For Kylie, 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 ultra-rare diseases that have never been treated before and we are delivering transformative therapies across multiple indications. 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 are advancing a pipeline of investigational therapies for rare and ultra-rare diseases.

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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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November 03, 2026

Ultragenyx Reports Third Quarter 2026 Financial Results, Announces Substantial Expense Reductions, and Reaffirms Profitability in 2027

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October 27, 2026

Ultragenyx to Host Conference Call for Third Quarter 2026 Financial Results and Corporate Update

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October 14, 2026

Ultragenyx Enters into Agreement to Sell Rare Pediatric Disease Priority Review Voucher for $203 Million

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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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