Vertex Pharmaceuticals Inc. is a Boston-based biotechnology company focused on the discovery, development and commercialization of therapies for serious diseases. Founded in 1989, Vertex built its reputation on research-driven drug development and is best known for its work in cystic fibrosis (CF), where its portfolio of small-molecule CFTR modulators transformed standards of care for many people with the disease. The company operates research and development, manufacturing and commercial organizations and serves patients and healthcare systems in multiple international markets.
Vertex’s marketed products center on CFTR modulators that target the underlying cause of cystic fibrosis rather than just treating symptoms. Its approved therapies include potentiators and corrector combinations that have expanded treatment options for people with different CFTR mutations. These drugs, introduced progressively since the early 2010s, are the cornerstone of Vertex’s commercial business and have enabled broad access programs and partnerships to reach CF patients globally.
Beyond cystic fibrosis, Vertex has invested heavily in next‑generation technologies and new therapeutic areas. The company is advancing programs in gene editing and cell therapy, including a collaboration with CRISPR Therapeutics to develop gene‑editing treatments for hemoglobinopathies such as sickle cell disease and beta‑thalassemia, and it has pursued stem cell–derived approaches for type 1 diabetes following its acquisition of Semma Therapeutics. Vertex’s pipeline also includes earlier‑stage research across other serious diseases where precision genetic or cell‑based approaches may offer durable benefit.
Vertex maintains a commercial presence in North America, Europe and other international markets while sustaining a large R&D footprint to support discovery and clinical development. The company is led by Chief Executive Officer Reshma Kewalramani and a management team with deep experience in both clinical development and commercial operations. Vertex emphasizes patient access, long‑term innovation and collaboration with academic and industry partners as it seeks to expand its impact beyond cystic fibrosis into additional rare and serious diseases.