Ultragenyx

Ultragenyx Pharmaceutical Inc. was founded to advance innovative medicines for rare and ultrarare diseases that have never been treated before. The company is dedicated to delivering transformative therapies across multiple indications, focusing on rare diseases, which often lack effective treatments. With one of the most robust and diverse clinical pipelines in the rare disease sector, Ultragenyx aims to catalyze progress in this field. The company's commitment extends beyond just drug development; they focus on doing the right things for patients both during the development phase and commercialization. This is crucial for delivering on the promise of these therapies in ways that are meaningful to rare disease communities.

Based in the United States, Ultragenyx strives to develop first-ever approved treatments for various rare and ultrarare genetic diseases. As they advance their pipelines, the company engages with rare disease communities to understand their unique challenges and needs. The insights gleaned through these collaborations inform Ultragenyx’s approach to therapy development. Overall, Ultragenyx believes in moving the rare disease community forward through innovative and responsible therapeutic solutions.