At Ultragenyx, we’re going beyond every day to transform the lives of people living with rare diseases and lead the future of rare disease medicine. We built our team and our company from the ground up to pioneer new medicines and approaches to drug development for rare and ultrarare diseases and overcome the challenges inherent to treating small patient populations and previously untreated conditions. In 12 years, we’ve achieved four approved therapies in five indications, with a robust pipeline of late-stage therapeutic candidates for debilitating conditions. Our approach to development is informed by patient experience and insights, and we share our science and expertise to help move the entire field forward.

“Ultragenyx was founded to advance innovative medicines for rare and ultrarare diseases that have never been treated before. We are delivering transformative therapies across multiple indications, and we have one of the most robust and diverse clinical pipelines in rare disease. Our focus is on doing the right things for patients both during development and commercialization to deliver on the promise of these therapies in a way that's meaningful for rare disease communities.”

Emil D. Kakkis, M.D., Ph.D.

Chief Executive Officer and President