PR001 Gets Fast Track Status for Parkinson Disease With GBA1 Mutation

PR1 Gets Fast Track Status for Parkinson Disease With GBA1 Mutation - Neurology Advisor


The Food and Drug Administration (FDA) has granted Fast Track designation to PR001 (Prevail Therapeutics) for the treatment of Parkinson disease patients with a GBA1 mutation (PD-GBA).
PR001 is an investigational single-dose gene therapy that utilizes an AAV9 viral vector to deliver the GBA1 gene to a patient’s cells. Mutations in the GBA1 gene can lead to a deficiency of beta-glucocerebrosidase, leading to an accumulation of glycolipids and lysosomal dysfunction in CNS cells. The Company believes that this is what leads to the inflammation and neurodegeneration observed in patients with PD-GBA.