X-linked retinitis pigmentosa (XLRP) is an incurable genetic disease that causes blindness in men. Credit: Tracy Spohn via Shutterstock.

Beacon Therapeutics has raised $170m in a Series B funding round to accelerate the development of its lead asset AGTC-501, for X-linked retinitis pigmentosa (XLRP).

The financing round was led by life sciences venture capital firm Forbion, alongside existing investors Syncona Limited, Oxford Science Enterprises and the University of Oxford, and also featured initial investments from TCGX and Advent Life Sciences.

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The British investment trust Syncona previously acquired Applied Genetic Technologies Corporation (AGTC) for $23.5m in October 2023. AGTC inherited a portfolio of ophthalmic pipeline candidates, including AFTC-501, an adeno-associated virus (AAV) gene therapy. XLRP is a genetic disorder that causes progressive vision loss, primarily in males, due to retinal degeneration.

AFTC-501, now transferred to Beacon, has been investigated in several clinical trials for XLRP. Promising interim data from the Phase II SKYLINE trial (NCT03316560) was announced in February 2024, showing that 63% of eyes treated with high-dose AGTC-501 experienced retinal sensitivity improvement of at least seven decibels (dB) in at least five loci after 12 months.

Following on from this data, the first patient was enrolled in the pivotal Phase II/III VISTA registrational trial in June 2024. In the announcement, Beacon said it will use the data from this and previous trials to support a biologics license application (BLA) in the US and a marketing authorisation application (MAA) in Europe.

XLRP is predominantly caused by mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene. AGTC-501 expresses the full-length RPGR protein and is therefore expected to rescue the function of both rod and cone photoreceptors. Beacon says this makes AGTC-501 unique from other treatment approaches since it addresses the entirety of photoreceptor damage.

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The London-based biotech launched in 2023 with $120m under its belt to develop gene therapies for ophthalmic indications. The latest round – which brings Beacon’s total funding up to $290m – will also be used to generate a Phase I/II trial for its dry age-related macular degeneration programme.

In the announcement accompanying the funding, Beacon’s CEO David Fellows said: “We are focused on progressing our pipeline of ophthalmic gene therapies to save and restore the vision of patients with a range of prevalent and rare retinal diseases that result in blindness.”

Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content.