MeiraGTx(US:MGTX) Globenewswire·2025-05-09 12:00Core Viewpoint - The FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to MeiraGTx's AAV-GAD for treating Parkinson's disease, indicating significant potential benefits based on clinical study data [1][2][3] Company Overview - MeiraGTx Holdings plc is a clinical-stage genetic medicines company with a focus on developing gene therapies, including AAV-GAD for Parkinson's disease [1][7] - The company has a broad pipeline with four late-stage clinical programs targeting various diseases, including inherited conditions and common diseases [7] Product Details - AAV-GAD is an investigational gene therapy designed to address the dysfunction of brain circuits in Parkinson's disease by promoting local production of GABA [6] - The therapy is administered via a one-time stereotactic infusion into the subthalamic nucleus, a critical area for movement regulation [6] Clinical Studies - The RMAT designation was awarded following positive results from three clinical studies: a Phase 1 study (n=12), a Phase 2 double-blind study (n=45), and a bridging study (n=14) [2][3] - The Phase 2 studies demonstrated significant benefits on the standard motor endpoint in Parkinson's disease, specifically in the UPDRS Part 3 [3] Regulatory Insights - RMAT designation allows for increased interaction with the FDA, facilitating expedited development and review processes for promising therapies [4] - The designation includes benefits similar to Fast Track and Breakthrough Therapy designations, such as rolling review and potential Priority Review for the biologics license application [4] Market Context - Parkinson's disease is the second most common neurodegenerative disease, affecting nearly one million people in the U.S. and over 10 million globally [5] - Many patients experience diminishing returns from traditional dopamine replacement therapies, highlighting the need for effective disease-modifying treatments [5]