RARE
Ultragenyx Pharmaceutical is a biopharmaceutical company focused on rare and ultra-rare genetic diseases with approved therapies and clinical-stage product candidates across multiple modalities including monoclonal antibodies, enzyme replacement therapy, small molecules, AAV gene therapy, and nucleic acids. Approved products include Crysvita, a fully human monoclonal antibody targeting FGF23 for X-Linked Hypophosphatemia and Tumor-Induced Osteomalacia, and Mepsevii, an intravenous enzyme replacement therapy for Mucopolysaccharidosis VII. The company retains global commercialization rights to its products and maintains a commercial organization across North America, the European Union, the United Kingdom, Latin America, and select international markets.