic fibrosis transmembrane conductance regulator (CFTR) mRNA directly to lung cells. This enables the production of functional CFTR protein in the lungs, aiming to restore chloride channel activity and address the underlying cause of CF regardless of specific genetic mutations. Unlike current CFTR modulators that are mutation-specific, ARCT-032 could potentially benefit all patients with CF. The drug has received orphan drug and rare pediatric disease designations from regulatory agencies in both the US and EU. Clinical trials have shown it is generally safe and well tolerated when administered via nebulizer[1][3][4][5][7].

Other names

mRNA CFTR therapeutic