The drug discovery and development pipeline for CF has never been more active. In this session, we will begin by reviewing the state of play with small molecule CFTR modulators, currently clinically available in single and dual agent form and in trials as triple agents. Recognizing that patients with certain, rarer mutations are more difficult to treat, presenters will then look to the future, considering best ways of testing drugs for these groups and reviewing progress with splice and nonsense mutations.
Review the current landscape of CFTR modulating agents and other drugs targeting the basic CFTR defect.
Illustrate the challenges inherent in drug development for patients with rare gene mutations.
Use two examples of rare mutation groups (splice mutations and premature truncation codons) to describe preclinical progress and anticipate routes to the clinic.
Note: Speaker Batsheva Kerem's presentation was not recorded at her request.
M.D., MBChB, MRCP,
Prof - Paed Respirology & Experimental Medicine