Ultragenyx Pharmaceutical Inc.

We are a biopharmaceutical company founded in April 2010 by President and Chief Executive Officer, Emil Kakkis, M.D., Ph.D. We focus on developing and commercializing novel products for patients with serious rare and ultra-rare genetic diseases. Our portfolio includes approved therapies and product candidates for diseases with high unmet medical need and clear biology, often lacking approved treatments for the underlying condition. Our business strategy centers on time- and cost-efficient drug development to deliver safe and effective therapies. We concentrate on rare and ultra-rare genetic diseases that currently have no approved drug therapy treating the underlying disease, often causing significant morbidity and/or mortality. We prioritize diseases with well-understood biology, believing that targeting known disease pathways increases development program success. Our treatment modalities include biologics, small molecules, adeno-associated virus (AAV) gene therapy, and nucleic acids. We generally in-license promising product candidates from academic institutions or derive them from partnerships with other pharmaceutical companies, retaining global commercialization rights.