Glycomine is targeting orphan disorders of glycosylation (sugar-coating of proteins). Remarkably, 50% of all proteins in our bodies are glycosylated. Attachment of specific sugars to the surface of proteins is required for correct protein folding and/or stabilization, dictates the adhesion and migration pattern of various immune cells, and controls the solubility and activity (protein-protein binding) of many enzymes. Patients with deficiencies in glycosylation have devastating and life-threatening disease with no therapeutic options. Glycomine is developing a means of delivering a sugar intermediate to cells that helps restore normal glycosylation in pre-clinical studies.Glycomine’s lead program seeks to provide treatment for Congenital Disorder of Glycosylation Type Ia(CDG). This disorder is severely debilitating and life-threating. Approximately 20% of suffers do not survive the first 5 years of life due to multiple organ failure. Living patients exhibit hypotonia (low
muscle tone), failure to thrive, hepathopathy (liver disease), coagulopathy (bleeding and abnormal
clotting), and chronic diarrhea and infections. Glycomine’s drug development program holds the
promise to provide the first therapies for patients and caregivers struggling with CDG.