Beacon Therapeutics enters gene therapy arena with $120m launch

Beacon Therapeutics has kickstarted its entry into the gene therapy field with a $120m Series A financing.

The British investment trust Syncona Limited launched the new ophthalmic gene therapy company by combining Applied Genetic Technologies Corporation’s (AGTC’s) late-stage X-linked retinitis pigmentosa (XLRP) programme with two proprietary preclinical programmes.

The funding, with participation from Oxford Science Enterprises (OSE), includes AGTC’s acquisition and capital to help accelerate Beacon Therapeutics’ candidate development. The total financing was £96m ($120m).

In late 2022, Syncona acquired AGTC for $23.5m and with it a portfolio of ophthalmic pipeline candidates. Amongst it was AGTC’s lead clinical candidate, AFTC-501, an adeno-associated virus (AAV) gene therapy for XLRP. The asset, now transferred to Beacon Therapeutics, is currently in Phase II clinical trials.

According to Beacon Therapeutics, AGTC-501 is unique from other treatment approaches since it addresses the entirety of photoreceptor damage – both rod and cone loss – caused by XLRP. Beacon Therapeutics says it expects 12-month data from the SKYLINE trial in H2 2023.

Beacon Therapeutics also has two preclinical assets in its portfolio: an intravitreally (IVT) delivered AAV based program for dry age-related macular degeneration (dry AMD) and a programme licensed from the University of Oxford that targets cone-rod dystrophy.

Beacon Therapeutics is the third gene therapy company Syncona has launched. Two previous portfolio investments, Nightstar and Gyroscope, were sold for $877m to Biogen and $1.5bn to Novartis respectively.

“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,” said David Fellows, Beacon Therapeutics’ CEO in a statement announcing the company launch.

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