VRTX

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, and Alyftrek for cystic fibrosis, where Vertex therapies remain the standard of care globally. Vertex is focused on diversifying its portfolio through Casgevy (gene-editing therapy for beta thalassemia and sickle-cell disease), Journavx (non-opioid pain drug for moderate-to-severe acute pain), and Palsonify (for acromegaly). Additionally, Vertex is evaluating small-molecule inhibitors of APOL1-mediated kidney diseases, cell therapies to deliver a functional cure for type 1 diabetes, povetacicept (for IgA nephropathy).