Ultragenyx cuts a fifth of staff, ends Wilson disease program

Ultragenyx said on Tuesday it will cut its headcount by about 20 percent to roughly 1,000 employees, terminate its UX701 gene therapy for Wilson disease after the program missed its study goals, and wind down its GTX-102 studies in Angelman syndrome.

Why it matters: The cuts are the price of the company's pivot from a broad gene therapy pipeline to commercial execution after its first two FDA approvals, with 2027 research spending guided to less than half its 2025 level and profitability reaffirmed for 2027.

Data as of Ultragenyx third quarter 2026 release issued November 3, 2026 via Globe Newswire, read in this run; figures and program decisions are the company's own. Endpoints News reporting the same day is consistent. Developing item.

Ultragenyx used its third quarter results on Tuesday to announce a broad cost reset: headcount falls by about 20 percent to a workforce of roughly 1,000, the UX701 program for Wilson disease is terminated after not achieving its study goals, and all ongoing studies of GTX-102 for Angelman syndrome will be wound down. The company expects the actions to cut 2027 research and development expenses by at least 50 percent against 2025, to less than $350 million, with selling, general and administrative expenses below $375 million, and it reaffirmed guidance for profitability in 2027.

The restructuring follows a quarter in which the FDA approved the company’s first two gene therapies, for glycogen storage disease type Ia and Sanfilippo syndrome type A, within a month of each other. Third quarter product revenue included $109 million from Crysvita and $30 million from Dojolvi, and the company said launches of the newly approved treatments are progressing with growing demand.

Ultragenyx cuts a fifth of staff, ends Wilson disease program

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