humanized FGF21

Humanized FGF21 (hFGF21) is an experimental gene therapy candidate being developed for the treatment of type 2 diabetes mellitus (T2DM) and obesity. The therapy utilizes an engineered adeno-associated virus (AAV) vector, specifically the HepaNeddM-AAV8 variant, to deliver a codon-optimized sequence of the humanized fibroblast growth factor 21 (FGF21) gene to the liver. FGF21 is a metabolic hormone that acts as an agonist at the FGFR1c/β-Klotho receptor complex, leading to improved glucose uptake, enhanced insulin sensitivity, and reduced hepatic lipid accumulation. This gene therapy approach aims to provide sustained therapeutic levels of FGF21, addressing the short half-life and potential immunogenicity issues associated with recombinant murine or human FGF21 proteins. Preclinical research conducted at the Indian Institute of Technology Kanpur has demonstrated that hFGF21 gene therapy can significantly reduce obesity and maintain normoglycemia in mouse models, and it is sometimes studied in combination with muscle-targeting miRNAs to mitigate potential side effects like muscle atrophy.

Other names

hFGF21hFGF-21hFGF 21codon-optimized humanized FGF21