MeiraGTx Holdings plc (NASDAQ:MGTX), a clinical-stage genetic medicines company, announced today that its gene therapy product AAV-GAD has been granted Regenerative Medicine Advanced Therapy (RMAT) designation by the U.S. Food and Drug Administration (FDA) for the treatment of Parkinson’s disease.
The RMAT designation, which expedites the development and review of promising therapies for serious conditions, was based on positive outcomes from three clinical studies. These studies showed AAV-GAD’s potential benefits when administered as a one-time treatment for Parkinson’s disease patients whose symptoms are not adequately controlled with current medications.
Dr. Alexandria Forbes, President and CEO of MeiraGTx, expressed excitement about the designation, emphasizing the clinically meaningful benefits observed in motor function and other Parkinson’s symptoms. Dr. Forbes also highlighted the significant findings from their partnership with Hologen, which applied AI technology to the Phase 2 study data, revealing potential disease-modifying changes in patients’ brain circuitry.
AAV-GAD is designed to target dysfunctional brain circuits by locally producing GABA, a neurotransmitter, through a minimally invasive procedure. The therapy aims to restore normal activity in the subthalamic nucleus, a region implicated in controlling movement.
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