Ultragenyxâ„¢ develops therapeutics for rare diseases, sometimes referred to as Orphan products. Founded in 2010, the company is led by Emil Kakkis, M.D., Ph.D., former CMO of BioMarin Pharmaceutical. Efficient and effective development strategies are essential for success in the rare disease space, in which information may be incomplete or ambiguous, and few development precedents exist. The management team's experience in selecting and developing products with clear mechanisms of action for untreated rare diseases will help Ultragenyx create life-changing therapeutics by leveraging existing yet undeveloped science for these little-known indications.