CFTR mRNA delivery with a novel non-LNP nanoemulsion formulation to cystic fibrosis differentiated human airway epithelium and airway organoid

Background: Cystic fibrosis (CF) is caused by dysfunctional mutations in the CF transmembrane regulator (CFTR) chloride channel. Currently approved CFTR modulators are not effective in all genotypes leaving some patients without a therapeutic option. A single universally applicable therapy that produces functional CFTR protein expression by CFTR gene delivery would benefit for all patients. Aim: T…