Biotech firm to tap blindness gene therapy

2014-01-30 20:51

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London - Oxford scientists have set up a biotechnology company to develop the use of gene therapy in treating blindness, and the Wellcome Trust medical charity has invested $20m in the start-up.

NightstaRx plans to develop and sell therapies for retinal dystrophies - degenerative conditions affecting vision - by building on work carried out at the University of Oxford's Nuffield Laboratory of Ophthalmology, it said on Thursday.

Oxford-based researchers reported two weeks ago that an early-stage clinical trial using gene therapy had shown promise in a handful of patients with a progressive form of blindness called choroideremia.

The company has an exclusive licence to the intellectual property underpinning the Oxford gene therapy programme.

Choroideremia is caused by a mutation in a gene that makes a protein called REP1. It affects an estimated one in 50 000 people and causes sufferers, who are mainly men, to lose their sight gradually as the cells in the retina degenerate.

Read more on:    health  |  research

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