First patients begin gene therapy treatment for blindness on NHS

The first patients in the UK have begun treatment with a next-gen gene therapy that can restore eyesight on the NHS.

Babies born with inherited retinal disorder Leber’s Congenital Amaurosis (LCA) have poor sight which swiftly deteriorates, with many ultimately losing their vision completely in childhood.

Voretigene neparvovec (marketed as Luxturna) is the first in a new generation of gene therapies that can be directly administered to patients, in this case through an injection. Many patients in the trials recovered their night time vision with this treatment.

Jake Ternent, patient at Moorfields Eye Hospital was the first in the UK to receive the treatment. He had no night time vision and his daylight vision has been deteriorating since childhood and he is now blind.

“After participating in 15 years of research I now have the golden opportunity to be the first person in the UK to have this treatment on the NHS,” he said.

“I am hopeful that this procedure will not only improve my own quality of life but that it will be able to help others with my condition too.”

Robert MacLaren, Consultant Ophthalmologist at Oxford University Hospitals, said: “I am delighted to see that gene therapy research has finally come to fruition and we now have our first approved treatment – in this case for a rare form of genetic blindness.

“The NHS has been leading the world in gene therapy research with several successful clinical trials.

“The recent decision by NICE to provide this novel treatment for our patients shows how the UK is maintaining its global position in delivering first class healthcare.”

NHS England reached a NICE-endorsed deal for voretigene neparvove with manufacturer Novartis to fund the drug.

It’s expected that this will provide access to treatment for the first time for patients living with a retinal dystrophy, caused by a specific gene mutation. Until now no treatment has been available and it is estimated that as many as 100 patients could benefit from the new gene therapy.

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