Phase II trial is underway investigating a gene therapy for Parkinson's

Axovant Gene Therapies Ltd., a company developing innovative gene therapies, has announced they are entering the next stage of a phase II gene therapy trial for Parkinson's.

The continuation of this trial of the AXO-lenti gene therapy follows positive intermediate results for the safety of the therapy and possible improvements in symptoms.

The trial is happening at a number of sites in the UK and will continue to assess if AXO-lenti is safe and can improve measures of Parkinson's symptoms in 30 people with the condition.


Scientists have found a way to re-programme viruses so they can be used to get important genetic instructions into the cells of our bodies.

The idea is to use these viruses to provide the genetic instructions cells need to change their fate  -  this could be by replacing a faulty gene with a functional one or providing the instructions the cell needs to make certain protective factors.


AXO–LENTI–PD, formally called OXB-102, works by introducing genes to improve dopamine production. It has been adapted from the ProSavin gene therapy so it can deliver 3 genes within a single carrier virus. These genes aim to increase dopamine production in the brain to improve symptoms of Parkinson's.

This therapy is injected directly into the brain and in this trial the researchers are investigating if one injection is enough to show long term benefits in people with Parkinson's.

Dr Katherine Fletcher, Research Communications Officer, comments:

"Research into gene therapies for Parkinson's is gaining momentum as these treatments have the potential to improve symptoms, and even slow or reverse Parkinson's. It is encouraging to see UK scientists playing a key role in this pioneering research.

"The hope with the AXO-lenti therapy is that is may have longer lasting effects than current Parkinson's medications.

"We will eagerly be following the progress of this trial."