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

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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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February 05, 2025

Ultragenyx Announces New Data Demonstrating that Treatment with UX111 AAV Gene Therapy Significantly Improved Clinical Function Across Multiple Developmental Domains in Children with Sanfilippo Syndrome Type A (MPS IIIA) Correlated with Sustained Reductions in CSF-HS

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January 17, 2025

Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)

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January 12, 2025

Ultragenyx Reports Preliminary 2024 Revenue, Financial Guidance for 2025, Pipeline Updates, and 2025 Milestones

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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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