Ultragenyx Pharmaceutical Inc. is a clinical-stage biopharmaceutical company. The Company is focused on the identification, acquisition, development, and commercialization of products for the treatment of genetic diseases. The Company is engaged in the identification, acquisition, development and commercialization of products for the treatment of rare and ultra-rare diseases segment. Its pipeline consists of two product categories: biologics, including a monoclonal antibody and enzyme replacement therapies, and small-molecule substrate replacement therapies. KRN23 is a fully human monoclonal antibody administered via subcutaneous injection. Recombinant human beta-glucuronidase (rhGUS), is an intravenous (IV), enzyme replacement therapy for the treatment of mucopolysaccharidosis 7 (MPS 7). It is developing UX007 for oral administration intended as a substrate replacement therapy. It is developing aceneuramic acid extended-release (Ace-ER) for the treatment of GNE myopathy.