Syncona launches new gene therapy company Beacon Therapeutics

Syncona Ltd has launched its third gene therapy company, Beacon Therapeutics Holdings Ltd, which will combine two proprietary preclinical ophtalmologic programmes of the British company with Applied Genetic Technologies Corp’s (AGTC’s) late-stage X-linked retinitis pigmentosa (XLRP) programme. Syncona has committed £75m to Beacon – including the capital already invested to acquire and restructure AGTC – and will have a 65.3% stake in the enterprise, with a current holding value of £60m. The total value of this Series A financing is £96m.

Syncona led the financing round alongside additional investors including Oxford Science Enterprises (OSE).
The Series A financing will be used to advance Beacon Therapeutics’ gene therapy candidates through to key value inflection points, including Beacon Therapeutics’ lead programme AGTC-501, which is currently in Phase II testing for the treatment of XLRP, an inherited monogenic disorder that leads to progressive vision loss in males. AGTC-501 has a strong body of clinical evidence having demonstrated meaningful efficacy and a good safety profile in its recent Phase I/II HORIZON trial. 12-month data from its Phase II SKYLINE trial is expected in H2/2023.

Beacon two pre-clinical programmes include an intravitreally delivered gene therapy for dry age-related macular degeneration (AMD), the leading cause of blindness in people over 60, and an in-licensed one from the University of Oxford that is targeting cone-rod dystrophy (CRD), a group of inherited vision loss disorders affecting the retina.

Beacon Therapeutics is Syncona’s third ophthalmic gene therapy company, following Nightstar, which sold for $877m, at a 4.5 multiple of cost, and Gyroscope, which was sold for up to $1.5bn, at a potential 5.1x multiple of cost.

David Fellows will lead Beacon Therapeutics as Chief Executive Officer (CEO), bringing 40 years’ experience in ophthalmology to the management team, including five years as Nightstar’s CEO. He will be joined by Chief Medical Officer (CMO) Dr Nadia Waheed, a retinal expert and former Chief Medical Officer of Gyroscope, and gene therapy expert Dr Abraham Scaria, who has joined as Chief Scientific Officer. Syncona Investment Management Limited CEO, Chris Hollowood, has been appointed as Chair and Syncona Lead Partner Elisa Petris will be joining the Board as a Director. Professor Robert MacLaren, Professor of Ophthalmology at the University of Oxford, will be an active advisor on Beacon Therapeutics’ CRD program and has also become a non-executive director.