Parkinson’s Therapy Gets Regenerative Medicine Status

FDA has granted MeiraGTx Holdings a regenerative medicine advanced therapy (RMAT) designation for AAV-GAD and its use for treating Parkinson’s disease not adequately controlled with anti-Parkinsonian medications. The investigational one-time infused gene therapy is designed to reprogram dysfunctional brain circuits through the local production of GABA, a chemical neurotransmitter that can help restore normal activity to critical cells.

The RMAT was awarded following a data presentation to FDA involving three clinical studies “demonstrating the benefit of AAV-GAD when administered in a one-time stereotactic infusion to the subthalamic nucleus in the brain,” the company says. “We have demonstrated disease-modifying changes in the circuitry of the brain of patients treated with AAV-GAD as well as potentially protective changes in the substantia nigra and regions of the brain involved in cognition and mood.”