Gene therapy shows durable vision gains in XLRP Phase 2 trials
Laru-zova, an investigational gene therapy for X-linked retinitis pigmentosa, continues to show durable improvements in visual function with a favorable safety profile in Phase 2 trials, according to a presentation at the EURETINA 2025 Conference.
Laru-zova was generally well-tolerated across the Phase 2 SKYLINE and DAWN trials, with participants demonstrating sustained improvements in key measures of visual function. In the DAWN trial, patients showed early gains in low luminance visual acuity (LLVA) and durable improvements in retinal sensitivity by microperimetry at 9 months and beyond. In the SKYLINE trial, high-dose participants maintained improvements in retinal sensitivity through 36 months, with greater responses compared to low-dose or untreated eyes.
“These new data updates reinforce our belief in the potential for laru-zova to be a meaningful treatment option for people living with XLRP,” said Daniel Chung, DO, MA, Chief Medical Officer of Beacon Therapeutics, in a press release.
Beacon is advancing the pivotal VISTA trial, which uses LLVA as its primary endpoint. Enrollment is complete, with topline results expected in the second half of 2026.
Reference
Anand R, et al. Subretinal gene therapy laru-zova for X-linked retinitis pigmentosa (XLRP): Phase 2 DAWN trial, preliminary month 9+ results. Presented at: EURETINA 2025; September 4-7, 2025; Paris, France.
