n-acetylmannosamine

N-acetylmannosamine (ManNAc) is an investigational small molecule monosaccharide being developed by Leadiant Biosciences, Inc., in collaboration with the National Institutes of Health (NIH), for the treatment of GNE myopathy. GNE myopathy is a rare, progressive genetic muscle disease caused by mutations in the GNE gene, which encodes the bifunctional enzyme UDP-N-acetylglucosamine 2-epimerase/N-acetylmannosamine kinase. This enzyme is critical for the biosynthesis of N-acetylneuraminic acid (sialic acid). ManNAc serves as the first committed precursor in the sialic acid pathway, bypassing the enzymatic defect to restore sialic acid production and improve protein sialylation in skeletal muscle. The drug has received orphan drug designation and has been evaluated in the Phase 2 MAGiNE clinical trial.

Other names

ManNAcN-acetyl-D-mannosamine monohydrate