Cystic fibrosis causes mucus buildup in lungs and digestive system, affecting 40,000 in the U.S. CFTR modulators like Vertex's Trikafta treat symptoms by correcting the CFTR protein. Five biotech companies—Enterprise Therapeutics, Krystal Biotech, Recode Therapeutics, Sionna Therapeutics, and Vertex Pharmaceuticals—are developing transformative treatments, including nebulized formulations, gene therapies, and small molecules targeting CFTR mutations. The global cystic fibrosis therapeutics market is growing, but high drug prices remain a concern.