Each day more are being discovered. The majority of rare diseases do not have even a single FDA approved treatment. Glycomine is developing therapeutics for diseases which have no treatment options.
We focus on genetic disorders of protein and lipid glycosylation, which often cause severe debilitation in patients. There are 100 known glycosylation disorders. Glycomine is currently addressing two disorders of glycosylation: PMM2-CDG and Ngly1 deficiency.
Glycomine’s therapeutic approach is to combine replacement therapies – substrates, enzymes, or proteins – with intracellular delivery vehicles consisting of bio-nanomaterials or ligands that target the molecules to the cell interior of clinically relevant organs.
Our PMM2-CDG program is currently in early pre-clinical development. We are at the early stages of research for Ngly1 deficiency.