Gene therapy for eye disease
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NHS To Offer New Gene Therapy Treatment For Rare Eye Disease

The gene therapy (Luxturna) to treat inherited retinal dystrophies in patients

Now for patients with a rare inherited eye disorder are set to be able to access a new gene therapy on the National Health Service that slows down the sight loss.Draft NICE guidance recommends gene therapy (Luxturna) to treat inherited retinal dystrophies in patients.

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The National Institute for Health and Care Excellence estimates just under Ninety people in England will be eligible for this treatment. Drug company Novartis agreed on a discount for the National Health Service on the £613,410 price.

Without the deal, the new gene therapy, voretigene neparvovec, would have breached the regulator’s budget impact test.

The inherited retinal dystrophies cause the gradual degeneration of the retina light-sensitive cells which results in sight loss in patients. They are usually diagnosed in childhood.

This condition causes a loss of vision which ultimately leads to almost total blindness. At the moment, there is no treatment available for this condition.

Patients with a mutation of the RPE65 gene will be suitable for the new gene therapy for this eye disease treatment. The RPE65 gene should provide the instructions to make a protein which is the key protein to normal vision.

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In the gene therapy treatment, a healthy copy of the gene is injected directly into the eye so that a working protein can be produced. But, patients have to have some functioning retinal cells for this to work.

The research study has shown that, in the short term, voretigene neparvovec improves vision as well as prevents the condition from getting even worse.

NICE said there was no long-term clinical evidence but it was biologically plausible; the gene therapy’s effect would continue for decades.

Draft recommendations on this treatment are now out for consideration by the company, healthcare professionals and even patient groups. If there are no appeals against this, NICE expects to publish its final guidance next month. The gene therapy for eye disease would then become available from January 2020.

Simon Stevens, chief executive of NHS England, said that the NHS is once again at the forefront of the genomic revolution with the patients in England among the first to benefit from this gene therapy for rare eye disease which is a revolutionary new form of treatment.

Sue Sharp, the deputy chief executive at the Royal Society for Blind Children, said that they see first hand the devastating effect of childhood sight loss in patients, and so they welcome news of this breakthrough gene therapy for eye disease and its impact on the lives of children with inherited retinal dystrophies disorder.

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Dr. Mariya Moosajee, the consultant ophthalmologist at Moorfields Eye Hospital, said that this is a piece of exciting news that will give hope to patients where previously there was none.

She added that the diagnosing and treating genetic eye conditions are extremely complex and challenging but now we are in a position to tell patients who have a gene therapy for eye disease or say, a genetic change in the RPE65 gene and the treatment is available on the NHS that may help to slow down their sight loss.


Ria Roy completed her Post Grad degree at the Visvesvaraya Technological University. She has a great grounding in the skills, including technical, analytical and research skills. She is a motivated life science professional with experience of working in famous research institutes