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Drug intelligence / Profile preview
AAV8-TBG-GCH1
- Development stage
- Preclinical
- Lead developer
- NYU Grossman Long Island School of Medicine
- Modality
- Gene Therapies
- Administration
- Intravenous
01
Overview
AAV8-TBG-GCH1 is an experimental adeno-associated virus serotype 8 (AAV8) gene therapy vector designed to deliver GTP cyclohydrolase 1 (GCH1) specifically to hepatocytes under the control of the liver-specific thyroxine-binding globulin (TBG) promoter. Developed by researchers at the NYU Grossman Long Island School of Medicine, AAV8-TBG-GCH1 is being investigated for the treatment of metabolic dysfunction-associated steatohepatitis (MASH) and liver fibrosis. GCH1 is a rate-limiting enzyme in the synthesis of tetrahydrobiopterin (BH4), a potent antioxidant cofactor that prevents lipid peroxidation and ferroptosis. By restoring GCH1 expression in aged or injured hepatocytes, the gene therapy aims to mitigate ferroptosis-promoted senescence, reduce mitochondrial DNA damage, and attenuate hepatic fibrosis.
- Other names
- AAV8-GCH1AAV-8-GCH1AAV 8-GCH1
02
Targets
GCH1 (GTP cyclohydrolase 1)
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