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Oxford University And Syncona Announce The Formation Of Nightstar, A Company Developing Retinal Gene Therapies

2/3/2014 7:42:37 AM

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Oxford University and Syncona announce the formation of Nightstar, a company developing retinal gene therapies

Syncona LLP, an independent subsidiary of the Wellcome Trust, announces a £12 million investment in NightstaRx Ltd (“Nightstar”) a spin-out from the University of Oxford and its research commercialisation company Isis Innovation. Nightstar will focus on the development and commercialisation of therapies for retinal dystrophies (degenerative conditions affecting vision).

The company’s first programme is a gene therapy for an inherited form of progressive blindness called choroideremia developed by Professor Robert MacLaren at Oxford’s Nuffield Laboratory of Ophthalmology. The therapy has shown extremely promising results in a clinical trial published in the Lancet* in mid-January.

The gene therapy uses a small modified virus, AAV.REP1 to deliver the correct version of the choroideremia (CHM) gene to cells in the retina of the eye. The Lancet reported that six months after treatment with this therapy, the first six patients showed improvement in their vision in dim light and two of the six were able to read more lines on the eye chart.

Choroideremia is an inherited X-linked cause of blindness. It is caused by mutations to the CHM gene which encodes Rab-escort protein 1 (REP1) and affects approximately 1 in 50,000 people. 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.

The vector used to treat choroideremia, AAV.REP1, was developed by a team of researchers, led by Professor Robert MacLaren of the University of Oxford, and is currently being studied in a 12 patient Phase I clinical trial supported by the Wellcome Trust and Department of Health.

Nightstar has an exclusive licence from Isis Innovation to the intellectual property which underpins this program.

Chris Hollowood, Partner at Syncona and Nightstar’s Chairman, commented: “The recent data publication in the Lancet is very compelling and we are delighted to be working with Professor MacLaren to provide the support required to bring this important therapy to choroideremia patients. We have appointed Dr. Melanie Lee as CEO of Nightstar; an experienced industry professional with both scientific and business acumen and we will augment the team over the coming weeks.”

Tom Hockaday, Managing Director of Isis Innovation, said: “The £12 million investment in Nightstar represents one of the largest investments in a new academic spin-out in Europe. Isis Innovation is very excited to have worked with Professor MacLaren since 2009 to protect this technology and we look forward to it benefitting patients.”

Dr Melanie Lee, CEO of Nightstar, commented: “Nightstar provides a great opportunity to move AAV.REP1 along the regulatory path to product approval for choroideremia patients and the exceptional data from the work undertaken by Professor MacLaren and his team is a strong foundation upon which to realise the commercial potential of the therapy.”

Professor Robert MacLaren, of the Nuffield Laboratory of Ophthalmology at the University of Oxford, said: “The initial clinical results for choroideremia gene therapy are very promising and they give us an indication of what this technology can achieve in the future. The Wellcome Trust and the University of Oxford are two of the worlds’ leading biomedical research organisations and they have worked closely together to support the programme. The involvement of Syncona through Nightstar will assist the clinical development, including the manufacture of AAV.REP1 to the stringent requirements needed for regulatory approval, which will expedite patients’ access to the therapy.”

*The Lancet –

Further information:


Chris Hollowood
+44 (0) 20 7611 2016

Melanie Lee
+44 (0) 20 7611 2016

JW Communications

Julia Wilson
+44 (0) 781 8430877

University of Oxford press release “Gene therapy trial shows promise for type of blindness”

About Nightstar Ltd

Nightstar is a biopharmaceutical company focused on the development and commercialisation of therapies for retinal dystrophies. Its lead programme is a retinal gene therapy for choroideremia, a rare inherited cause of blindness that affects around 1 in 50,000 people. Nightstar’s shareholders include Syncona LLP, University of Oxford, Wellcome Trust and Imperial Innovations PLC – the technology transfer company of Imperial College.

About Choroideremia

Choroideremia is an inherited disorder that leads to progressive loss of vision due to degeneration of the choroid and retina which is caused by a lack of Rab Escort Protein-1 (REP-1) and occurs almost exclusively in males. The first symptoms occur in childhood, with night blindness being the most common first symptom. As the disease progresses, there is loss of peripheral vision or ‘tunnel vision’, and later a loss of central vision. Progression of the disease continues throughout the individual’s life, although both the rate and the degree of visual loss can vary, even within the same family. There is currently no treatment or cure for this disease.

For patients interested in participating in choroideremia trials please follow this link:

About Syncona

Syncona LLP was founded in 2012 and operates as an evergreen investment company, taking an active role in identifying, supporting and developing technologies with the potential to significantly impact the healthcare market of the future. The Company is an investor that can take the long view when necessary, able to concentrate investment into opportunities as technology is validated and is an independent subsidiary of the Wellcome Trust who invested the initial £200m capitalisation. Syncona provides financial resources to individuals and companies to advance the Wellcome Trust’s vision of achieving extraordinary improvements in human and animal health by supporting the brightest minds in biomedical research and the medical humanities. For further information, please visit Syncona’s website at:

About Isis Innovation

Isis Innovation is the research and technology commercialisation company of the University of Oxford. The Company provides access to technology from Oxford researchers through intellectual property licensing, spin-out company formation and material sales, and to academic expertise through Oxford University Consulting.

Isis is the highest university patent filer in the UK and is ranked 1st in the UK for university spin-outs, having created more than 100 new companies in 25 years. In the last financial year Isis has completed 395 licenses and consulting agreements with clients in 21 countries. The Isis Enterprise innovation management consultancy works with university, government and industrial clients from offices around the world.

For updates on innovations from Oxford, follow Isis on LinkedIn and Twitter or subscribe at

About Imperial Innovations

Since founding in 1986, Imperial Innovations has invested in the most promising technologies from our brightest scientists. Today it is connected with four of the world’s top research universities: Imperial College London, Cambridge and Oxford Universities and University College London. These centres of scientific excellence between them have a research income of over £1.3 billion per annum. Imperial Innovations supports scientist-entrepreneurs in the commercialisation of their ideas. It adds value by leading the formation of new spin-out companies, providing investment, encouraging co-investment as well as through its operational expertise. It also runs an Incubator in London that is the initial home for many of its technology spin-outs.

Originally formed as the Technology Transfer office for Imperial College – a role it still carries out – Imperial Innovations is today a significant technology investor and has built a portfolio of around 90 companies. Almost £150m has been invested by Innovations and £0.5bn has been raised in total by the portfolio companies. In 2013, a £30 million loan facility was agreed with the European Investment Bank (EIB) for investment in biotech and therapeutics businesses.

Imperial Innovations invests in the most promising opportunities from whichever technology sector they arise and has built particular expertise in the key sectors of: Therapeutics, Medtech and devices, Engineering and materials and ICT. For further information please visit Imperial Innovations website at:

Grant funded trials

The phase 1 clinical trial is funded by the Healthcare Innovation Challenge Fund, a parallel funding partnership between the Wellcome Trust and the Department of Health to stimulate the creation of innovative healthcare products, technologies and interventions and to facilitate their development for the benefit of patients in the NHS and beyond. Plans for an independent, grant-funded Phase II study are already underway. Nightstar will work together with these grant-funded studies to provide the shortest path to regulatory approval, producing Phase III GMP material, offering clinical and regulatory advice and funding any required pivotal clinical trials.

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