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mongersen
- Development stage
- Discontinued
- Lead developer
- Bristol Myers Squibb
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Oral
01
Overview
Mongersen is an investigational antisense oligonucleotide designed to target and downregulate Smad7 mRNA. By inhibiting Smad7, Mongersen restores the activity of transforming growth factor-beta 1 (TGF-β1), a cytokine with anti-inflammatory properties that plays a key role in regulating immune responses in the gut. The drug was primarily developed for the treatment of Crohn's disease, aiming to reduce chronic inflammation associated with this condition. Mongersen is administered orally and acts locally within the gastrointestinal tract[4][5][8]. Despite promising early-phase results, later phase 3 clinical trials failed to demonstrate efficacy over placebo in patients with active Crohn's disease[5][6][8].
- Other names
- mongersen sodium
02
Targets
SMAD7 (Mothers against decapentaplegic homolog 7)
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