Gene therapy treatment for rare inherited blindness commences in England

  • NHS England
  • 17 Feb 2020

  • curated by Pavankumar Kamat
  • UK Medical News
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The first patients in England with a rare inherited retinal disorder have been treated using a revolutionary new gene therapy to restore their vision, as part of the NHS Long Term Plan.

Individuals born with Leber’s Congenital Amaurosis (LCA) have poor eyesight which deteriorates rapidly, ultimately leading to complete loss of vision for some patients in their childhood.

The pioneering treatment known as voretigene neparvovec (Luxturna) is the first in its generation of injectable gene therapies. Clinical trials have shown favourable recovery of night-time vision with this treatment.

The first patient in the UK received the treatment at Moorfields Eye Hospital in London. The treatment will be initially available for adults at three specialist centres in Manchester, London and Oxford. Great Ormond Street Hospital in London has also commenced treatment on two children with LCA.

In October 2019, voretigene neparvovec was approved for treating inherited retinal dystrophies mediated by RPE65 gene mutations, after NHS England reached an agreement with the manufacturer Novartis to fund the drug.

Professor Stephen Powis, NHS Medical Director, said: "Once again, the NHS is at the forefront of the genomic revolution with patients in England among the first to benefit from this new form of treatment – a modern day miracle – as part of the Long Term Plan."