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Cystic fibrosis treatment: targeting the basic defect

ABSTRACT Introduction: Cystic Fibrosis (CF) is a disease caused by different class mutations in the CF transmembrane conductance regulator (CFTR) gene. It can therefore benefit from a personalized medicine approach based on the individual genotype of each patient. Areas covered: This review provides a detailed overview of the current major development of new CF treatments that target the basic CF defect. The review summarizes gene therapy, mRNA repair strategies, read-through agents, and CFTR-modulators (potentiators, correctors, stabilizers, amplifiers and different combination therapies). Expert opinion: We are currently perhaps at the most exciting stage in the history of CF, with the potential to cure the disease now on the horizon. The good results obtained with ivacaftor in patients with at least one gating mutation have encouraged researchers to develop agents targeting the basic CF defect; such agents are designed for use as monotherapy or combination therapy in patients with other genotypes. However, disease aspects such as pharmacoeconomics, drug–drug interactions, use in infants, or the need for additional endpoints in future clinical trials, may ultimately hinder research and the potential availability of novel drugs for CF patients.