Sunday, 31 May 2020

Oxford Univ spin off gets $20 mn for retinal gene therapy

04 February 2014 | News | By BioSpectrum Bureau

Good news for eye patients - Oxford University and Wellcome Trust-subsidiary Syncona form Nightstar

Good news for eye patients - Oxford University and Wellcome Trust-subsidiary Syncona form Nightstar

Singapore: Syncona, an independent subsidiary of the Wellcome Trust, has made a $19.60 million (£12 million) investment in NightstaRx, which is a spin-out from the University of Oxford and its research commercialization company Isis Innovation. Nightstar will focus on the development and commercialization of therapies for retinal dystrophies, which are 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 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 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.

Mr Chris Hollowood, partner, Syncona, and Nightstar's chairman, commented that, "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."

Mr Tom Hockaday, MD, Isis Innovation, said that, "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, 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."


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