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AAV8-FGF21
- Development stage
- Preclinical
- Lead developer
- AstraZeneca
- Modality
- Gene Therapies
- Administration
- Intramuscular, Intravenous
01
Overview
AAV8-FGF21 is an investigational gene therapy platform consisting of an adeno-associated virus serotype 8 (AAV8) vector engineered to express fibroblast growth factor 21 (FGF21). Developed by multiple organizations including AstraZeneca, Rejuvenate Bio, and academic institutions, this gene therapy is designed to achieve sustained, liver-specific or tissue-specific overexpression of functional FGF21 following a single administration. FGF21 is a metabolic hormone that regulates glucose and lipid homeostasis, energy expenditure, and insulin sensitivity. In preclinical models, AAV8-FGF21 has demonstrated therapeutic potential in reversing type 2 diabetes, mitigating obesity, improving cardiac insulin resistance, and preventing cardiac hypertrophy, chronic kidney disease, and hepatic steatosis/fibrosis associated with metabolic dysfunction-associated steatohepatitis (MASH).
- Other names
- AAV-FGF21AAV-FGF-21AAV-FGF 21AAV8-Fgf21AAV-8-Fgf21AAV 8-Fgf21AAV8-hAAT-FGF21AAV-8-hAAT-FGF21AAV 8-hAAT-FGF21
02
Targets
FGFR-KLB complex (Fibroblast growth factor receptor 1c / beta-Klotho complex)FGFR2cFGFR3cFGFR4 (Fibroblast growth factor receptor 4)KLB (Beta-Klotho)FGFR1 (Fibroblast growth factor receptor 1)FGFR2 (Keratinocyte growth factor receptor)FGFR3 (Fibroblast growth factor receptor 3)
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