Unmet Need

Cystic fibrosis (CF) is a rare genetic disease that causes persistent lung infections and, over time, limits the patient’s ability to breathe over time. In people with CF, a defective gene (CFTR) causes a thick, sticky build-up of mucus in the lungs, pancreas, and other organs. People with the disease inherit two copies of the defective CFTR gene, one from each parent. In the lungs, the mucus clogs the airways and traps bacteria, leading to recurrent infections, lung inflammation and damage, and, eventually, respiratory failure. Patients with CF are treated with physiotherapy, postural drainage and antibiotics, which together help remove mucus from their lungs, improve their ability to breathe, and address chronic lung infections.