Therapy Areas: Inflammatory Diseases
Prevail Therapeutics IND Active for Phase 1/2 Trial of its Gene Therapy PR001 to Treat Parkinson's Disease Patients with GBA1Mutations
6 June 2019 - - The US Food and Drug Administration has accepted US-based gene therapy company Prevail Therapeutics Inc.'s Investigational New Drug application for its lead gene therapy programme, PR001, the company said.
PR001 is a potentially disease-modifying, single-dose, AAV9-based gene therapy being developed for the treatment of Parkinson's disease patients with a GBA1 mutation (PD-GBA).
The active IND allows Prevail to initiate a Phase 1/2 clinical trial that will investigate the safety and tolerability of PR001, and will also measure key biomarkers and exploratory efficacy endpoints, in patients with PD-GBA. The company plans to begin dosing patients in the trial this year.
Parkinson's disease is a chronic, progressive neurodegenerative disorder that affects up to one m people in the United States and more than 7m people worldwide.
PD-GBA affects 7% to 10% of the total Parkinson's disease population worldwide and an estimated 90,000 individuals in the United States alone. 
GBA1 encodes the lysosomal enzyme, beta-glucocerebrosidase, or GCase. Mutations in the GBA1 gene lead to a deficiency of GCase, resulting in lysosomal dysfunction in CNS cells, which we believe leads to the inflammation and neurodegeneration present in PD-GBA. 
GBA1 mutations impact the risk of developing Parkinson's disease as well as many other aspects of the disease course, including the severity, age of onset and rate of progression of disease and the likelihood of dementia.
There are no treatments available that modify the progressive course or the underlying disease process of Parkinson's disease.
Prevail Therapeutics is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases.
Prevail was founded by Dr. Abeliovich in 2017, through a collaborative effort with The Silverstein Foundation for Parkinson's with GBA and OrbiMed, and is headquartered in New York, NY.