Axo–Lenti–PD^
Gene-based treatment for Parkinson’s disease
Status: Phase II
Parkinson’s disease
Parkinson’s disease is caused by the degeneration of nerve cells in part of the brain, the substantia nigra. This leads to the loss of dopamine, which causes nerve cells to activate without normal control and leaves patients with symptoms such as tremor, muscle stiffness or slow physical movements. The cause of the nerve cell loss is unclear; however research indicates it is likely a combination of genetic and environmental factors.
Treatment
Axo–Lenti–PD1 is a second-generation gene therapy, which utilises Oxford Biomedica’s LentiVector® platform technology to deliver three genes that encode key dopamine synthesis enzymes. When injected into the brain’s striatum, Axo–Lenti–PD1 genetically modifies cells to produce dopamine, replacing that which is lost during the course of the disease. Unlike current drug treatment, which loses efficacy with long-term use, Axo–Lenti–PD1 is designed to provide patient benefit for a number of years following a single administration.