ProSavin

ProSavin is an experimental gene therapy developed for the treatment of Parkinson's disease. It uses a lentiviral vector (based on equine infectious anemia virus) to deliver three genes—tyrosine hydroxylase, aromatic amino acid dopa decarboxylase (AADC), and GTP-cyclohydrolase 1—directly into the striatum of the brain. These genes encode enzymes essential for dopamine biosynthesis, effectively reprogramming non-dopaminergic striatal neurons to produce and secrete dopamine locally. The goal is to provide continuous, localized dopamine replacement as an alternative to oral dopaminergic therapies, which are associated with motor fluctuations and off-target effects over time. ProSavin was developed by Oxford BioMedica and has undergone Phase I/II clinical trials demonstrating a favorable safety profile but only moderate efficacy in improving motor symptoms in patients with advanced Parkinson's disease[1][2][3][4][5][6][7]. Development of ProSavin has since been superseded by an optimized version called AXO-Lenti-PD (OXB-102)[1].

Brand names

ProSavin

Other names

Lenti-TH-AADC-CH1Lenti-TH-AADC-CH-1Lenti-TH-AADC-CH 1