VRTX

Vertex Pharmaceuticals Incorporated is a global biotechnology company. The Company is focused on investing in scientific innovation to create transformative medicines for people with serious diseases, with a focus on specialty markets. It 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). It 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.