Drug intelligence / Profile preview

miR-124

Development stage
Phase 2
Lead developer
Viridian Therapeutics
Modality
RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intratumoral, Intravenous, Intrathecal
01

Overview

miR-124 is a highly conserved, brain-enriched microRNA that serves as a critical regulator of neuronal differentiation and a potent tumor suppressor. In the central nervous system, it maintains the quiescent state of neural stem cells and promotes their maturation into neurons. In oncology, particularly in glioblastoma multiforme (GBM), miR-124 is frequently downregulated, which contributes to increased tumor stemness, invasiveness, and resistance to therapy. As a therapeutic agent, miR-124 mimics or viral vectors are used to restore its expression, thereby post-transcriptionally repressing a network of oncogenic targets including **Lhx-2**, **CDK6**, and **STAT3**. This restoration inhibits the maintenance of glioma stem cells (GSCs) and suppresses epithelial-mesenchymal transition (EMT). Beyond oncology, miR-124 is investigated for its anti-inflammatory properties, as it modulates the cholinergic anti-inflammatory pathway and is a key mediator in the mechanism of action for drugs like obefazimod in inflammatory bowel disease.

Other names
microRNA-124microRNA124microRNA 124miR-124-3pmiR124-3pmiR 124-3p
02

Targets

LHX2CDK6 (Cyclin-dependent kinase 6)

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