UX016

UX016 is an investigational oral prodrug of sialic acid (N-acetylneuraminic acid) being developed by Ultragenyx Pharmaceutical for the treatment of GNE myopathy, a rare genetic muscle-wasting disorder. The disease is caused by mutations in the GNE gene, which encodes an enzyme critical for the biosynthesis of sialic acid, leading to deficient sialylation of muscle proteins. UX016 acts as a substrate replacement therapy; as a prodrug, it is designed to improve bioavailability and cellular uptake compared to standard sialic acid. Once metabolized into active sialic acid, it bypasses the metabolic defect to restore protein sialylation and potentially preserve muscle function. The asset was licensed from the University of California, San Diego, and has received support from the GNE Myopathy Research Fund for its clinical development.

Other names

Aceneuramic Acid Prolonged-Releasesialic acid prodrugN-acetylneuraminic acid prodrug