November 19, 2020 -- The U.S. Patent and Trademark Office (USPTO) has issued a composition of matter patent (No. 10,837,028) to Prevail Therapeutics for its adeno-associated viral vector (AAV) used in PR001, an experimental gene therapy program for the treatment of Parkinson's disease with mutations in the BGA1 gene and neuropathic Gaucher disease.
PR001 utilizes AAV9 to deliver a healthy copy of the GBA1 gene to the brain. The company is currently dosing patients in a phase I/II clinical trial of PR001 and expects to provide biomarker and safety analysis on a subset of patients by mid-2021. They also expect to initiate enrollment in a phase I/II trial of PR001 for type 2 Gaucher disease in the fourth quarter of 2020 and anticipate an update on the trial in 2021.
PR001 has previously been granted fast track designation for both indications and rare pediatric disease and orphan drug designation for Gaucher disease by the U.S. Food and Drug Administration.