Vertex Pharmaceuticals Incorporated - VRTX

Vertex Pharmaceuticals Incorporated, a biotechnology company, develops medicines for serious diseases. The company focuses on developing and commercializing therapies for the treatment of cystic fibrosis (CF) and advancing its research and development programs. It markets ORKAMBI (lumacaftor in combination with ivacaftor) to treat patients with CF 12 years of age and older who are homozygous for the F508del mutation in their cystic fibrosis transmembrane conductance regulator (CFTR) gene; KALYDECO (ivacaftor) for the treatment of patients with CF who have specific mutations in their CFTR gene, including the G551D mutation; and SYMDEKO (tezacaftor in combination with ivacaftor) to treat patients with CF 12 years of age and older who are F508del homozygous or who have 1 mutation that is responsive to tezacaftor/ivacaftor. The company also develops VX-659 and VX-445, which are CFTR corrector compounds in Phase II clinical trial; VX-150 that is in Phase II clinical trial to treat pain; VX-128, which is in Phase I clinical trial for the treatment of pain; CTX001, an investigational gene editing treatment that is in Phase I/II trial for the treatment of beta-thalassemia and sickle cell disease; pimodivir (JNJ-63623872), which is in a Phase III clinical development program to treat influenza; and VX-210 to treat acute spinal cord injury. It sells its products primarily to specialty pharmacy providers in North America, as well as government-owned and supported customers internationally. The company has collaborations with Cystic Fibrosis Foundation Therapeutics Incorporated; CRISPR Therapeutics AG; Moderna Therapeutics, Inc.; BioAxone Biosciences, Inc.; Parion Sciences, Inc.; Merck KGaA; Janssen Pharmaceuticals, Inc; and Q-State Biosciences, Inc. The company was founded in 1989 and is headquartered in Boston, Massachusetts.