MAAVRX

Gene therapy company focused on Parkinson’s disease (PD) with the aim to deliver life-long restoration of L-DOPA in the brain. There is a high unmet need in PD because oral L-DOPA, the main treatment today, has variable absorption, resulting in fluctuating plasma and brain levels of L-DOPA producing dyskinesia and “off” periods resulting in poor control of symptoms. Maavrx’s lead candidate is a one-time injection of AAV gene therapy into the putamen that transduces cells to express the enzymes tyrosine hydroxylase and GTP cyclohydroxylase 1 resulting in more sustained brain level of L-DOPA.