Prevail Therapeutics Inc. provided an update on its investigational program, PR001, an AAV9-based gene therapy delivering the GBA1 gene, for the neuronopathic Gaucher disease (nGD) indication. Prevail has granted a compassionate use request for the administration of PR001 to a single patient with Type 2 Gaucher disease via a compassionate use pathway, following approval by an international regulatory authority, and the patient was recently dosed. Type 2 Gaucher disease is the more severe form of nGD, which presents in infancy and involves rapidly progressing neurodegeneration leading to death in infancy or early childhood. Separately, as announced on December 26, 2019, Prevail’s Investigational New Drug (IND) application for PR001 for the treatment of nGD is now active. The company is proceeding with its Phase 1/2 clinical trial for Type 2 Gaucher disease patients and expects to initiate patient dosing during the first half of 2020. The company also plans to initiate a Phase 1/2 clinical trial for Type 3 Gaucher disease patients in the second half of 2020 under the same IND. Prevail intends for the company’s clinical studies to serve as the primary option for patients to receive access to any of Prevail’s investigational drugs. Prevail is also developing PR001 for Parkinson’s disease patients with a GBA1 mutation (PD-GBA). The Company has an active IND for PR001 for the treatment of PD-GBA and the PROPEL Phase 1/2 clinical trial for PD-GBA patients is now recruiting. Gaucher disease is a lysosomal storage disorder caused by mutations in the glucocerebrosidase gene GBA1, leading to multi-organ pathology. Patients with severe mutations in the GBA1 gene can present with neuronopathic Gaucher disease, also termed Type 2 or Type 3 Gaucher disease. Type 2 Gaucher disease presents in infancy and involves rapidly progressive neurodegeneration leading to death in infancy or early childhood. Type 3 Gaucher disease typically presents in childhood and can involve neurological manifestations such as gaze and motor abnormalities and seizures. There are no therapies approved by the FDA for the treatment of neuronopathic Gaucher disease.