Targeted Genetics initiates phase I trial for childhood blindness

University College London’s Institute of Ophthalmology and Moorfields Eye Hospital, with funding from the UK Department of Health, are trialing the treatment in adults and children with a progressive deterioration in vision caused by an abnormality in the RPE65 gene.

In affected individuals, this defect prevents normal function of the retina, the light-sensitive layer of cells at the back of the eye. The therapy involves an adeno-associated virus vector manufactured by Targeted Genetics to deliver a normal copy of the RPE65 gene into the cells of the retina to help them to function normally.

Professor Robin Ali, who is leading the investigation, said: “We have been developing gene-based treatments for eye disease for almost 15 years but until now we have been evaluating the technology only in the laboratory.

“Testing it for the first time in patients is very important and exciting and represents a significant step towards establishing gene therapy for the treatment of many different eye conditions.”