Oxford Univ Spin Off Gets $20 Mn for Retinal Gene Therapy

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Oxford Univ spin off gets $20 mn for retinal gene therapy 04 February 2014 | News | By BioSpectrum Bureau 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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Robert Maclaren
INTERVIEW IN CONVERSATION WITH Robert MacLaren, Professor of Ophthalmology, University of Oxford Professor Robert MacLaren gave the Keeler Lecture at the Royal College of Ophthalmologists Annual Meeting in May 2019 on gene therapy for retinitis pigmentosa. We caught up with him afterwards to find out more. people in the developed world, since we now well and we will have treatments available have treatments for diabetic retinopathy. for our patients in the UK, but we’re not quite There is a huge unmet need. We have also got there yet. one gene that has been approved so it is up to us to try and find other genes and get them What do you hope will be made through to clinical trials. possible through gene therapy in the future? There was a lot of media attention The ideal, of course, would be to prevent earlier this year about gene therapy someone from going blind who would trials in age-related macular otherwise loose their sight from a genetic degeneration (AMD). How would disease. Genetic mutations are occurring What are the key messages of your this work? all the time, so when you have a baby, for Keeler Lecture? The concept of gene therapy is to use a virus instance, that child will have 70 mutations Gene therapy is now actually an approved to deliver a gene under the retina to the that are not in the DNA of either the mother treatment, so it is not just about research, retinal epithelium. We’ve demonstrated or the father. Most of them are silent and do but it’s actually something patients can have.
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