Data Readout

Beacon's laru-zova gene therapy nears pivotal XLRP readout

The VISTA trial's 12-month topline data, expected in the second half of 2026, will be the first pivotal test of a subretinal gene therapy for RS1-linked XLRP.

By AppliedXLJuly 13, 2026Trial NCT04850118

Executive Summary

Beacon Therapeutics is heading toward the first pivotal data readout for a gene therapy targeting the RS1 gene in X-linked retinitis pigmentosa, a disease with no approved treatment that alters its course.
The trial finished enrollment above its original target and has sat in a non-recruiting holding pattern for a year, so the remaining variable is the data itself, not trial execution.
The nearest same-target program is still in early-stage testing for a related but distinct retinal disease, leaving this readout without a direct pivotal-stage precedent to benchmark against.
A positive functional-vision result would give gene replacement therapy its first pivotal validation in this specific inherited retinal disease and inform how regulators and rival programs treat the broader RS1 and XLRP gene-therapy class.

The trial

VISTA, registered as NCT04850118, is a Phase 2/3 study evaluating laru-zova (laruparetigene zovaparvovec, formerly AGTC-501) in patients with X-linked retinitis pigmentosa caused by variants in the RS1 gene. Beacon Therapeutics said it completed enrollment in the trial in July 2025 and expects twelve-month topline data in the second half of 2026. The trial's primary outcome measure is the proportion of participants achieving at least a 15-letter increase from baseline in low-luminance visual acuity. NCT04850118+1

Enrollment and timing

The trial enrolled 85 patients, above its original 63-patient target set when the study began recruiting in March 2024. The primary completion date has moved twice since the trial's initial registration, from January 2024 to August 2025 and then to June 30, 2026, as the sponsor's enrollment and follow-up timelines extended. The trial has held Active, not recruiting status since July 16, 2025, consistent with a study that has finished dosing and is now in its 12-month follow-up period ahead of the readout. NCT04850118

The competitive field

No gene therapy has been approved for X-linked retinitis pigmentosa. Beacon's asset is the only Phase 2/3 program targeting RS1 in this indication; the nearest same-target comparator, Atsena Therapeutics' ATSN-201, is a Phase 3 program for RS1-associated X-linked retinoschisis, a related but distinct retinal disease. Programs targeting the RPGR gene, including Janssen's Phase 2 and Phase 3 XLRP gene therapies, share the same modality and indication but a different genetic target. With no validated disease-modifying mechanism established in this indication, a result showing durable functional-vision gains that hold through the full follow-up period would be the finding that distinguishes laru-zova from the broader gene-therapy field testing this disease.

Sponsor track record

Beacon Therapeutics has completed all three of its previously tracked trials with no terminations recorded, a small sample that nonetheless establishes an operational baseline heading into its first pivotal readout. The company appointed Ryan Robinson as Chief Financial Officer in March 2026, a move it tied to preparing for the potential commercialization of laru-zova. Beacon

This analysis was produced using AI-assisted reporting systems, AppliedXL data, and official public records. These systems undergo editorial review, quality checks, and regular audits by human experts. Errors may still occur, as with any automated system. Always consult the linked primary sources. Read our AI Editorial Policy.