Prevail Therapeutics (NASDAQ:PRVL) received FDA fast track designation for PR001, a gene therapy candidate for the treatment of Parkinson’s disease (PD).
PR001 is a single-dose gene therapy for PD patients with a mutation in the glucocerebrocidase-1 gene, which is believed to play a role in the disease. The mutation is observed in some 7% to 10% of all PD cases.
“With no treatments available that modify the progressive course or the underlying disease process of PD, a potential disease-modifying therapy like PR001 could significantly transform the lives of patients with this disease,” Dr. Asa Abeliovich, Prevail’s founder and CEO, said in a statement.
The FDA accepted Prevail’s IND application for PR001 in June 2019. The Company plans to begin dosing patients in a Phase 1/2 clinical trial in the second half of 2019.
