New vision: Gene therapy that hopes to restore sight to the blind to be tested on humans

© Fabrizio Bensch
A new gene manipulation technique that hopes to restore sight to the visually impaired will be tested on humans in the next three years.

Research conducted at the University of Manchester has been licensed by US clinical-stage biotechnology company Acucela, which will develop and commercialize the therapy, starting human trials in the next three years.

The therapy is for the treatment of an inherited retinal condition called Retinitis Pigmentosa(RP), which affects one in 4,000 people in the US, Europe and Asia. There is currently no effective treatment for the degenerative disease which often begins in childhood.

It uses optogenetic technology, and involves the use of a specially designed virus to give the DNA instructions for the rhodopsin light-sensing protein into neurons in the inner nuclear layer of the retina.

The technology was previously tested on mice in trials led by Paul Bishop, Professor of Ophthalmology at the University of Manchester and proved to be effective at restoring a visual response in a mouse model of retinal degeneration.

It will now be studied across a variety of genetic mutations in RP and related conditions, with the ultimate aim to assess the ability of the process in restoring some level of vision in patients who are legally blind.

Professor Bishop told Optometry Today that the partnership with Acucela was very significant. “It would be very exciting to be able to offer them {his patients} the prospect of a treatment that will improve their vision,” he said.

CEO of Acucela, Dr. Ryo Kubota, said the development is considerable for patients “who have lost much of their vision as well as their hope”.