This session will describe recent advances towards treatment of cystic fibrosis, and the pipeline for new and emerging therapies. Interventions that target important disease manifestations, emerging small molecules designed to overcome rare and fundamental defects in CFTR, and molecular repair of the CF gene will be discussed.
Describe leading-edge programs directed towards cystic fibrosis palliation, including novel experimental therapies intended to overcome progressive tissue injury.
Compare and distinguish among major initiatives pursuing gene editing as a means to effectively treat a diversity of CF subclasses.
Summarize parallel development of experimental treatment strategies, and the prevailing rationale intended to advance new and effective therapies for the disease.