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# Parkinson’s Therapy Gets Regenerative Medicine Status
- URL: https://www.fdaweb.com/parkinsons-therapy-gets-regenerative-medicine-status/
- Published: 2025-05-09T12:00:00.000Z
- Updated: 2026-09-14T15:11:29.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5159152

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.”