Prevail Therapeutics receives FDA Fast Track Designation for PR001
Prevail Therapeutics announced that the U.S. Food and Drug Administration has granted Fast Track Designation for the Company's lead gene therapy program, PR001, for the treatment of Parkinson's disease patients with a GBA1 mutation. PR001 is a potentially disease-modifying, single-dose, AAV9-based gene therapy being developed for the treatment of PD-GBA and neuronopathic Gaucher disease. Following the FDA's acceptance of Prevail's Investigational New Drug application in June, the Company is on track to begin dosing patients in a Phase 1/2 clinical trial in the second half of 2019. The trial will investigate the safety and tolerability of PR001, and will also measure key biomarkers and exploratory efficacy endpoints, in patients with PD-GBA.