Prevail Therapeutics Provides Program Update on PR001 in Parkinson’s Disease with GBA1 Mutations and Neuronopathic Gaucher Disease
Prevail Therapeutics Inc. (PRVL)
Last prevail therapeutics inc. earnings: 11/12 09:30 am
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Source: GlobeNewswire
NEW YORK, Sept. 10, 2019 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ: PRVL), a biotechnology company developing AAV-based gene therapies for patients with neurodegenerative disorders, today provided an update on the Company’s clinical development of PR001, a potentially disease-modifying, single-dose gene therapy in development for Parkinson’s disease with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease (nGD). PR001 for Treatment of PD-GBA Prevail has an open Investigational New Drug Application (IND) for PR001 for the treatment of PD-GBA, the first indication the Company is pursuing. The Company is in the process of activating clinical sites for its Phase 1/2 clinical trial in PD-GBA and is on track to initiate patient dosing this year. Approximately 7%-10% of Parkinson’s disease patients harbor an underlying mutation in the GBA1 gene, and it is estimated that there are 90,000 PD-GBA patients in the U.S. alone. No therapies currently available have shown the a
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