Vertex Pharmaceuticals, Inc. is a global biotechnology company, which engages in the business of discovering, developing, manufacturing, and commercializing small molecule drugs for patients with serious diseases. The company is headquartered in Boston, Massachusetts and currently employs 5,400 full-time employees. The firm is focused on investing in scientific innovation to create transformative medicines for people with serious diseases, with a focus on specialty markets. The company has four approved medicines that treat the underlying cause of cystic fibrosis (CF), and one approved therapy that treats severe sickle cell disease (SCD) and transfusion dependent beta thalassemia (TDT). Its pipeline includes clinical-stage programs in CF, SCD, TDT, acute and neuropathic pain, APOL1-mediated kidney disease, type 1 diabetes, myotonic dystrophy type 1 and alpha-1 antitrypsin deficiency. Its marketed medicines are TRIKAFTA/KAFTRIO, SYMDEKO/SYMKEVI (tezacaftor/ivacaftor and ivacaftor), ORKAMBI (lumacaftor/ivacaftor) and KALYDECO (ivacaftor). The company is developing non-autologous (allogeneic) fully differentiated, stem-cell derived islet cell therapies designed to replace insulin-producing islet cells that are destroyed in people with T1D.