Ultragenyx Cuts About 20% of Staff and Scraps Wilson Disease Gene Therapy UX701
- Ultragenyx is cutting roughly 20% of its workforce, leaving about 1,000 employees, and shrinking its real estate footprint.
- UX701 for Wilson disease is terminated: the fourth cohort showed clinical activity in some patients but not enough to move to the pivotal stage.
- Angelman GTX-102 studies are being wound down; the company says it is pivoting to commercial execution after its first two FDA approvals.
💡 Points Based on public reports
This is the second flagship exit in two months, following the September Angelman Phase 3 miss, so the pipeline now rests on the two newly approved products. For UX701 the bar was also high: Wilson disease already has inexpensive oral therapies, so a gene therapy needed a clear functional edge. What to watch is which mid-stage assets survive the smaller R&D footprint.
Ultragenyx to lay off 20% of employees, scrap a Wilson disease gene therapy ↗