WS14.02 Cystic fibrosis personalised medicine; paediatric in vitro airway cell models to predict CFTR modulator patient outcomes

Abstract

Objectives: CF is a multisystemic genetic disease with early mortality due to respiratory failure from obstructive lung disease. CFTR modulator drugs that target the dysfunctional disease causing chloride channel, demonstrated efficacy in improving clinical biomarkers. However, heterogeneity of response to drug was seen in clinical trials. Primary cell models, created from a patient’s own stem cells, have been proposed as an in vitro test to predict an individuals’ response to treatment. We aimed to investigate the relationship between matched paediatric in vitro cell models and the in vivo response to a CFTR modulator.