Drug intelligence / Profile preview

MRG-229

Development stage
Discontinued
Lead developer
Viridian Therapeutics
Modality
Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intravenous, Subcutaneous
01

Overview

MRG-229 is a **second-generation synthetic oligonucleotide** designed as a mimic of microRNA-29 (miR-29), a microRNA that is found at abnormally low levels in multiple pathological fibrotic conditions[1][5][7][10][11]. It is chemically modified for increased stability and is conjugated to a PDGFbetaR-specific bicyclic peptide (BiPPB) for targeted cellular internalization[3][4][5]. MRG-229 acts as a **microRNA modulator**, aiming to restore miR-29 levels to suppress fibrosis-associated gene expression, thereby reducing collagen and extracellular matrix protein deposition, regulated upstream through the **transforming growth factor beta (TGF-β) pathway** and other profibrotic mechanisms[1][5][9]. Preclinical studies have demonstrated robust antifibrotic activity, favorable safety, and a lack of clinically significant toxicity in animal models[2][5][12]. The molecule was developed primarily for the treatment of **idiopathic pulmonary fibrosis (IPF)**, but development for this indication was discontinued in preclinical stage as of July 2024[1].

Other names
miR-29 mimicmiR29 mimicmiR 29 mimicsecond-generation miR-29 mimic
02

Targets

COL1A1 (Collagen type I alpha 1 chain)PDGFRB (Platelet-derived growth factor receptor beta)ACTA2 (Alpha-smooth muscle actin)

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