Beacon's XLRP gene therapy meets its pivotal endpoint

Beacon Therapeutics reported on 21 September 2026 that VISTA, the pivotal trial of its gene therapy laru-zova (laruparetigene zovaparvovec), met the primary endpoint the FDA had endorsed. The trial enrolled 85 males aged 12 to 48 with X-linked retinitis pigmentosa, an inherited disease that destroys the retina’s light-sensing cells from the periphery inward.

The endpoint was a gain of at least 15 letters of low-luminance visual acuity, reading an eye chart in dim light, at month 12. It was reached by 31.0% of patients on the high dose (p=0.0019) and 24.1% on the low dose (p=0.0106), against 0% of untreated controls. On a looser threshold of 10 letters, the responder rates were 48.3% and 58.6%. Treatment-emergent adverse events related to laru-zova occurred in 25% of high-dose and 38% of low-dose patients.

Beacon describes VISTA as the first pivotal XLRP trial to reach its primary endpoint with statistical significance. The therapy has now been given to 110 participants across five years of clinical work, and the company plans a rolling Biologics License Application later in 2026.