AAV-GAD

AAV-GAD is an investigational gene therapy for Parkinson's disease. It uses an adeno-associated virus (AAV) vector to deliver the glutamic acid decarboxylase (GAD) gene directly into the subthalamic nucleus of the brain. The expressed GAD enzyme increases production of gamma-aminobutyric acid (GABA), an inhibitory neurotransmitter that helps normalize overactive neuronal circuits implicated in Parkinson’s motor symptoms such as tremor, rigidity, and difficulty walking. This approach aims to restore balance in basal ganglia circuitry by boosting local GABA levels, similar in effect to subthalamic deep brain stimulation but via a molecular mechanism. The therapy has shown significant improvements in motor function and quality of life scores in clinical trials[1][2][3][5]. Originally developed by Neurologix and now led by MeiraGTx—with manufacturing collaboration from Hologen—the program is Phase 3 ready with ongoing commercial-scale manufacturing[6][8].

Other names

AAV2-GADAAV-2-GADAAV 2-GAD