A Clinical Trial of ARCT-032 For Adults with Cystic Fibrosis
Cystic fibrosis is a life-shortening disease with a worldwide distribution. Mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene result in a reduction or absence of CFTR protein and/or function in the airways, which causes drying of the airway surfaces and thick, sticky airway mucus.
ARCT-032 (study treatment) is an inhaled messenger RNA (mRNA) therapy. It aims to deliver a normal copy of CFTR mRNA to lung cells using a lipid-mediated delivery system called LUNAR®.
Lung cells then use the instructions in the mRNA to create functional CFTR protein.
