Patients gain or maintain vision following gene therapy for choroideremia

Success from an initial UK trial of 14 patients has prompted expansion to a larger international trial involving 100 patients in the EU and North America


Promising results have been reported from a pioneering gene therapy for choroideremia.

A total of 14 patients received a single injection in the back of the eye with a virus containing the gene they were missing.

The 12 patients who underwent the procedure without complications either gained or maintained vision in the treated eye over a five year period.

During the same time, only 25% of the eyes were left untreated as controls maintained vision.

The gene therapy was generally well tolerated with no significant safety concerns.

Ophthalmologist, Professor Robert MacLaren, led the trial which began at Oxford Eye Hospital in 2011.

He said the trial had made a “big difference” to the lives of patients.

“The early results of vision improvement we saw have been sustained for as long as we have been following up these patients and in several the gene therapy injection was over five years ago,” Professor MacLaren highlighted.

The success of the trial led to a larger international study involving 100 patients in Europe and North America.

The treatment is being developed by Nightstar Therapeutics, a company established by University of Oxford and life sciences investor, Syncona.

If the international trial is successful, it could lead to the therapy being approved by global regulatory bodies.

The full paper detailing the results of the gene therapy trial are published in Nature Medicine