Ultragenyx focuses on developing therapeutics for rare and ultra-rare disorders, leveraging experienced rare-disease development specialists. Its lead product is UX-001, a first-in-class therapy for Hereditary Inclusion Body Myopathy (HIBM) expected to enter the clinic in 2011. The company plans to advance multiple rare disease product programs, create new product candidates, and pursue partnerships. Ultragenyx was formed and initially funded by Emil D. Kakkis in April 2010, drawing on his prior experience at BioMarin. The organization emphasizes efficient development strategies for indications with limited precedents and incomplete information. Early financial and strategic support comes from institutional and individual backers.

Total raised

$45M

Funding rounds

1

Latest round

Series A

Latest activity

Jun 2011