MeiraGTx Holdings has preliminary data from a small clinical trial showing their gene therapy for Parkinson’s disease, AAV-GAD, is safe and effective. The therapy aims to increase production of a neurotransmitter called GABA to alleviate motor symptoms. Results from a Phase 1 bridging study showed significant improvement in motor examination scores in patients who received the therapy compared to those who received a low dose or a sham treatment. MeiraGTx plans to discuss the results with regulators for a Phase 3 study. Other companies like Bayer and Aspen Neuroscience are also developing one-time treatments for Parkinson’s using different approaches.
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