The abstract titled, MONGERSEN, AN ORAL SMAD7 ANTISENSE OLIGONUCLEOTIDE, IN ACTIVE CROHN'S DISEASE, can be viewed here.
"GED-0301, a first-in-class oral antisense therapy, has the potential to
change the treatment landscape for Crohn's disease," said
The investigational oral drug GED-0301 is an antisense oligonucleotide that targets the messenger RNA (mRNA) for Smad7, thereby reducing Smad7 protein levels. In patients with Crohn's disease, abnormally high levels of Smad7 interfere with TGF-β1 anti-inflammatory pathways in the gut, leading to increased inflammation. The distinct formulation of the tablet is designed to release GED-0301 into the far end of the small intestine (the terminal ileum) and near the end of the colon (right colon), where it is thought to act locally to reduce Smad7 levels.
About Crohn's Disease
Crohn's disease is an immune-mediated, chronic inflammatory condition of the gastrointestinal tract. Crohn's disease may affect any part of the GI tract from the mouth to the anus but most commonly affects the end of the small bowel (the ileum) and the beginning of the colon. Symptoms often include abdominal pain, diarrhea, fatigue, fever, weight loss and malnutrition. The worldwide incidence rate varies between 0.1-16/100,000 persons. The exact cause of Crohn's disease is unknown and there is no cure.
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Source: Celgene International Sàrl
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