Prevail Therapeutics is applying precision medicine to the development of gene therapies to slow or stop disease progression in patients with Parkinsonâs disease and other neurodegenerative disorders caused by lysosomal dysfunction. Leveraging recent breakthroughs in human genetics and transformative successes of AAV-based gene therapy, our goal is to create disease-modifying treatments that dramatically improve the treatment of neurodegenerative diseases in genetically-defined patient populations.
We are developing a broad pipeline of gene therapies for neurodegenerative diseases with high unmet medical need. Our lead program is PR001 for the treatment of Parkinsonâs disease with GBA1 mutation, or PD-GBA, and neuronopathic Gaucher disease. Our pipeline also includes programs aimed at the treatment of frontotemporal dementia with GRN mutation and synucleinopathies.
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