Ultragenyx Raises $45M in Series A to Progress Lead Orphan Disease Candidate into Clinic

Orphan disease therapeutics startup Ultragenyx Pharmaceutical raised $45 million in a Series A financing round co-led by TPG Biotech and Fidelity Biosciences. Additional investors included HealthCap and Pappas Ventures.

Ultragenyx will use the funds to expand and progress its rare disease products pipeline, including lead candidate UX-001, for the treatment of hereditary inclusion body myopathy, which is expected to start in clinical development during 2011. UX-001 is an extended-release formulation of sialic acid.

The firm’s pipeline currently includes four other lysosomal disease/enzyme therapy candidates. It projects another two products may reach the clinic within the next one to two years. Founded in 2010 by Emil Kakkis, M.D., formerly BioMarin Pharmaceuticals’ CMP, Ultragenyx aims to select and develop candidates with clear mechanisms of action against untreated rare diseases.