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AT-1001 is an orally administered zonulin receptor antagonist developed for treatment of Celiac Disease.
Zonulin is an endogenous signaling protein that transiently and reversibly opens the tight junctions between the cells of epithelial and endothelial tissues such as the intestinal mucosa, blood brain barrier and pulmonary epithelia. Zonulin appears to be involved in many disease states in which leakage occurs via paracellular transport across epithelial and endothelial tight junctions, and thus may play an important potential role in the treatment of autoimmune and inflammatory diseases.
Celiac disease is a T-cell mediated auto-immune disease that occurs in genetically susceptible individuals and is characterized by small intestinal inflammation, injury and intolerance to gluten. According to the National Institutes of Health, CD affects approximately 3 million Americans. The only current treatment for CD is complete elimination of gluten from the diet, which results in remission for some patients.