GLM101

GLM101 is an investigational mannose-1-phosphate replacement therapy being developed by Glycomine for the treatment of phosphomannomutase 2-congenital disorder of glycosylation (PMM2-CDG), also known as CDG Type Ia. PMM2-CDG is a rare, life-threatening genetic disorder caused by mutations in the PMM2 gene, leading to a deficiency of the enzyme phosphomannomutase 2 and resulting in impaired glycosylation. GLM101 is designed to deliver mannose-1-phosphate directly into cells via intravenous infusion, thereby bypassing the defective enzyme and restoring pathway function. The active ingredient is alfa-D-mannopyranosyl phosphate dipotassium, formulated within liposomes for cellular delivery. GLM101 has received Orphan Drug Designation in both the U.S. and Europe as well as Rare Pediatric Disease Designation in the U.S., and it is currently being evaluated in Phase 2 clinical trials with demonstrated proof-of-concept data showing improvement in ataxia among patients with PMM2-CDG[1][3][5][6].

Other names

alfa-D-mannopyranosyl phosphate dipotassium