Drug intelligence / Profile preview

AAV-FGF21 + SIRT1

Development stage
Preclinical
Lead developer
Indian Institute of Technology, Kanpur
Modality
Gene Therapies
Administration
Intravenous
01

Overview

AAV-FGF21 + SIRT1 is an experimental combination gene therapy developed by researchers at the Indian Institute of Technology Kanpur. It utilizes bioengineered "LiverLock" AAV vectors, which incorporate Neddylation-site modifications to enhance liver-specific delivery and expression of human fibroblast growth factor 21 (FGF21) and sirtuin 1 (SIRT1). This geroprotective approach is designed to counteract the progressive decline in organ function and the increased risk of metabolic disorders associated with aging. Preclinical studies in aged mouse models have demonstrated that a single systemic administration can significantly reduce obesity, improve glucose clearance, enhance muscle strength and endurance, and alleviate age-related complications such as hepatic steatosis and cardiac hypertrophy. The therapy works by upregulating longevity factors and improving mitochondrial biogenesis and quality control markers, including TFAM, PGC-1α, and Opa1.

Other names
AAV-geroprotective combination therapyFGF21 + SIRT1 gene therapy
02

Targets

FGFR1 (Fibroblast growth factor receptor 1)SIRT1 (NAD-dependent protein deacetylase sirtuin-1)

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