Prevail Therapeutics is applying precision medicine to the development of gene therapies to slow or stop disease progression in patients with Parkinson’s 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 programs include PR001 and PR006. PR001 is a potentially disease-modifying, single-dose AAV9 gene therapy delivering the GBA1 gene. PR001 is currently in Phase 1/2 clinical trials for patients with Parkinson's disease with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease (nGD). PR006 is a potentially disease-modifying, single-dose AAV9 gene therapy delivering the GRN gene. PR006 is currently in a Phase 1/2 clinical trial for patients with frontotemporal dementia with GRN mutations (FTD-GRN). We are also conducting preclinical studies of PR004 for the treatment of synucleinopathies and developing a broad pipeline of potentially disease-modifying AAV gene therapies for the potential treatment of a range of neurodegenerative diseases with high unmet medical need.
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