VERTEX PHARMACEUTICALS INC / MA

Vertex Pharmaceuticals is a biotechnology company focused on developing and commercializing medicines for serious diseases with concentrations in specialty markets. The company's primary revenue comes from its cystic fibrosis (CF) franchise, which includes approved therapies such as TRIKAFTA/KAFTRIO, SYMDEKO/SYMKEVI, ORKAMBI, and KALYDECO. These CF treatments target patients with specific genetic mutations and represent the company's most established commercial business. Beyond CF, Vertex markets CASGEVY for sickle cell disease and transfusion-dependent beta thalassemia, representing an expansion into genetic blood disorders.

The company's pipeline includes treatments in clinical development across multiple therapeutic areas. JOURNAVX is in development for acute pain, while earlier-stage programs address conditions such as Type 1 Diabetes (VX-264), myotonic dystrophy type 1 (VX-670), autosomal dominant polycystic kidney disease (VX-407), and APOL1-mediated kidney disease (inaxaplin). VX-522, a CFTR mRNA therapeutic, is progressing through Phase 1/2 trials as a potential treatment targeting the underlying cause of cystic fibrosis.

Vertex operates with approximately 6,400 full-time employees and maintains a global presence across the United States, Europe, and international markets. The company distributes its products through specialty pharmacies, wholesalers, retail pharmacies, hospitals, and clinics. Founded in 1989 and headquartered in Boston, Massachusetts, Vertex is listed on Nasdaq.