Manchester Royal Eye Hospital administers its first gene therapy treatment

Manchester Royal Eye Hospital (MREH) achieves a historical milestone, as it delivers gene therapy at this hospital for the first time. MREH is participating in a gene therapy clinical trial, in collaboration with Saint Mary’s Hospital, for patients with a rare genetic eye condition, x-linked retinitis pigmentosa (XLRP).

XLRP, for which there is currently no effective treatment, is one of the most common causes of blindness in young people.

XLRP is an inherited condition that eventually leads to blindness in adult men, most often by the end of their fourth decade. It affects the retina’s ability to respond to light, a result of abnormalities in the photoreceptors in the back of the eye.

In more than two-thirds of cases, the disease is caused by a mutation to the retinitis pigmentosa GTPase regulator (RPGR) gene. Common early symptoms include difficulty seeing at night in young males and a progressive loss in the visual field and clarity of vision as they age.

The gene therapy approach being used aims to introduce functional copies of the faulty RPGR gene into the cells of the retina. The purpose of the study is to evaluate the safety and efficacy of the treatment of XLRP in patients with the RPGR mutation.  In Manchester, the study is being conducted through a collaboration between MREH, Saint Mary’s Hospital and the NIHR Manchester Clinical Research Facility, a specialist facility for early phase clinical research.

The study is sponsored by NightstaRx Limited (Nightstar), a biopharmaceutical company based in London. Manchester was one of the sites chosen to conduct the study due to MREH’s world-leading expertise in ophthalmology and Saint Mary’s Hospital’s expertise in genetics.

The surgeon leading the trial in Manchester is Professor Paulo Stanga at MREH, University of Manchester and Manchester Vision Regeneration (MVR) Lab at NIHR Manchester Clinical Research Facility.  Professor Stanga said:

Gene therapy is an exciting and new area that could potentially offer a cure across a number of disease areas.  We’re delighted to be able to offer our patients the opportunity to participate in this trial for this new treatment for X-linked retinitis pigmentosa.  This is a devastating condition for which there’s currently no effective treatment.

“Our ambition is to be able to offer our patients appropriate treatment across the whole spectrum of this disease.  This latest study is an opportunity for us to try and stabilise disease progression for those with an early stage of a specific type of retinitis pigmentosa, known as X-linked.”


Professor Graeme Black, Consultant in Genetics and Ophthalmology, University of Manchester/Central Manchester University Hospitals NHS Foundation Trust adds:

Manchester is a leading centre for the diagnosis and treatment of inherited genetic eye diseases.  The co-location of Manchester Royal Eye Hospital and Saint Mary’s Hospital, which is home to the Manchester Centre for Genomic Medicine means we have a cohort of patients who could potentially benefit from this new treatment.

Dave Fellows, Chief Executive Officer of Nightstar commented: “This study highlights our commitment to helping patients with the devastating consequences of inherited retinal diseases such as X-linked retinitis pigmentosa. We aim to leverage our existing relationships with ophthalmology centres of excellence, such as Manchester, to conduct multicentre clinical trials. Our goal is to be the leader in ocular gene therapy.”