Clinical Development

  Clinical Development / Medical Need

Medical Need

Celiac disease, also known as celiac sprue, is a T-cell-mediated, chronic inflammatory disease of the intestine caused by an auto-immune-like reaction to dietary gluten proteins in genetically susceptible individuals. When a person with celiac disease consumes gluten proteins in wheat, rye and barley, the individual’s immune system responds by triggering T-cells to fight the offending proteins (antigens), causing inflammation of the small intestine and inhibiting the absorption of important nutrients into the body.

Medical Need

Genetic testing indicates the vast majority of individuals diagnosed with celiac disease carry the two genes that code for the immune recognition protein named HLA-DQ2.5, the remaining 10% of patients have genes for the closely related HLA-DQ2.2 or HLA-DQ8. These immune recognition proteins selectively bind and present peptide fragments from partially-digested gluten to T cells, facilitating the immune reaction causing celiac disease.

Celiac disease is becoming increasingly prevalent, and it is estimated that the disease currently affects 2 to 3 million Americans and about 1% of the population in North America, Europe, Australia, the Middle East and North India. Over 80% of cases remain unrecognized in America and Europe, and those diagnosed experience symptoms for many years and undergo a variety of medical investigations before being tested for celiac disease.

Currently, diagnosis of celiac disease involves a blood test for the detection of specific antibodies, followed by a gastroscopy and collection of biopsies from the small intestine that are examined for inflammatory damage typical of untreated celiac disease. Unfortunately, these methods are invasive and inconvenient, but also unreliable when gluten has been removed from the diet for an extended period of time.

With rapidly increasing numbers of patients being diagnosed with celiac disease, there is a growing need for improved, less intrusive treatments and diagnostics. The only intervention available today is a strict, lifelong gluten-free diet (GFD), which imposes a significant burden, negatively affects quality of life, and can be difficult to follow since even minute amounts can trigger an immune reaction. Additionally, GFD is not a desirable or healthy diet unless there is a medical need to avoid gluten, as the glycemic content and poor nutritional value may lead to increased risk for metabolic syndrome. A disease-modifying approach that resets the immune system when exposed to gluten would allow patients to return to good health and improved quality of life.


  1. European Society for Pediatric Gastroenterology, Hepatology, and Nutrition guidelines for the diagnosis of coeliac disease.
  2. Husby S, Koletzko S, Korponay-Szabó IR, Mearin ML, Phillips A, Shamir R, Troncone R, Giersiepen K, Branski D, Catassi C, Lelgeman M, Mäki M, Ribes-Koninckx C, Ventura A, Zimmer KP; ESPGHAN Working Group on Coeliac Disease Diagnosis; ESPGHAN Gastroenterology Committee; European Society for Pediatric Gastroenterology, Hepatology, and Nutrition. J Pediatr Gastroenterol Nutr. 2012;54(1):136-60.
  3. Taylor KB, Truelove SC, Thomson DL, Wright R. An immunological study of coeliac disease and idiopathic steatorrhoea. Serological reactions to gluten and milk proteins. Br Med J. 1961;2(5269):1727-31.
  4. Anderson, WH, and Mackay, IR.  Gut reactions – from celiac affection to autoimmune model.  N Engl J Med. 2014; 371 (1): 6-7.
  5. Sollid LM, Jabri B. Triggers and drivers of autoimmunity: lessons from coeliac disease. Nat Rev Immunol. 2013;13(4):294-302.
  6. Anderson RP, Jabri B. Vaccine against autoimmune disease: antigen-specific immunotherapy. Curr Opin Immunol. 2013;25(3):410-7.
  7. Choung RS, Ditah IC, Nadeau AM, Rubio-Tapia A, Marietta EV, Brantner TL, Camilleri MJ, Rajkumar SV, Landgren O, Everhart JE, Murray JA. Trends and racial/ethnic disparities in gluten-sensitive problems in the United States: findings from the National Health and Nutrition Examination Surveys from 1988 to 2012. Am J Gastroenterol. 2015;110(3):455-61.
  8. See JA, Kaukinen K, Makharia GK, Gibson PR, Murray JA. Practical insights into gluten-free diets. Nat Rev Gastroenterol Hepatol. 2015;12(10):580-91.