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

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

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

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

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