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. Source
No articles found.
Allenaâs lead product candidate, reloxaliase, is a first-in-class, oral enzyme t...
Allenaâs lead product candidate, reloxaliase,...
DarioHealth Corp. (NASDAQ: DRIO) is a leading global Digital Therapeutics (DTx) co...
DarioHealth Corp. (NASDAQ: DRIO) is a leading g...
Ironwood Pharmaceuticals (Nasdaq: IRWD) is a GI-focused healthcare company dedicat...
Ironwood Pharmaceuticals (Nasdaq: IRWD) is a GI...
Pacific Biosciences' mission is to transform the way humankind acquires, processes...
Pacific Biosciences' mission is to transform th...
Headquartered in Bedford, Massachusetts, Anika Therapeutics, Inc. develops, manufa...
Headquartered in Bedford, Massachusetts, Anika ...
Fisher Wallace manufactures and markets wearable medical devices for the treatment...
Fisher Wallace manufactures and markets wearabl...
Sangamo Therapeutics, Inc. is focused on translating ground-breaking science into ...
Sangamo Therapeutics, Inc. is focused on transl...
Orchard Therapeutics is a fully integrated commercial-stage biopharmaceutical comp...
Orchard Therapeutics is a fully integrated comm...
Fibrocell is a cell and gene therapy company focused on improving the lives of peo...
Fibrocell is a cell and gene therapy company fo...
Intra-Cellular Therapies is a biopharmaceutical company developing novel drugs, le...
Intra-Cellular Therapies is a biopharmaceutical...
Join the National Investor Network and get the latest information with your interests in mind.