Beacon Therapeutics' laruparetigene zovaparvovec met the primary endpoint in the pivotal VISTA trial in X-linked retinitis pigmentosa (XLRP), with both tested doses producing significantly higher rates of clinically meaningful low-luminance visual acuity improvement than untreated control. Beacon said it plans to begin a rolling BLA submission before year-end for the gene therapy, which is being developed for a disease with no approved treatments.
Laru-zova delivers a functional copy of the full-length RPGR ORF15 gene using an adeno-associated virus vector, with the aim of restoring function across both rod and cone photoreceptors affected by RPGR mutations. The VISTA primary endpoint measures low-luminance visual acuity, a functionally relevant measure in XLRP because patients typically experience early difficulty seeing in dim-light conditions before more advanced visual loss develops.
The result also distinguishes Beacon's program from MeiraGTx's botaretigene sparoparvovec, another RPGR-directed AAV gene therapy that failed to meet the primary endpoint in the Phase III LUMEOS trial in May 2025. MeiraGTx subsequently reacquired bota-vec from Johnson & Johnson in April 2026 and has said it still intends to pursue regulatory filings. LUMEOS used a navigation-based functional vision endpoint, while VISTA's FDA-endorsed primary endpoint focused on low-luminance visual acuity.
VISTA enrolled 85 male patients aged 12 to 48 with RPGR-associated XLRP and randomized them to high-dose laru-zova, low-dose laru-zova, or an untreated control. At Month 12, 31.0% of patients in the high-dose group and 24.1% in the low-dose group achieved at least a 15-letter improvement in low-luminance visual acuity, compared with no responders in the control group. At least a 10-letter improvement was achieved by 48.3% and 58.6% of patients in the two dose groups, respectively, versus 3.7% of controls.