Post by Ultragenyx

Today we announced that the U.S. FDA has cleared the Investigational New Drug (IND) application for UX016, a small molecule prodrug of sialic acid being evaluated as a substrate replacement therapy for GNE myopathy.  The UX016 program is externally funded through clinical proof-of-concept, including a Phase 1/2 study expected to begin in the second half of 2026. Read the full announcement: https://lnkd.in/gMMPtEmt