Drug intelligence / Profile preview

FGF21 + miR-223

Development stage
Preclinical
Modality
Recombinant Proteins and Enzymes, RNA Therapeutics → Nucleic Acid Therapeutics, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
Administration
Intravenous
01

Overview

FGF21 + miR-223 refers to an engineered combination therapy delivered via extracellular vesicles (EVs) that display fibroblast growth factor 21 (FGF21) on their surface and encapsulate microRNA-223 (miR-223) inside. This dual-modality approach is designed for the treatment of metabolic dysfunction-associated steatohepatitis (MASH), a progressive liver disorder. The EVs efficiently target the liver, where FGF21 acts to improve hepatic steatosis and metabolic function, while miR-223 reduces inflammation and fibrosis. Preclinical studies in human liver cell lines and mouse models have shown that this combination significantly reduces lipid accumulation, inflammation, and fibrotic markers in the liver. The therapeutic effect is dependent on both components; blocking either FGF21 or miR-223 diminishes efficacy. Compared to conventional FGF21 treatment, this EV-based delivery system offers improved safety by avoiding adverse effects such as bone density loss[1][2][3].

02

Targets

FGFR1 (Fibroblast growth factor receptor 1)

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