AXO-Lenti-PD, also known as OXB-102, is an investigational gene therapy for Parkinson’s disease. The therapy delivers three genes in vivo via a lentiviral vector to encode a set of critical enzymes required for dopamine synthesis in the brain and is expected to provide patient benefit for many years following a single administration. AXO-Lenti-PD is a next-generation gene therapy designed to further increase endogenous dopamine production over the first-generation product, ProSavin, by modifying the payload configuration.
Preclinical studies directly comparing AXO-Lenti-PD to ProSavin demonstrate increased AADC activity and dopamine productivity of the new vector configuration. Oxford BioMedica has successfully completed a phase I/II study for ProSavin, which met its primary endpoint. The results, which were published in The Lancet in 2014, demonstrate favorable safety and tolerability and a statistically significant improvement from baseline of motor function as measured by the UPDRS Part III score at 6 and 12 months (p=0.0001). This improvement has been observed to be sustained in patients for up to six years despite the progressively degenerative nature of Parkinson’s disease.