PR006
GRN-associated Frontotemporal Dementia
Phase 1/2Active
Key Facts
Indication
GRN-associated Frontotemporal Dementia
Phase
Phase 1/2
Status
Active
Company
About Prevail Therapeutics
Prevail Therapeutics is a clinical-stage gene therapy company targeting neurodegenerative and rare diseases, most notably Parkinson's disease associated with GBA1 mutations and Gaucher disease. Its approach utilizes adeno-associated virus (AAV) vectors to deliver functional genes directly to the central nervous system, aiming to restore critical enzyme function. The company was acquired by Eli Lilly and Company in 2021, providing it with significant resources and strategic backing. Its lead program, PR001, is in clinical development for both GBA1-Parkinson's disease and neuronopathic Gaucher disease.
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