Updated on 4 February 2014
Dr Melanie Lee, CEO, Nightstar, revealed that, "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 realize the commercial potential of the therapy."
Professor Robert MacLaren, Nuffield Laboratory of Ophthalmology, University of Oxford, pointed out that, "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."