Beacon lights up with $170m for retinal disease gene therapy

UK-headquartered Beacon Therapeutics has raised a whopping $170 million in second-round financing that will be used to advance a gene therapy for an inherited cause of blindness through a pivotal trial.

The Series B – led by Forbion and joined by other investors, including Syncona, Oxford Science Enterprises, and Oxford University Innovation – takes the total raised by Beacon since its formation last year to $290 million, making it a rising star on the UK life sciences scene.

It is focusing on retinal diseases, with its most advanced programme AGTC-501 for X-linked retinitis pigmentosa (XLRP), which is in the phase 2/3 VISTA trial.

AGTC-501 is vying to become the first-ever treatment for XLRP, an inherited condition affecting one in 40,000 people globally that causes boys to develop night blindness by the age of 10, and generally leads to legal blindness when they reach their early forties.

The gene therapy was acquired from US biotech AGTC in 2022 by Syncona, which set up and bankrolled Beacon in its early days. Meanwhile, Beacon’s pipeline also features two preclinical-stage programmes for dry age-related macular degeneration (AMD) and cone-rod dystrophy (CRD).

Part of the latest financing will be used to start a phase 1/2 study of the candidate for dry AMD, a leading cause of irreversible vision loss in people over 60 with no approved treatments.

The first patient was enrolled into the VISTA study last month, and Syncona reckons that efficacy results will be available sometime in 2026.

The gene therapy has already generated efficacy and safety data in a phase 1/2 trial called HORIZON, as well as 12-month data from the phase 2 SKYLINE study, including improvements in visual function in treated versus untreated eyes and a favourable safety profile. 24-month data from SKYLINE is due in the latter half of this year.

XLRP is predominantly caused by mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene, and AGTC-501 is designed to deliver a full-length replacement for the RPGR protein.

Other gene therapy candidates coming down the industry pipeline for XLRP include Johnson & Johnson/ MeiraGTx’s botaretigene sparoparvovec (bota-vec, formerly AAV-RPGR), which reported phase 1/2 results in 2022 and is in the phase 3 LUMEOS trial, and Frontera’s FT-002, which started phase 1/2 clinical testing last year.

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