As our knowledge for CFTR structure has improved, searches for mutation-specific as well as mutation-independent tactics have begun to emerge and open the path to revolutionizing our approach in treating CF patients. The presenters will share preclinical data for innovative therapeutic strategies, and describe new biomarkers useful in clinical trials, focusing on the infant cohort.
Discuss the potential development and therapeutic applications of 2 mutation independent strategies aiming to repair the CFTR gene (gene therapy) or the airway tissue (stem cell therapy).
Discuss the rationale for triple combination therapeutic regimens for F508Del-CFTR.
Assess knowledge into novel biomarkers for clinical trials in cystic fibrosis.