Beacon Therapeutics Announces Positive Phase 2 Interim 6-Month Data from DAWN Trial of Laru-zova in Patients with X-linked Retinitis Pigmentosa (XLRP) at ARVO 2025

Core Insights - Beacon Therapeutics announced promising 6-month interim safety and efficacy results from the Phase 2 DAWN trial of laru-zova for patients with X-linked retinitis pigmentosa (XLRP) [1][2] - Laru-zova demonstrated improvements in visual function, including low luminance visual acuity and microperimetry, and was generally well-tolerated by participants [1][5] Company Overview - Beacon Therapeutics is a clinical-stage biotechnology company focused on developing treatments for blinding retinal diseases, with laru-zova as its lead program [11] - The company has a scientific foundation that includes a late-stage clinical candidate for XLRP and two preclinical programs targeting dry age-related macular degeneration and inherited cone-rod dystrophy [11] Product Details - Laru-zova (laruparetigene zovaparvovec) is a gene therapy designed to restore the function of rods and cones in XLRP by delivering a functional copy of the RPGR gene [2][6] - The therapy has received several designations from regulatory agencies, including Regenerative Medicine Advanced Therapy (RMAT) and Fast Track from the FDA, and Orphan Drug Designation from both the FDA and EMA [6] Clinical Trials - The DAWN trial is an open-label study assessing the efficacy, safety, and tolerability of laru-zova in participants with XLRP who have previously received gene therapy [3][8] - The VISTA trial is a pivotal Phase 2/3 study currently enrolling participants to evaluate the efficacy and safety of laru-zova compared to an untreated control group [9] Disease Context - XLRP is an inherited retinal disease affecting approximately 1 in 25,000 males, leading to progressive vision loss and eventual blindness, with no approved treatments available [10]