Boost for UK biotech Nightstar after gene therapy treatment improves vision in blind people
A gene therapy treatment for blindness discovered by a biotech company spun out of Oxford University could be close to commercial success after results from a five-year trial showed all patients who were treated with the drug benefitted from it.
Nightstar Therapeutics, founded by UK-listed investment company Syncona and Oxford University, reported that all 14 patients in the trial at Oxford Eye Hospital either gained or maintained their vision after being injected in the back of the eye with a virus containing the missing gene responsible for the disease, known as choroideremia.
Three-quarters of the patient group that did not receive the treatment lost vision.
The trial will bolster Nightstar, which listed on the Nasdaq stock market in the US last month. Nightstar is 38pc owned by Syncona, a company that is in turn part-owned by the Wellcome Trust and Cancer Research UK. Syncona is listed on the London Stock Exchange and invests in healthcare companies and backs researchers developing promising new treatments for disease.
Choroideremia is the most common cause of untreatable blindness in young people. Nightstar has just started enrolling patients in a phase three clinical trial, which is expected to read out next year.
The results from the five-year study suggest the treatment, called NSR-REP1, has a good chance of passing the final phase three trial.
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