Drug intelligence / Profile preview

miR-124-NP

Development stage
Preclinical
Lead developer
University of Texas MD Anderson Cancer Center
Modality
Gene Therapies, Nanoparticles → Drug Delivery Systems, RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intratumoral
01

Overview

miR-124-NP is a liposomal nanoparticle (LNP) formulation designed to deliver a plasmid DNA (pDNA) vector encoding microRNA-124 (miR-124). Developed by researchers at The University of Texas MD Anderson Cancer Center and Texas Tech University Health Sciences Center, this gene therapy aims to treat melanoma by inhibiting the Wnt/beta-catenin signaling pathway. miR-124 acts as a tumor suppressor that modulates the epithelial-mesenchymal transition (EMT) by decreasing the expression of SLUG, SNAIL, and vimentin. In preclinical murine models, intratumoral administration of miR-124-NP significantly reduced tumor burden and suppressed tumor growth by up to 50% without observed toxicity.

Other names
miR-124-loaded LNPsmiR124-loaded LNPsmiR 124-loaded LNPsliposomal nanoparticle-mediated miR-124 plasmid

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