Positive step forward for retina gene therapy

As vision research continues to advance, there is a real sense that successful, long lasting gene therapy is within reach. A UK-based company called NightstaRx Ltd recently published promising results showing long-term benefits from their clinical trial of a gene therapy for choroideremia, a rare inherited retinal degenerative disease.

This Phase 1 trial was carried out at the University of Oxford where it was run by Professor Robert MacLaren, who spoke at the Fighting Blindness Retina Conference in 2014. In total six participants took part in the trial. The researchers have now reported that five of these patients had either maintained or improved vision in their treated eye three and a half years after the therapy. Two of the people involved were able to read three additional lines on an eye chart. Prof MacLaren has said that “this shows that the visual acuity gains were real and also long lasting”.

These results move this therapy a step closer to reaching people with choroideremia. However, although the trial highlighted the potential benefits and safety of gene therapy, the number of patients included was very small. NightstaRx are planning to carry out a Phase 2 trial called ‘REGENERATE’ in the near future, which would involve a larger number of participants.

Fighting Blindness is very pleased to see such positive results from a gene therapy trial. This is promising not only for the choroideremia therapy that is being tested but also for other gene therapy trials which are currently being carried out for other inherited retinal degenerative conditions.