Beacon Gains $170M for Gene Treatments

In a Series B fundraising round, Beacon Therapeutics has secured $170 million to expedite the creation of AGTC-501, its key product for treating X-linked retinitis pigmentosa (XLRP).

AFTC-501 was previously studied in many clinical studies for XLRP; it is now at Beacon. In February 2024, encouraging preliminary findings from the Phase II SKYLINE study (NCT03316560) were released. It revealed that after a year, 63% of eyes administered with high-dose AGTC-501 had improved retinal sensitivity by no fewer than seven decibels (dB) in a minimum of five loci.

Based on these findings, the pivotal Phase II/III VISTA registrational study saw the enrolment of its first participant in June 2024. Beacon stated in the release that it would endorse a biologics licence application (BLA) in the US and a marketing authorisation application (MAA) in Europe with the results from this and prior studies.

With $120 million under its management, the London-based company began developing gene treatments for ocular diseases in 2023. Beacon will additionally utilise the most recent round of funding, which raises the organization’s overall investment to $290 million, to create a Phase I/II study for its dry age-related macular degeneration programme.