12 June 2023: Syncona Ltd has introduced its third gene therapy company Beacon Therapeutics Holdings Ltd. Beacon Therapeutics will bring together two preclinical ophthalmologic programs owned by Syncona with Applied Genetic Technologies Corp's (AGTC's) advanced X-linked retinitis pigmentosa (XLRP) program. Syncona has allocated £75 million to Beacon, which includes the previously invested funds used to acquire and restructure AGTC. As a result, Syncona will hold a 65.3% ownership stake in Beacon, currently valued at £60 million. The total amount raised in the Series A financing for this venture is £96 million.

Beacon Therapeutics plans to utilise the funds raised in the Series A financing to advance their gene therapy research and reach significant milestones that will enhance their value. This includes the progression of their leading program, AGTC-501, which is currently undergoing Phase II testing as a treatment for X-linked retinitis pigmentosa (XLRP), an inherited disorder causing progressive vision loss in males. AGTC-501 has shown promising results in its recent Phase I/II HORIZON trial, demonstrating both meaningful effectiveness and a favourable safety profile. The Phase II SKYLINE trial is underway, and the company expects to release the 12-month data in the second half of 2023.

Beacon has two pre-clinical initiatives in its portfolio. The first one focuses on developing a gene therapy for dry age-related macular degeneration (AMD), which is the primary cause of blindness in individuals aged 60 and above. This therapy is administered through intravitreal delivery. The second program, acquired from the University of Oxford, aims to address cone-rod dystrophy (CRD), a set of inherited disorders that result in vision loss by targeting the retina.

Chris Hollowood, Chief Executive Officer of Syncona Investment Management Limited, said in its official press release, "By acquiring AGTC and combining its late-stage programme with highly complementary and innovative science from the University of Oxford and elsewhere, we have created an exciting new gene therapy company in an area where we have a great breadth and depth of knowledge and expertise. The creation of this unique company demonstrates Syncona’s creativity and the proactive and proprietary nature of our approach to sourcing new opportunities.

As Syncona continues to invest in cutting-edge treatments, the launch of Beacon Therapeutics reaffirms their commitment to advancing gene therapies and providing hope for those affected by vision loss. With Beacon's expertise and Syncona's support, the future holds great potential for groundbreaking advancements in the field of ocular gene therapy.