Erick Crombez, M.D., Ph.D., Chief Medical Officer, Ultragenyx Gene Therapy and Inborn Errors of Metabolism

Novato, CA


Virtual Presentation

Ultragenyx was founded to create a steady pipeline of new and effective therapies for patients with rare and ultra-rare diseases. It is our shared belief that we will achieve this by creating a people-first ethos that puts patient communities and our employees at the center of our approach and mission. We are targeting multiple therapeutic areas in which we have deep expertise: endocrine/bone diseases, metabolic diseases, and central nervous system/muscle diseases. We put patients and their caregivers at the center of our work, involving them at all stages of therapeutic development. This provides us with an understanding of the impact a disease has on a patient – biologically, functionally, and emotionally – so we can address the entire person and not just their symptoms. All of our efforts are rooted in our commitment to provide patients and their families with courage and confidence today.


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