P079 Personalized CFTR modulator therapy for G85E and N1303K homozygous patients with cystic fibrosis

Abstract

Objectives: The cystic fibrosis (CF) transmembrane conductance regulator (CFTR) modulators elexacaftor/tezacaftor/ivacaftor (ETI) are approved for people with CF (pwCF) and at least one F508del-allele in Europe. In the US, ETI label was expanded by >100 rare CFTR mutations responsive in fisher rat thyroid cells, including G85E, but not N1303K. However, the effect of ETI on G85E or N1303K CFTR proteins is unknown.