Beacon Therapeutics' XLRP Gene Therapy Laru-zova Meets Primary Endpoint in Phase 2/3 VISTA: 31.0% High-Dose and 24.1% Low-Dose Patients Gain 15+ Letters of Low-Luminance Vision vs None in Controls
Beacon Therapeutics reported on Monday, September 21, 2026 that the Phase 2/3 VISTA trial of laruparetigene zovaparvovec (laru-zova), an AAV-based gene therapy that delivers full-length RPGR, met its primary endpoint in X-linked retinitis pigmentosa (XLRP). At month 12, 31.0% of high-dose and 24.1% of low-dose patients had at least a 15-letter improvement in low-luminance visual acuity, versus no patients in the untreated control group, in a trial of 85 males aged 12 to 48. Beacon says no other late-stage XLRP trial has met its primary endpoint, and it reported a favorable safety profile with mostly mild-to-moderate ocular adverse events. The company plans a rolling BLA submission later this year and will present more data at the AAO annual meeting on October 10, 2026; it cites XLRP prevalence of about 1 in 25,000 males. Syncona is among Beacon's investors.