At the American Academy of Ophthalmology (AAO) 2026 meeting, Beacon Therapeutics presented detailed results from its pivotal VISTA trial of laru-zova, an investigational gene therapy for X-linked retinitis pigmentosa (XLRP) caused by RPGR mutations.
The trial met its FDA-endorsed primary endpoint:
at Month 12, 31.0% of participants in the high-dose group and 24.1% in the low-dose group improved by at least 15 letters on a low-luminance visual acuity test, compared with none in the untreated control group. The reported p-values were 0.0019 and 0.0106, respectively.
At the ≥10-letter improvement threshold, response rates were 48.3% with high dose, 58.6% with low dose, and 3.7% in the control group.
Beacon also reported early, sustained improvement in macular sensitivity in treated participants. The therapy’s safety and tolerability profile was described as favorable and consistent with earlier studies; reported eye-related adverse events were mostly mild to moderate and often associated with the surgical procedure.
VISTA enrolled 85 male participants and compared two doses delivered to one eye against an untreated control. Laru-zova remains investigational; the company says it plans to use VISTA alongside data from other studies to support regulatory filings.
Sources:
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