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Rec2-FGF21
- Development stage
- Preclinical
- Lead developer
- Kriya Therapeutics
- Modality
- Gene Addition/Replacement → Gene Therapies, Gene Editing → Gene Therapies, Gene Silencing → Gene Therapies
- Administration
- Intraperitoneal
01
Overview
Rec2-FGF21 is a recombinant adeno-associated virus (rAAV) gene therapy vector designed for the targeted delivery of the fibroblast growth factor 21 (FGF21) gene to visceral adipose tissue. It utilizes the engineered Rec2 serotype, which exhibits superior transduction efficiency in adipose tissue compared to standard serotypes. The vector employs a dual-cassette system: one cassette drives the constitutive expression of the FGF21 transgene, while the second expresses a liver-specific microRNA to minimize off-target effects in the liver. By increasing systemic levels of FGF21 through adipose tissue secretion, the therapy activates the FGF21 receptor complex (comprising FGFR1 and β-klotho), leading to improved insulin sensitivity, reduced hepatic steatosis, and enhanced metabolic health. It has been primarily evaluated in preclinical research using insulin-resistant BTBR mouse models.
- Other names
- visceral adipose tissue-directed FGF21 gene therapy
02
Targets
FGFR1 (Fibroblast growth factor receptor 1)
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