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AAV8-IRA
- Development stage
- Preclinical
- Lead developer
- Complutense University of Madrid
- Modality
- Gene Therapies
- Administration
- Intravenous
01
Overview
AAV8-IRA is a preclinical gene therapy candidate consisting of a recombinant adeno-associated virus serotype 8 (rAAV8) vector encoding the human insulin receptor isoform A (IRA) transgene under the control of a hepatospecific promoter, such as alpha-1 antitrypsin (AAT). Developed by researchers at the Complutense University of Madrid and the University of Navarra, it is designed for the treatment of type 2 diabetes mellitus and insulin resistance. By selectively overexpressing the insulin receptor isoform A in hepatocytes, the therapy aims to promote hepatic glucose uptake and glycogen storage, thereby ameliorating hyperglycemia and reducing the compensatory hyperinsulinemia that leads to pancreatic beta-cell failure.
- Other names
- AAV8-INSR-AAAV-8-INSR-AAAV 8-INSR-ArAAV8-IRArAAV-8-IRArAAV 8-IRA
02
Targets
INSR (Insulin receptor)
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