UltraGenyx Pharmaceutical

Novato, CA

Ultragenyx is a privately held biotechnology company focused on rare genetic disorders, founded by Emil Kakkis, MD, PhD, the former CMO of BioMarin, who is widely recognized as a leader in the rare disease field.  Its mission is to build a first-in-class rare disease company addressing proteins or small molecules with clear disease targets and treatment mechanisms which have been overlooked and are ready for development. Its lead program for a substrate replacement therapy for Hereditary Inclusion Body Myopathy (HIBM) has initiated Phase 1 trials.