MeiraGTx data bring early POC for Parkinson’s gene therapy and localized gene expression strategy
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Early efficacy data for a Parkinson’s disease gene therapy from MeiraGTx bring proof of concept for the mechanism. The results also help validate the idea that gene therapy may be a useful tool for driving localized expression of therapeutic proteins — not just replacing mutated genes.
In the Phase II MGT-GAD-025 study of AAV-GAD from MeiraGTx Holdings plc (NASDAQ:MGTX), five patients treated with a higher 21 x 10^10 vector genome (vg) dose of the gene therapy achieved a mean 18-point improvement over baseline in the 132-point Unified Parkinson’s s Disease Rating Scale (UPDRS) Part 3, which measures motor functions, six months after the treatment. Patients scored above 25 at the time of enrollment, but the baseline scores were not disclosed. The improvement was statistically significant. The company said a 5-10 point improvement is clinically meaningful...