NightstaRx closes $35 million Series B funding to advance Inherited Retinal Disease pipeline

London, 9 November 2015 – NightstaRx Ltd ("Nightstar"), a biopharmaceutical company specialising in developing gene therapies for inherited retinal dystrophies, announces a $35 million Series B financing round. The round was led by New Enterprise Associates (NEA), one of the world's leading venture capital firms. Founding investor Syncona, an independent subsidiary of the Wellcome Trust, also contributed to the round. In conjunction with the Series B, Nightstar has expanded its pipeline with 5 further programme licences from the University of Oxford through its technology commercialisation subsidiary, Isis 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.

In association with the financing, the Company has added David Mott to the Board of Nightstar. David is currently General Partner of NEA where he also chairs and sits on a number of Boards. Chris Hollowood, Partner of Syncona will continue as Chairman of Nightstar.

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 published in the Lancet* in January 2014. This approach uses a viral vector known as adeno-associated virus (AAV) to deliver a wild-type copy of the Rab-escort protein 1 (REP-1) gene (AAV2-REP1) 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."
– Ends –
Further information:
David Fellows
+44 (0) 20 7611 2048
JW Communications Julia Wilson

NEA +44 (0) 781 8430877

+1 (301) 272-2300
Kate Barrett
Isis Innovation +44 (0)1865 280867
Renate Krelle

Notes for Editors:
About Nightstar
Nightstar is a private biopharmaceutical company, spun-out from the University of Oxford in 2014, focused on the development of therapies for retinal dystrophies. The Company's lead programme is a retinal gene therapy for choroideremia, a rare inherited cause of blindness that affects around 1 in 50,000 people. Gene therapy has the potential to be an effective treatment for choroideremia and a range of other retinal dystrophies. The Company's lead investor, Syncona LLP, an independent subsidiary of the Wellcome Trust, is 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.

About New Enterprise Associates, Inc
NEA is a global venture capital firm focused on helping entrepreneurs build transformational businesses across multiple stages, sectors and geographies. With nearly $17 billion in cumulative committed capital since inception NEA invests in technology and healthcare companies at all stages in a company's lifecycle, from seed stage through IPO. The firm's long track record of successful investing includes more than 200 portfolio company IPOs and more than 320 acquisitions.
About Syncona
Syncona LLP was founded in 2012 and operates as an evergreen investment company, taking an active role in identifying, developing and funding technologies with the potential to significantly impact the healthcare market of the future. Syncona can take the long view when necessary, able to concentrate investment into opportunities as technology is validated. Syncona is a subsidiary of the Wellcome Trust. For further information please visit the Company's website at:
About University of Oxford Medical Sciences Division
Oxford University's Medical Sciences Division is one of the largest biomedical research centres in Europe, with over 2,500 people involved in research and more than 2,800 students. The University is rated the best in the world for medicine and life sciences, and it is home to the UK's top-ranked medical school. It has one of the largest clinical trial portfolios in the UK and great expertise in taking discoveries from the lab into the clinic. Partnerships with the local NHS Trusts enable patients to benefit from close links between medical research and healthcare delivery.
About Isis Innovation
Isis Innovation Ltd is the research and technology commercialisation company of the University of Oxford. We provide 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 over 110 new companies in 25 years. In the last financial year we completed 529 licenses and consulting agreements. Isis Enterprise, our innovation management consultancy, works with university, government and industrial clients from offices around the world.
About Choroideremia
Choroideremia is an inherited X-linked recessive disease which inevitably causes blindness. It is caused by mutations to the CHM gene which encodes Rab-escort protein 1 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. For patients interested in participating in choroideremia trials please follow this link: