Gene therapy for blindness

Cutting edge therapies for eye diseases help improve quality of life, extend the working lives of treated patients, and reduce treatment costs.

gene therapy operationGene therapy operation at Oxford Eye Hospital.
Inherited retinal degenerations are the most common cause of untreatable blindness in young people and are well suited to gene therapy. The slow rate of degeneration means that there is time in which to replace the defective genes before cells die. In addition, the eye has only a small amount of tissue, and is compartmentalised anatomically. Its function is easy to test, and the fellow untreated eye allows for comparison.

Professor Robert MacLaren heads up a gene therapy research programme which is leading to new treatments for hereditary eye diseases. These treatments will not only improve the quality of life of affected individuals, but will also be of economic benefit to the NHS (through reduced treatment costs) and to the UK economy (extending the working lives of patients).

In 2014, Nightstar Therapeutics was spun out of the MacLaren gene therapy research programme, supported by a £12 million investment from the Wellcome Trust (through Syncona Partners).

This was followed in 2015 by a further investment of £23 million. This will allow researchers to develop the gene therapies for choroideremia, and to work on X-linked retinitis pigmentosa as well as four other gene therapy research programmes. These six molecular innovations are currently in clinical trials across 11 different countries.

Nightstar Therapeutics underwent a successful Initial Public Offering in 2017 – less than four years after its creation. It is now listed on NASDAQ with a market capitalisation in excess of $500 million and has become the largest dedicated retinal gene therapy company in the world.

Funded by: National Institute for Health Research, Wellcome Trust, Medical Research Council and Nightstar Therapeutics