Portfolio

Autolus is a CAR T cell therapy company, applying its extensive programming capabilities to develop advanced autologous T cell therapies that have the potential to deliver life-changing therapies to patients with cancer and autoimmune diseases.

Beacon is a leading ophthalmic gene therapy company with a purpose to restore and improve the vision of patients with a range of prevalent and rare retinal diseases that result in blindness. Syncona launched Beacon in June 2023, having invested £75 million of the £96 million Series A financing. As part of this Series A financing, AGTC was combined with new Syncona company, Beacon Therapeutics, merging AGTC’s lead clinical gene therapy asset with two exciting pre-clinical programmes, including one from the University of Oxford. In July 2024 Syncona committed an additional $42.5 million (£33.5 million) to a $170 million (£134 million) Series B financing, bringing the total amount that Beacon has raised in funding to date to approximately $290 million.

Syncona believes the eye is a very attractive target for AAV gene therapy, as exemplified by the number of programmes in development and the approval of voretigene neparvovec (Luxturna) by the FDA in 2017. It is a small, compartmentalised, tissue to which gene therapy vectors have been demonstrated to be delivered directly in a safe manner, meaning that the vector can reach the target cells more readily and lower doses are required. Furthermore, the target cells are terminally differentiated and therefore AAV gene therapy can provide a prolonged effect, as demonstrated by the various programmes in the clinic to date.

Beacon is Syncona’s third investment in the ophthalmic gene therapy space, in which Syncona has an impressive track record of creating, building and scaling gene therapy companies, with Nightstar being sold for $877m, at a 4.5x multiple of cost, and Gyroscope up to $1.5bn, at a potential 5.1x multiple of cost.

Beacon’s lead asset, AGTC-501, is a gene therapy program currently in Phase II clinical trials for the treatment of XLRP, an inherited monogenic recessive disorder that causes progressive vision loss in boys and young men. Most people with the disease progress to becoming legally blind by their 40s. XLRP is predominantly caused by mutations in the RPGR gene. AGTC-501 expresses the full length RPGR transgene, thereby addressing the full complement of photoreceptor damage caused by XLRP, including both rod and cone loss. There are no approved treatments for XLRP and the programme has orphan drug designations from both the FDA and the European Commission.

Beacon represents a significant opportunity for Syncona to apply its deep expertise in retinal gene therapy to a late-stage clinical asset where Syncona already has prior expertise. We believe that the platform potential of Beacon is incredibly exciting, and has the potential to drive near-term value for our shareholders.

Spur Therapeutics is a clinical-stage biotechnology company focused on developing life-changing gene therapies for debilitating chronic conditions.

iOnctura is a clinical-stage oncology company developing innovative therapies for neglected and hard-to-treat cancers.

Quell Therapeutics was founded with the aim of developing engineered T regulatory (Treg) cell therapies. Tregs are a subset of T cells with the potential to downregulate the immune system.

Anaveon is a clinical phase biopharmaceutical company that specialises in the development of treatments for diseases with immune system dysfunction.

Based in Cambridge, UK, Mosaic is an oncology therapeutics company with a highly specialised drug discovery platform, focused on discovering, developing and commercialising novel, targeted, therapies to target cancers in areas of high unmet need. Syncona led a £22.5 million Series A financing of the company in 2022, committing £16.5 million alongside Cambridge Innovation Capital.

Resolution is developing macrophage cell therapies to repair inflammatory organ damage.

Purespring seeks to advance gene therapies for the treatment of chronic renal diseases that are currently poorly addressed with existing treatments.

Forcefield is a pioneer of best-in-class therapeutics to protect heart function by arresting the loss of cardiomyocytes following myocardial infarction (heart attacks). Myocardial infarction (MI) triggers the irreversible loss of large numbers of cardiomyocytes, or heart cells, which can lead to a cascade of events leading to heart failure. Up to 30% of cardiomyocytes can be lost during and immediately after MI.

OMass Therapeutics is an Oxford University spin-out that is using novel biochemistry techniques, native mass spectrometry and custom chemistry to deliver novel medicines against highly validated but inadequately drugged targets, with a focus on immunological and rare diseases.

Kesmalea is a small molecule drug discovery company established with the aim of creating a new generation of oral drugs addressing diseases through modulating protein homeostasis. Syncona led a £25 million Series A financing in the company in 2022, committing £20 million alongside Oxford Science Enterprises. An additional £5.0 million was raised in 2023 with Syncona committing £4.0 million.

Yellowstone is pioneering soluble T-cell receptor (TCR)-based therapies to unlock a new class of cancer therapeutics.

Slingshot, the Syncona Accelerator, is focused on accumulating a pipeline of early-stage programmes, identified from world-leading academic institutions, and accelerating their development towards the clinic.