Empowered Patient Podcast

Dr. Charlotte McKee, Chief Medical Officer at Sionna, describes the nature of cystic fibrosis (CF, a genetic disease caused by a mutation in the single CFTR gene. While current CFTR modulator therapies do not address the most common mutation, Sionna's novel oral medicine is designed to target the previously undruggable NBDI domain of the CFTR protein. This new therapy aims to lead to better lung function and prevent the accumulation of permanent damage to other organs like the pancreas, gut, and liver.  Charlotte explains, "Cystic fibrosis is a genetic disease. The gene was actually discovered in 1989 for cystic fibrosis. It's considered a rare disease, but it's a relatively large rare disease. And one of those rare diseases that is potentially fatal, as you mentioned, it's thought of as a lung disease. And most patients, if their life is shortened, it's typically because of lung disease, because the lungs can be very severely affected. But the protein is caused by a genetic mutation in a gene called CFTR, a