Researchers win €1m prize for gene therapy for eye disease

Two UCL Institute of Ophthalmology researchers, Professors Robin Ali and James Bainbridge, are among six international winners of the largest prize in vision research, the 2018 António Champalimaud Vision Award, worth €1 million. The winners are being recognised for developing a successful gene therapy treatment for a genetic retinal disorder, a form of Leber Congenital Amaurosis, which causes childhood blindness. This groundbreaking research demonstrated the potential for future developments in gene therapy to cure other inherited diseases. Building on a previous finding that a RPE65 gene mutation renders children functionally blind from birth, the research teams engineered a functional replacement of RPE65 using gene augmentation therapy in the eye, restoring vision to children and adults. It works by injecting a deactivated virus into the eye that contains a working copy of the RPE65 gene. The award is given out annually by the Champalimaud Foundation, based in Portugal. The researchers are now working to optimise the technique and to develop new gene therapy treatments for other inherited retinal conditions.