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 rare and ultra-rare diseases, with a focus on serious, debilitating genetic diseases. Its pipeline consists of two product categories: biologics, including a monoclonal antibody and enzyme replacement therapies, and small-molecule substrate replacement therapies. Its products are KRN23 (UX023), rhGUS (UX003), rhPPCA (UX004), Triheptanoin (UX007) and SA-ER (UX001). KRN23 is a fully human monoclonal antibody administered via subcutaneous injection. rhGUS is an intravenous (IV), enzyme replacement therapy for the treatment of mucopolysaccharidosis 7. rhPPCA is an enzyme replacement therapy for galactosialidosis. Triheptanoin is a medium odd-chain triglyceride of seven-carbon fatty acids. SA-ER is for the treatment of hereditary inclusion body myopathy (HIBM).