For Avery and Addison, we go beyond every day

Advancing medicines for rare diseases

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Leading with purpose. Every day.

“Ultragenyx was founded to advance innovative medicines for rare and ultrarare diseases that have never been treated before. We are delivering transformative therapies across multiple indications, and we have one of the most robust and diverse clinical pipelines in rare disease. Our focus is on doing the right things for patients both during development and commercialization to deliver on the promise of these therapies in a way that's meaningful for rare disease communities.”

Emil D. Kakkis, M.D., Ph.D.
Chief Executive Officer and President

Leadership

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Our team of passionate biopharmaceutical leaders combines deep expertise in drug development and commercialization.

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Pipeline

Pipeline Card

We have one of the largest and most diverse pipelines in rare disease.

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Grow with us

Grow with us

We’re growing fast and expanding across functions, including on our Commercial and Medical Affairs teams! Join us to lead the charge in shaping the future of rare disease medicine!

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Latest news

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July 14, 2025

Health Canada Extends the Approval of Evkeeza® (evinacumab) to Children as Young as 6-months Old with Homozygous Familial Hypercholesterolemia (HoFH)

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July 11, 2025

Ultragenyx Receives Complete Response Letter from FDA for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA)

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July 09, 2025

Ultragenyx and Mereo BioPharma Announce UX143 Phase 3 Orbit Study for Osteogenesis Imperfecta Progressing to Final Analysis

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Rare Perspective Home

Rare perspective

Insights and information on our approach to collaborating with rare disease communities, supporting our teammates at Ultragenyx, and developing new therapies for rare disease

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