£23 million boost for Oxford spinout company

Founding investor Syncona, an independent subsidiary of the Wellcome Trust, also contributed to the funding. Nightstar has also expanded its pipeline with 5 further programme licences from the University of Oxford through its technology commercialisation subsidiary,Oxford University Innovation. The funds will enable Nightstar to continue clinical development of its gene therapy for choroideremia and advance multiple retinal gene therapy programs into human trials.

Choroideremia is an inherited disease that inevitably causes blindness. It is caused by a mutation in the X chromosome and affects approximately 1 in 50,000 people. It is more likely to affect boys, who have only one X chromosome, as girls must have the mutation in both of their chromosomes to experience the disease. The first symptom of the condition is usually an impairment of night vision which often occurs in early childhood. This is followed by progressive narrowing of the field of vision, as well as a decrease in the ability to see details, culminating in blindness, most commonly in late adulthood. No effective treatment currently exists.

Nightstar’s lead programme is a gene therapy for an inherited form of progressive blindness, choroideremia, developed by Professor Robert MacLaren at Oxford’s Nuffield Laboratory of Ophthalmology. The therapy has shown promising results in a clinical trial, which was published in the Lancet in January 2014. This approach uses a genetically modified virus to deliver an unmutated  copy of the relevant gene into the retinal cells of choroideremia patients.

David Fellows, CEO of Nightstar said: 'We are delighted to welcome a high quality investor such as NEA who supports our goal to build Nightstar into a leading commercial retinal gene therapy enterprise. Our mission is to restore or maintain sight in patients suffering from a range of untreatable blinding diseases and the participation of both NEA and Syncona is essential to realising that mission.'

Dave Mott, Partner at NEA said: 'The Nightstar team has made ground breaking progress in gene therapy treatment for retinal dystrophies. We are very excited about the technology, the team and the product candidates at Nightstar. We look forward to working with them to advance their pipeline and ultimately bring therapies to market that make a difference in these sight-threatening disorders.'

Robert MacLaren, Professor of Ophthalmology at the University of Oxford said: 'Gene therapy has huge potential as a treatment for many patients who are suffering from retinitis pigmentosa and other genetic forms of blindness. We have established an internationally renowned team around the choroideremia programme and I am delighted that this additional funding will allow us to develop our other retinal gene therapy projects into real treatments for patients.'