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AAV-IDE
- Development stage
- Preclinical
- Modality
- Gene Addition/Replacement → Gene Therapies, Gene Editing → Gene Therapies, Gene Silencing → Gene Therapies
- Administration
- Parenteral
01
Overview
AAV-IDE is a gene therapy candidate utilizing an adeno-associated virus (AAV) vector to deliver and overexpress the insulin-degrading enzyme (IDE) gene. IDE is a zinc-binding metalloendopeptidase known for its role in the clearance of amyloidogenic proteins, including insulin and amyloid-beta. In preclinical models of Parkinson's disease, AAV-IDE-mediated overexpression has been shown to reduce the accumulation of pathological α-synuclein, alleviate motor deficits, and protect dopaminergic neurons from neurotoxicity. The neuroprotective mechanism is partially attributed to the suppression of the Hippo signaling pathway, specifically through the inhibition of MST1/2 kinases, which reduces neuronal apoptosis and α-synuclein-induced toxicity.
- Other names
- AAV-hIDEAAV-Insulin-Degrading Enzyme
02
Targets
IDE (Insulin-degrading enzyme)
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