Founded in 2010, Ultragenyx Pharmaceutical Inc. specializes in treatments for rare genetic diseases that typically lack approved therapies. The company's commercial portfolio includes Crysvita for X-linked hypophosphatemia, Dojolvi for long-chain fatty acid oxidation disorders, Evkeeza for homozygous familial hypercholesterolemia, and Mepsevii for mucopolysaccharidosis type VII. Ultragenyx operates a diverse clinical pipeline spanning biologics, small molecules, AAV gene therapies, and mRNA therapeutics. The company maintains a global footprint with commercial operations in North America, Europe, Latin America, and Asia, alongside a dedicated gene therapy manufacturing facility in Massachusetts. By focusing on diseases with clear biology and small patient populations, Ultragenyx employs a specialized approach to drug development and commercialization.
