DTX401

About Ultragenyx Pharmaceutical

Ultragenyx is a mission-driven, commercial-stage biotech dedicated to pioneering novel treatments for serious rare and ultra-rare genetic diseases. Founded in 2010, the company has successfully transitioned to a revenue-generating entity with three approved products and has built one of the largest and most diverse clinical pipelines in the rare disease sector. Its strategy employs a flexible, multi-platform approach—including gene therapy, mRNA, and enzyme replacement—to target the root cause of monogenic disorders. Led by CEO Emil D. Kakkis, M.D., Ph.D., Ultragenyx operates globally with a deep commitment to underserved patient communities.

Therapeutic Areas

Other Glycogen Storage Disease Type Ia (GSDIa) Drugs