Ultragenyx Cuts About 20% of Staff, Ends Two Rare Disease Drugs

Ultragenyx Pharmaceutical, a rare disease drugmaker based in Novato, California, is cutting about 20% of its workforce. The company disclosed the cuts on Nov 3, 2026, alongside its third quarter results. It is also ending work on two experimental treatments, one for Angelman syndrome and one for Wilson disease.

What happened

Ultragenyx said the reduction will leave it with approximately 1,000 employees. It expects the job cuts to be mostly done by the end of 2026. The company expects about $25 million in severance and other one-time restructuring charges in the fourth quarter.

Two programs are being dropped:

• GTX-102 for Angelman syndrome. Ultragenyx is winding down all remaining studies. In September, the drug failed a key study, missing both its primary and secondary goals, according to BioPharma Dive.
• UX701 for Wilson disease. The company is ending this gene therapy because it did not meet its study goals. Ultragenyx said some patients responded, but most did not reach the bar it had set.

The company also plans to shrink its real estate footprint. It expects 2027 research and development spending to fall below $350 million. That would be at least a 50% drop from 2025. It expects sales and administrative costs below $375 million in 2027.

Why it matters

GTX-102 was once seen as a possible blockbuster. Some analysts had estimated peak sales of $1.8 billion or more, BioPharma Dive reported. Angelman syndrome has no approved treatment. When the study failed, Ultragenyx lost almost half its value in a single trading day.

The company is now leaning on drugs it already sells. Third quarter revenue was $173 million, including $109 million from Crysvita. Its net loss narrowed to $118 million, from $180 million a year earlier.

Cash is a pressure point. Ultragenyx held $401 million in cash and marketable securities at Sept 30, 2026, down from $737 million at the end of 2025. It agreed in October to sell two priority review vouchers for a combined $413 million. It expects net proceeds of about $350 million. The company says it still expects a full-year profit in 2027.

The vouchers came from two gene therapy approvals this year. The FDA approved Genglycos in August and Fayuvi in September.

What it means for people in the industry

This is not the first cut at Ultragenyx this year. It reported $30 million in restructuring costs in the first nine months of 2026. The company's release did not say which sites or teams are affected.

Research work will narrow. Ultragenyx will focus on a phase 3 gene therapy for OTC deficiency, with more data expected in the first half of 2027. It will also look for a path forward for setrusumab in brittle bone disease. Watch for regulator feedback on setrusumab and for the OTC data next year.

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Sources

• Ultragenyx Reports Third Quarter 2026 Financial Results, Announces Substantial Expense Reductions, and Reaffirms Profitability in 2027, Ultragenyx (SEC filing)
• Ultragenyx cuts staff after another study fails, STAT
• Ultragenyx to weigh 'significant' cost cuts as Angelman drug fails key study, BioPharma Dive