AAV-PHP.B-GBA1

AAV-PHP.B-GBA1 is an adeno-associated virus (AAV) gene therapy designed to treat neurodegenerative synucleinopathies, such as Parkinson's disease. It utilizes the PHP.B capsid, an engineered variant of AAV9 characterized by its exceptional ability to cross the blood-brain barrier (BBB) and achieve widespread transduction of the central and peripheral nervous systems following non-invasive intravenous administration. The vector delivers the *GBA1* gene, which encodes the lysosomal enzyme glucocerebrosidase (GCase). By restoring GCase activity, the therapy facilitates the clearance of pathological α-synuclein aggregates, thereby reducing neuroinflammation and preventing neuronal loss. Preclinical studies in mouse models of synucleinopathy have demonstrated that this approach can restore physiological enzyme levels and lead to significant behavioral recovery.