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AAV-GAD67 gene therapy
- Development stage
- Unknown
- Lead developer
- MeiraGTx Holdings
- Modality
- Gene Therapies
- Administration
- Intraparenchymal, Intraperitoneal
01
Overview
AAV-GAD67 gene therapy is a therapeutic approach using adeno-associated virus (AAV) vectors to deliver the GAD1 gene, which encodes the 67 kDa isoform of glutamic acid decarboxylase (GAD67). GAD67 is a key enzyme that catalyzes the conversion of the excitatory neurotransmitter glutamate into the inhibitory neurotransmitter gamma-aminobutyric acid (GABA). This modality is designed to restore the balance between neuronal excitation and inhibition in neurological disorders. In Parkinson's disease, it has been developed as part of a dual-vector system (AAV-GAD or NLX-P101) to modulate the subthalamic nucleus. In epilepsy research, it is being explored as a method to enhance tonic inhibition and suppress seizures in drug-resistant patients.
- Other names
- AAV-GAD67AAV-GAD-67AAV-GAD 67AAV-GAD1 gene therapyAAV-GAD-1 gene therapyAAV-GAD 1 gene therapy
02
Targets
HSPG (Basement membrane-specific heparan sulfate proteoglycan core protein (perlecan))GAD1 (Glutamate decarboxylase 1)
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