Beacon Therapeutics' gene therapy succeeds in phase 2 trial for rare inherited eye disease

Beacon Therapeutics announced on September 21, 2026, that its gene therapy, laru-zova, met its primary goal in a trial of 85 male subjects with X-linked retinitis pigmentosa, a rare disease with no approved treatments. The therapy improved low-light reading ability, whereas the control group saw no improvement. CEO Lance Baldo stated this positive readout gives Beacon a chronological advantage over competitors like Johnson & Johnson and Biogen. Beacon plans to discuss marketing submissions with global regulators.

Phase 2 trial results

• ▪In the 12-month trial of laru-zova, a significant proportion of participants receiving the therapy improved their ability to read at least 15 additional letters in low-light conditions
• ▪No participants in the untreated control group of the 12-month trial of laru-zova for X-linked retinitis pigmentosa achieved the improvement of reading at least 15 additional letters in low-light conditions
• ▪Beacon Therapeutics announced on September 21, 2026, that its experimental gene therapy, laru-zova, met its primary goal in a 12-month trial evaluating 85 male subjects aged 12 to 48

X-linked retinitis pigmentosa background

• ▪X-linked retinitis pigmentosa is an inherited eye disease that predominantly affects boys and young men, beginning with night blindness and leading to progressive vision loss and a narrowing of the peripheral field of vision
• ▪According to National Institutes of Health data, X-linked retinitis pigmentosa affects approximately 4 in 100,000 males across the United States and Europe, and currently has no approved treatments

Competitive advantage claims

• ▪Beacon Therapeutics Chief Executive Officer Lance Baldo stated that the company has a chronological advantage as the only company with a positive readout on its primary endpoint within a pivotal phase three study for X-linked retinitis pigmentosa
• ▪Beacon Therapeutics succeeded in developing a treatment for a genetic eye disease where larger drugmakers, including Johnson & Johnson and Biogen, have failed

Beacon Therapeutics profile

• ▪Beacon Therapeutics announced plans to hold discussions with global regulatory authorities regarding a marketing application submission based on the results of its 12-month trial of 85 male subjects with X-linked retinitis pigmentosa treated with its gene therapy, laru-zova
• ▪Beacon Therapeutics is a privately held company developing experimental gene therapies, including laru-zova for the treatment of X-linked retinitis pigmentosa