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Drug intelligence / Profile preview
AAV.EpoR76E
- Development stage
- Preclinical
- Lead developer
- University of Iowa
- Modality
- Gene Therapies, Recombinant Proteins and Enzymes
- Administration
- Intramuscular
01
Overview
**AAV.EpoR76E is a gene therapy consisting of an adeno-associated viral (AAV) vector encoding a modified form of erythropoietin, EpoR76E, in which the arginine at position 76 is replaced by glutamate. EpoR76E retains neuroprotective and neurotrophic activities but lacks erythropoietic (blood-forming) activity, reducing risk of increased hematocrit and related side effects. The therapy is delivered via intramuscular injection and yields sustained systemic expression of EpoR76E. It has been shown in preclinical models to prevent cognitive decline, neuronal loss, synaptic protein loss, and amyloid-β accumulation in Alzheimer's disease models, and also to protect dopaminergic neurons in Parkinson’s disease models and retinal ganglion cells in glaucoma models.**[1][5]
- Other names
- AAV-EpoR76EAAV-EpoR-76EAAV-EpoR 76EAAV2/5.CMV.EpoR76E
02
Targets
INSRR (Insulin receptor-related receptor)Erythropoietin Receptor
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