Beacon Therapeutics spun out by Oxford and Syncona raises £96 million for retinal gene therapy

Monday 12th Jun 2023, 11.46am

At the heart of Beacon’s mission is Professor Robert MacLaren, a distinguished academic leader in the world of ophthalmology and co-founder of Beacon Therapeutics. For the past ten years, Professor MacLaren has tirelessly explored the application of gene and cell therapies to combat sight loss across various complex conditions, including macular degeneration, a condition which affects an estimate 1 in 200 people in the UK by the aged of 60. MacLaren’s ground-breaking work, emanating from the Nuffield Laboratory of Ophthalmology in the Department of Clinical Neurosciences and supported by the NIHR Oxford Biomedical Research Centre (BRC), laid the foundation for Beacon Therapeutics.

‘As a retinal specialist, I became interested in gene therapy when I noticed impressive advancements in the understanding of genetics, but nothing to treat genetic diseases causing blindness in young people,’ explained Professor MacLaren, Professor of Ophthalmology at the University of Oxford. ‘Genetic diseases are now the most common cause of untreatable blindness in young people and this motivates us to search for solutions,” said MacLaren. “I was driven to bridge this gap by taking the science, developing it further and then testing it in clinical trials. This translational approach proved successful as it required both clinical expertise and scientific knowledge.’

This passion led to the creation of Nightstar Therapeutics, a spinout company created with Oxford University Innovation in 2014. Nightstar developed rapidly, licensing additional research programs and attracting over £75m in external investment. The company held its initial public offering on the NASDAQ within just 3 years, the fastest an Oxford University company had ever grown from spinout to publicly traded company. The flotation was later followed by an acquisition in 2019 by biotech firm Biogen for $800m – which also stands as a record merger and acquisition for a spinout born out of the University. Despite very promising results in clinical trials, Biogen decided to reduce its focus on ophthalmology to concentrate on neurological diseases, which has resulted in some of this vital intellectual property being returned to the University.

‘Venture capitalists recognised in both Nightstar and now Beacon Therapeutics, that we have not just an exploratory research programme but a clinical trial with real patient data, and positive data at that. Hence, they were willing to invest significantly,’ said MacLaren. ‘Our approach to de-risking has been a crucial step. As a practicing ophthalmologist, I have a deep understanding of the diseases and can assess the technical details and potential risks. Many programmes fail to reach clinical trials due to perceived high risks, and some trials reveal unexpected toxic effects. By successfully translating the work from the lab into a clinical trial, we significantly de-risk the programme for investors.’

‘Due to the large number of patients with genetic eye conditions now being assessed in our clinics, we can explore different clinical trial approaches and measures of efficacy that could be more suitable for regulatory approval,’ explained MacLaren. “Our plan is to propose alternatives to regulators and demonstrate their potential to improve the sight of many affected by blindness, whilst also allowing for more efficient and cost-effective trials with smaller patient populations. In brief, we provide not just the novel gene therapy technology, but also the detailed clinical trial design that will lead to its approval.”
Beacon Therapeutics has acquired a pre-clinical asset from Professor MacLaren’s lab targeting cone-rod dystrophy (CRD) which is caused by a null mutation in the Cadherin Related Family Member 1 (CDHR1) gene.

The launch of Beacon Therapeutics represents one of the largest biotech launches in recent UK memory. With strong links to the University of Oxford and the unprecedented support of investors such as Syncona and Oxford Science Enterprises, Beacon Therapeutics is poised to revolutionise the field of ophthalmology.

Professor Irene Tracey, Vice Chancellor at The University of Oxford, said: ‘Beacon Therapeutics represents the culmination of years of dedication from Robert, his colleagues within the University, the visionaries at Nightstar, and supporters ranging from the NIHR BRC through to Syncona. Beacon Therapeutics will push the boundaries of gene therapy and provide hope to those affected by blindness.’