Vertex Pharmaceuticals (United States)

Vertex Pharmaceuticals is a global biotechnology company that transformed cystic fibrosis (CF) treatment. Based in Boston, key products include Trikafta, Symdeko, and Orkambi for cystic fibrosis. Vertex also developed Casgevy (exagamglogene autotemcel) with CRISPR Therapeutics, the first FDA-approved CRISPR gene-editing therapy for sickle cell disease and beta-thalassemia. Vertex’s research extends to pain, kidney disease, and other serious conditions.