Sep 21, 2026 —
Beacon Therapeutics reported positive topline results from VISTA, the pivotal Phase 2/3 trial of laru-zova (laruparetigene zovaparvovec), an AAV gene therapy for X-linked retinitis pigmentosa (XLRP). It is the first XLRP gene therapy to meet the primary endpoint of a pivotal trial, an indication where earlier programs from Janssen and Biogen came up short.
XLRP is an inherited retinal disease caused by mutations in the RPGR gene. It progressively destroys photoreceptors and affects roughly 1 in 25,000 males. Laru-zova is a single subretinal injection of an AAV vector carrying a functional copy of RPGR.
VISTA enrolled 85 male patients aged 12 to 48, with low-light visual acuity response at Month 12 as the FDA-endorsed primary endpoint. A gain of at least 15 letters in low-light visual acuity was reached by 31.0% of the high-dose group (p=0.0019) and 24.1% of the low-dose group (p=0.0106), versus none of the untreated controls.
