BBC News: Gene therapy to halt rare form of sight loss

BBC news logo

18 February 2020
Source: BBC News

A new gene therapy has been used to treat patients with a rare inherited eye disorder which causes blindness. It’s hoped the NHS treatment will halt sight loss and even improve vision. Matthew Wood, 48, one of the first patients to receive the injection, told the BBC: “I value the remaining sight I have so if I can hold on

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New gene therapy could lead the way for possible treatment for Stargardt disease

Fight for Sight logo

10 February 2020
Source: Fight for Sight

A leading Professor of Ophthalmology at the University of Oxford has shared the details of his research which could pave the way for a new treatment for sight loss condition Stargardt disease. In the latest episode of eye research charity Fight for Sight’s podcast Eye Research Matters, Professor Robert MacLaren discusses his pioneering research into using gene therapy to treat

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Retina UK publishes unique insight into the experiences of people with inherited retinal conditions

Retina UK logo

13 September 2019
Source: Retina UK

Evidence of the real-life experiences, challenges and expectations of almost 1,000 people affected by inherited sight loss conditions have been published by national sight loss charity Retina UK. The charity presented some of the key findings from its 2019 Sight Loss Survey when it was invited to represent its community during the recent NICE consultation for the gene therapy Voretigene

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Retina UK: Gene therapy for RPE65 gene approved for use in EU

23 November 2018
Source: Retina UK

The gene therapy Luxturna has won European regulatory approval for the treatment of Inherited retinal dystrophy caused by mutations in the RPE65 gene. This milestone decision was made by the European Medicines Agency (EMA) on Thursday, 22 November. Luxturna is the first and only gene therapy available in Europe to treat inherited retinal disease. It had already been approved for

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The Guardian: US drug firm offers cure for blindness – at $425,000 an eye. With comment from RP Fighting Blindness

4 January 2018

Spark Therapeutics says ‘responsible price’ for Luxturna gene therapy ensures access for patients with retinal defect. A drug whose inventors claim it can cure a rare form of blindness is to be one of the most expensive medicines ever sold at $850,000 (£630,000). Luxturna is injected directly into the eye to address the root cause of visual impairment by replacing

Read more The Guardian: US drug firm offers cure for blindness – at $425,000 an eye. With comment from RP Fighting Blindness

Gene therapy company submit an application to NICE for the world’s first ever treatment for an inherited eye disease

10 August 2017
Source: Fight for Sight

Spark Therapeutics, an American gene therapy company, has published potentially ground-breaking data from the first ever Phase 3 clinical trial for an inherited retinal disease (IRD). This is the first time any treatment for a genetic disease has reached this stage. Published in the Lancet in July, the results illustrate how a new gene therapy drug called voretigene neparvovec could

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University of Oxford: BBC: Hereditary blindness cure tested

5 April 2017

A virus carrying corrective DNA into cells at the back of the eye is being tested to treat one of the most common forms of hereditary blindness in young men and boys. It targets XLRP (X-linked retinitis pigmentosa), which is progressive and currently incurable. Professor Robert MacLaren of Oxford University is the leader of this first trial on patients in

Read more University of Oxford: BBC: Hereditary blindness cure tested