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Gastroenterologists are familiar with inhibition of tumor necrosis factor-alpha (TNF-α) as a means of treating Crohn disease. Another potential immune-pathway target involves the cytokine transforming growth factor (TGF) β-1. TGF-β-1 has immunosuppressive properties, and its activity in Crohn disease is decreased by elevated levels of SMAD7, a protein that binds TGF-β receptor and prevents its activity. Mongersen is a 21-base single-stranded oligonucleotide that binds to SMAD7 RNA and facilitates its degradation, thereby reducing SMAD7 levels and allowing normal TGF-β-1 signaling.
In the current industry-funded, phase II, randomized trial, investigators compared the efficacy of mongersen (10 mg, 40 mg, or 160 mg daily for 2 weeks) with place…