Close to 130,000 people globally are affected by cystic fibrosis, a rare genetic disease which without treatment leads to respiratory infections and respiratory failure. Cystic fibrosis (CF) causes thick, sticky mucus to build up in the lungs, digestive tract, and other organs because of mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. These mutations prevent chloride from moving to the cell surface, and prevent the movement of water in tissues, which is necessary for preventing the buildup of mucus in CF. When the CFTR protein isn’t working properly, epithelial cells that line the passageways of the lungs, pancreas, and other organs produce mucus that can clog airways and ducts, making it difficult to breathe and digest food. Despite the advancement made in CF with current therapies, up to 15% of patients do not benefit from current therapies.
RIG-301 could offer a potential effective treatment for a broader range of CF patients as compared to current clinical interventions.