During this session, we will explore data supporting the development of therapies to address the basic defect in CF including gene editing, mRNA read-through, and CFTR protein modulation. The session will cover CFTR therapeutic development along the entire pathway from pre-clinical technical advancements and target identification to results of Phase I, II and III clinical trials.
1. Identify appropriate molecular targets for CFTR-directed therapeutics. 2. Review safety of potential new CFTR-directed therapies. 3. Critically evaluate efficacy data from CFTR modulator clinical trials.