Keyword: Ultragenyx

Ultragenyx is abandoning its glucose transporter type-1 deficiency syndrome program after its drug UX007 missed its endpoints in a phase 3 trial.

David Thompson has left Azure Biotech to join Inozyme Pharma as chief scientific officer as the company transitions to the clinical stage.

Bob Hugin retires from Celgene, Voyager founder steps down as CEO, Boehringer named new U.S. chief to replace retiring Fonteyne.

Ultragenyx Pharmaceuticals chalked up another win for its rare bone disease drug, burosumab, keeping it on track for a U.S. regulatory filing.

The deal will give Arcturus a route to public investors at a time when interest in RNA drugs is flying high on the back of Alnylam’s phase 3 success.

Ultragenyx is putting up $138 million in cash to wrestle gene therapy biotech Dimension away from its first suitor.

A GNE myopathy trial missed its primary endpoint and all key secondary endpoints, delivering a blow to Ultragenyx as it enters a critical period.

Ultragenyx needed a win for its lead drug burosumab after a midstage pipeline failure and delivered with a good set of phase 3 results.

Shares in Ultragenyx weakened after a midpipeline drug candidate missed its targets in a trial for a rare genetic disorder.

Despite only being a very small trial and with lingering questions over the primary endpoint, analysts were overall positive on Ultragenyx’s late-stage data for the incredibly rare genetic, metabolic disorder mucopolysaccharidosis 7.