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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.
Gilead Sciences develops and markets therapies to treat life-threatening infectious diseases, with the core of its portfolio focused on HIV and hepatitis B and C. Gilead's acquisition of Pharmasset brought rights to hepatitis C drug Sovaldi, which is also part of newer combination regimens that remain standards of care. Gilead is also growing its presence in the oncology market via acquisitions, led by CAR-T cell therapy Yescarta/Tecartus (from Kite) and breast and bladder cancer therapy Trodelvy (from Immunomedics).