UK-US alliance explores gene therapies to avert blindness
Gyroscope Therapeutics in Stevenage and the University of Pennsylvania have joined forces to develop novel gene therapies to counter serious eye diseases that can lead to blindness.
The sponsored research alliance unites bleeding edge technology from the UK clinical-stage gene therapy company with expertise from the University of Pennsylvania and the Penn Center for Advanced Retinal and Ocular Therapeutics (CAROT).
Gyroscope has an exclusive option to the intellectual property associated with, and arising from, the research conducted under the agreement.
A team of researchers from CAROT and Gyroscope will work together to explore specific gene therapy targets for glaucoma, optic neuritis and retinitis pigmentosa.
“Too many people around the globe face a life with limited vision or complete blindness because current treatment options for many serious eye diseases are so limited,” said Khurem Farooq, CEO at Gyroscope.
“Gene therapy has the potential to be a completely new way of approaching these diseases, and we are very excited to work with a team of world leaders in ophthalmic gene therapy research at the University of Pennsylvania to evaluate new targets for these conditions.”
Glaucoma is a leading cause of irreversible blindness globally. An estimated 80 million people have glaucoma worldwide and this number is expected to increase to more than 111 million by 2040. There is no cure.
If it is caught early, people with glaucoma can be treated with surgery or medication to help control the disease. Because glaucoma typically does not cause pain, it often progresses silently and is not diagnosed until the optic nerve is irreparably damaged.
Retinitis pigmentosa refers to a group of rare genetic retinal diseases that cause progressive loss of night and peripheral vision. The condition is often diagnosed in childhood or adolescence and can lead to legal, and sometimes complete, blindness.
An estimated 300,000 people worldwide have RP, mainly caused by a genetic variant inherited from one or both parents.
Optic neuritis occurs when the optic nerve is damaged as a result of inflammation. Symptoms of optic neuritis include temporary vision loss in one eye and pain with eye movement.
Optic neuritis is closely associated with multiple sclerosis (MS): It is the first sign of MS in 20 per cent of patients and occurs during the course of the disease in 50 per cent of victims.