Beacon Therapeutics to develop new gene therapies for retinal diseases with $120 million in new funding


The company’s pipeline includes treatments for XLRP, AMD, and cone-rod dystrophy (CRD).

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The company’s pipeline includes treatments for XLRP, AMD, and cone-rod dystrophy (CRD). (Image Credit: Adobe Stock/DigitalGenetics)

Beacon Therapeutics Holdings Limited will develop new gene therapies to treat a range of rare and prevalent retinal diseases that result in blindness. With $120 million (£96 million) in funding from Syncona Limited, with additional investors including OSE, use capital to take its development candidates through to value inflection points.

Beacon Therapeutics’s lead clinical asset is AGTC-501, a gene therapy program currently in Phase II clinical trials for the treatment of XLRP that was acquired as part of Syncona’s acquisition of AGTC in November 2022. XLRP is predominantly caused by mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene. Unlike other approaches in the space, AGTC-501 correctly expresses the full length RPGR protein, thereby addressing the entirety of photoreceptor damage caused by XLRP, including both rod and cone loss.

Beacon Therapeutics’s first pre-clinical asset is an intravitreally (IVT) delivered novel AAV based program for dry Age-related Macular Degeneration (dry AMD). The second pre-clinical asset is targeting cone-rod dystrophy (CRD) which is caused by a null mutation in the Cadherin Related Family Member 1 (CDHR1) gene. The program has been licensed from the laboratory of Professor Robert MacLaren, Professor of Ophthalmology at the University of Oxford. Prof. MacLaren, a world leading retinal expert and an experienced biotech company founder and a Co-Founder of Beacon Therapeutics, will become a key scientific advisor to the company and will join the board of directors.

To bolster its pipeline in the future, Beacon Therapeutics also has access to a target generation technology platform that will identify, screen, and search secreted proteins in the ophthalmology space.

Beacon Therapeutics will be led by David Fellows, the former Chief Executive of Nightstar Therapeutics with over 40 years’ experience in the ophthalmology field. He will be joined by retinal expert Dr. Nadia Waheed as Chief Medical Officer, formerly from Gyroscope Therapeutics. Dr. Abraham Scaria also joins the team from AGTC as Chief Scientific Officer bringing 25 years of experience in gene therapy, ranging from discovery research to early-stage clinical trials.

David Fellows, Chief Executive Officer of Beacon Therapeutics commented in the press release: “Beacon Therapeutics combines a broad development pipeline, a deep scientific foundation, a strong clinical network, and a highly experienced management team to drive forward a unique late-stage clinical and pre-clinical pipeline. With the 12-month data from our Phase II SKYLINE trial for AGTC-501 expected shortly and two highly innovative and differentiated pipeline assets for prevalent and rare blinding diseases, we are excited to be building a new leader in the ophthalmic gene therapy space.”

Chris Hollowood, CEO of Syncona Limited, the lead investor in Beacon Therapeutics commented: “By acquiring AGTC and combining its late-stage program 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. We look forward to working closely with Beacon Therapeutics’ exceptional management team to advance these assets through the clinic and towards people suffering from debilitating retinal diseases.”

Professor Robert MacLaren, Professor of Ophthalmology, University of Oxford, and Co-Founder of Beacon Therapeutics commented: “I have dedicated many years to translating pioneering gene therapies from the lab through to clinical trial phase with the support of my team in the Nuffield Laboratory of Ophthalmology and NIHR Oxford Biomedical Research Centre (BRC). Beacon Therapeutics is quite unique in being a company that at launch is already underpinned by excellent clinical trial data. 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.”

Katya Smirnyagina, Senior Partner, Life Sciences, Oxford Science Enterprises (OSE) added: “Beacon Therapeutics is truly an exciting new company built around an established scientific foundation and a wealth of experience in the gene therapy and ophthalmology space. We are delighted that expertise spun out from the University of Oxford will become a key component to drive the future success of the business that could bring significant change in the treatment of a broad range of retinal diseases that cause blindness.”

Broad ophthalmic development pipeline

The company’s lead clinical gene therapy program AGTC-501 has a strong body of clinical evidence targeting XLRP, an inherited rare form of retinitis pigmentosa that causes progressive vision loss in boys and young men. AGTC-501 demonstrated meaningful efficacy and a good safety profile in the recent Phase I/II HORIZON trial and expects to be publishing 12-month data from its Phase II SKYLINE trial in the second half of 2023. In addition, Beacon Therapeutics is awaiting feedback from the US Food and Drug Administration (FDA) regarding the study design of its upcoming VISTA clinical trial, a Phase II/III study to assess the effect of AGTC-501 on the symptoms of retinitis pigmentosa in additional patients with XLRP.

The first preclinical program features intravitreally (IVT) delivered gene therapy for dry AMD. IVT delivery provides access to a significantly greater number of patients than sub-retinal delivery as it can be administered within an outpatient clinic rather than requiring surgery.

The second preclinical program, CRD, targets mutations in the CDHR1 gene, which may cause central vision loss similar to dry AMD in older people and severe sight loss in younger patients.

The Company has a new location in London, UK, complementing existing facilities in Boston, Massachusetts and Alachua, Florida, US.


Beacon Therapeutics launches with £96 million ($120 million) to develop a new generation of gene therapies for retinal diseases resulting in blindness. Press release. Beacon Therapeutics, June 12, 2023. Accessed June 12, 2023.

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