Gene therapy company focused on Parkinson’s disease
Gene therapy company focused on Parkinson’s disease with the aim to deliver life-long restoration of L-DOPA in the brain. Lead candidate MRX001 is a one-time AAV gene therapy injected into the putamen that transduces cells to express the enzymes tyrosine hydroxylase and GTP cyclohydroxylase 1, resulting in sustained brain level of L-DOPA