PMM2-CDG (also known as CDG-1A or CDG Type 1a) is a rare genetic disease of glycosylation. While currently approved therapies only treat symptoms and are not disease-modifying, Glycomine hopes to change that with the clinical development of GLM101, a mannose-1-phosphate replacement therapy.
Rare genetic diseases are often caused by genetic mutations that lead to protein dysfunction and disruption of biochemical pathways. Glycomine is developing replacement therapies, where delivering a substrate, enzyme, or protein has the potential to restore the missing function. GLM101 is Glycomine’s first drug from its platform and is currently in clinical trials in the U.S. and Europe for the treatment of PMM2-CDG.
Glycomine Announces Encouraging Efficacy Data from Ongoing Phase 2 Clinical Study in PMM2-CDG
The Phase 2 clinical study of GLM101, a potential treatment for PMM2-CDG, began in January 2023.
Data from Glycomine’s natural history study informs update to standard of care.
GLM101 is a mannose-1-phosphate replacement therapy in clinical development as a potential PMM2-CDG treatment.