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Ultragenyx to cut about 20% of staff and discontinue Wilson disease gene therapy

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On November 3, 2026, Ultragenyx said it plans to cut about 20% of its workforce, leaving roughly 1,000 employees. The company expects the reductions to be largely complete by year-end. The company is ending development of UX701, its investigational AAV9 gene therapy for Wilson disease. In the Cyprus2+ dose-finding study, some patients showed clinical activity, but the results did not meet the company’s goal for most patients to achieve a biochemical response and stop standard treatment. Ultragenyx said UX701 had a favorable safety profile; the decision was based on not meeting efficacy goals, not a safety concern. The company plans to share study results at a future scientific meeting. The cuts are part of a broader cost-reduction push as Ultragenyx shifts toward commercial execution and aims for profitability in 2027. Other measures include winding down ongoing GTX-102 studies for Angelman syndrome and reducing real-estate and other expenses. Ultragenyx estimated about $25 mil...

Boehringer Ingelheim and Sino announce potential $7B-plus cancer-drug deal

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T-Therapeutics raises $133 million to advance cancer and autoimmune therapies

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Evident Vascular wins FDA clearance for AI-enabled IVUS platform

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Tecvayli moves toward first-relapse use, with uptake shaped by logistics

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Debate grows over HHS policy allowing limited compounding of six unapproved peptides

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Cepheid wins FDA clearance for multi-virus hemorrhagic fever test

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Sanofi ends Nurix STAT6 degrader program soon after Phase 1 launch

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AbbVie licenses Nxera’s deprioritized IBD drug in deal worth up to $1.47 billion

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Servier’s $1.55B sevasemten bet hits pivotal trial setback

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CVS plans to separate its pharmacy and healthcare businesses

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Midstream Health raises $20 million to improve hospital finances

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What the US midterm elections could mean for biopharma

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