AXO-Lenti-PD is an investigational gene therapy for Parkinson’s disease that delivers three genes encoding a critical set of enzymes required for dopamine synthesis in the brain. AXO-Lenti-PD delivers the following genes:
- Tyrosine hydroxylase (TH): converts tyrosine to L-dopa
- Cyclohydrolase 1 (CH1): rate-limiting enzyme for synthesis of essential cofactor in TH activity
- Aromatic L-amino acid decarboxylase (AADC): converts L-dopa to dopamine
AXO-Lenti-PD is a next-generation gene therapy with a modified payload configuration of the predecessor product, ProSavin®, to further improve endogenous dopamine production and provide patient benefit for multiple years following a single administration. ProSavin successfully completed a Phase 1/2 study 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 of motor function as measured by the UPDRS Part III score at 6 and 12 months. This improvement was sustained in most patients for up to four years despite the progressively degenerative nature of Parkinson’s disease.