Mongersen (GED-0301) is a specific and orally active SMAD7 antisense oligonucleotide. Mongersen restores TGF-β1 activity leading to inhibition of inflammatory signals. Mongersen can attenuate Crohn's disease-like experimental colitis in mice[1][2].
Mongersen (125 μg/mouse; p.o. twice a week for 3 weeks) reduces TNBS- or DSS-induced colonic inflammation and colonic fibrosis of mice[2].
[1]. Monteleone G, et, al. Mongersen, an oral SMAD7 antisense oligonucleotide, and Crohn's disease. N Engl J Med. 2015 Mar 19;372(12):1104-13.
[2]. Izzo R, et, al. Knockdown of Smad7 With a Specific Antisense Oligonucleotide Attenuates Colitis and Colitis-Driven Colonic Fibrosis in Mice. Inflamm Bowel Dis. 2018 May 18;24(6):1213-1224.