May 08, 2026 —
Beacon Therapeutics reported new 12-month data from the Phase II DAWN trial evaluating laruparetigene zovaparvovec, also known as laru-zova, in patients with X-linked retinitis pigmentosa (XLRP). The results were presented at the ARVO 2026 Annual Meeting in Denver and showed sustained improvements across several measures of visual function, reinforcing the potential of AAV-based gene therapy for this progressive inherited retinal disease.
XLRP is a rare inherited retinal disorder that causes progressive vision loss and can lead to legal blindness. The disease is commonly associated with mutations in the RPGR gene, and there are currently no approved treatment options. Laru-zova is designed to address the underlying genetic cause of XLRP by delivering a functional copy of the RPGRORF15 gene, with the goal of restoring the natural function of both rods and cones.
In the Phase II DAWN trial, laru-zova was generally well tolerated over 12 months. Patients demonstrated sustained improvements in key functional endpoints, including low luminance visual acuity (LLVA) and mean macular sensitivity measured by microperimetry, consistent with previously reported nine-month data. These measures are particularly important in inherited retinal disease because they help capture changes in vision under low-light conditions and assess retinal sensitivity in regions critical for functional vision.
Among patients receiving the high-dose AAV gene therapy, 50% achieved at least a two-line improvement from baseline, defined as 10 or more ETDRS letters, while 25% achieved at least a three-line improvement, defined as 15 or more ETDRS letters. In the low-dose group, 67% of patients achieved at least a two-line improvement from baseline. Sustained improvement in microperimetry mean sensitivity was also observed compared with baseline.
Beacon is continuing to advance laru-zova across multiple clinical studies. The therapy is being evaluated in the pivotal VISTA trial, for which enrollment was completed in June 2025, with topline data expected in the second half of 2026. Dosing has also begun in the LANDSCAPE trial, which is evaluating laru-zova in male participants aged 12 to 50 years with confirmed XLRP at U.S. sites.
The 12-month DAWN results add to the growing clinical evidence supporting ocular AAV gene therapy as a potential disease-modifying approach for inherited retinal diseases. For XLRP, where progressive photoreceptor dysfunction leads to irreversible vision loss, durable improvements in visual function and retinal sensitivity could represent an important step toward a first therapeutic option for patients.
