Prevail Therapeutics is focused on developing innovative gene therapies aimed at addressing the underlying genetic causes of neurodegenerative diseases, particularly Parkinson's disease and frontotemporal dementia. The company employs a precision medicine approach, utilizing adeno-associated virus (AAV)-based gene therapies to target specific genetic mutations that contribute to disease progression. Their lead product candidates, PR001 and PR006, are designed to deliver healthy copies of the GBA1 and GRN genes, respectively, to restore normal cellular function and mitigate the effects of lysosomal dysfunction, which is a key factor in the pathogenesis of these disorders. By leveraging recent advancements in human genetics and gene therapy technology, Prevail aims to provide disease-modifying treatments that can significantly improve patient outcomes.

The therapeutic strategies being developed by Prevail Therapeutics include both intra-cisterna magna injections and intravenous infusions, tailored to the specific needs of the diseases being targeted. For instance, PR001 is being evaluated in clinical trials for its efficacy in treating patients with Parkinson's disease associated with GBA1 mutations and Type 1 Gaucher disease, while PR006 is aimed at patients with frontotemporal dementia linked to GRN mutations. The use of AAV9 as a delivery vector is particularly noteworthy, as it has demonstrated the ability to effectively reach the central nervous system, ensuring that the therapeutic genes can exert their intended effects. Overall, Prevail Therapeutics is at the forefront of developing gene therapies that hold the potential to alter the course of debilitating neurodegenerative diseases.

United States

Prevail Therapeutics was acquired by Eli Lilly and Company.

At Prevail Therapeutics, we are developing a pipeline of gene therapies to slow or stop the underlying disease process for patients with Parkinson’s disease and other neurodegenerative disorders. We are leveraging recent breakthroughs in human genetics that point to potential disease-modifying targets for neurodegenerative diseases. Our programs utilize a precision medicine approach targeting patient populations with urgent unmet needs, where there are currently no available therapies that modify the progressive course of their disorders.

Prevail Therapeutics

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