Syncona launches ophthalmic gene therapy venture Beacon Therapeutics


The new company debuted with £96 million in funding to target retinal diseases.

A scientist working at a computer station in a lab, where a gene is mapped on the screen behind her. Image credit: ©RFBSIP –

The new company, Beacon Therapeutics, will focus on "rare and prevalent retinal diseases." Image credit: ©RFBSIP –

Life sciences investment trust Syncona launched its newest venture, Beacon Therapeutics, on Monday. In a news release, representatives from Beacon Therapeutics spoke to the company’s development pipeline and gene therapies already underway. Beacon Therapeutics aims to be a leader in the ophthalmic gene therapy space, with a focus on "rare and prevalent retinal diseases that result in blindness."1

According to the release, Syncona Limited partnered with additional investors including OSE to provide the new therapeutic venture with £96 million ($120 million) in funding. In November 2022, Syncona acquired Applied Genetic Technologies Corporation (AGTC). Beacon Therapeutics called AGTC-501, a gene therapy program currently in Phase II clinical trials, its “lead clinical asset.” In a press release, the company said AGTC-501 will be used for treatment of X-linked retinitis pigmentosa (XLRP).2

The new company also announced two preclinical assets.1 First, an intravitreally (IVT) delivered novel AAV based program for dry age-related macular degeneration (dry AMD). The second is an asset targeting cone-rod dystrophy (CRD) caused by a null mutation in the CDHR1 gene.

Beacon Therapeutics launched a new location in London, UK. This is the company’s first facility in Europe; it has two US-based locations in Boston, Massachusetts and Alachua, Florida.

Professor Robert MacLaren, Professor of Ophthalmology, University of Oxford, a co-founder of Beacon Therapeutics, provided a statement for the news release. “Beacon Therapeutics is quite unique in being a company that at launch is already underpinned by excellent clinical trial data,” MacLaren said.1 “The company has both highly innovative retinal gene therapy programmes and the means to target them to the main causes of blindness in both the young and old.”

David Fellows will serve as the Chief Executive Officer of Beacon Therapeutics. Retinal expert Dr Nadia Waheed will serve as Chief Medical Officer. Dr Abraham Scaria was announced as Chief Scientific Officer, coming aboard from AGTC.


1. Beacon Therapeutics launches with £96 million ($120 million) to develop a new generation of gene therapies for retinal diseases resulting in blindness. News release. Syncona Ltd; June 12, 2023. Accessed June 12, 2023.
2. Launch of Beacon Therapeutics and New Scenic Clinical Trial Sponsorship. News release. Beacon Therapeutics; June 12, 2023. Accessed June 12, 2023.
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