Vertex Pharmaceuticals is a global biotechnology company that discovers and develops small-molecule drugs for the treatment of serious diseases. Its key drugs are Kalydeco, Orkambi, Symdeko, and Trikafta/Kaftrio for cystic fibrosis, where Vertex therapies remain the standard of care globally. Vertex has diversified its portfolio through Casgevy, a gene-editing therapy for beta thalassemia and sickle-cell disease. Additionally, Vertex is evaluating small-molecule inhibitors targeting acute and chronic pain using nonopioid treatments, and small-molecule inhibitors of APOL1-mediated kidney diseases. Vertex is also investigating cell therapies to deliver a potential functional cure for type 1 diabetes.