Beacon gene therapy VISTA trial reports positive results for X-linked retinitis pigmentosapositive results for X-linked retinitis pigmentosa

What is X-linked retinitis pigmentosa?

X-linked retinitis pigmentosa (XLRP) is an inherited retinal disease most commonly caused by faults in the retinitis pigmentosa GTPase regulator (RPGR) gene, although other genes can also cause the condition. Normally, the protein produced from the RPGR gene acts like a traffic controller, helping molecules get where they need to go inside photoreceptors, the light-sensing cells of the retina. However, in most cases of XLRP, this organisation breaks down due to faulty RPGR, and the photoreceptors start to die off.

The condition primarily affects males and typically begins in childhood, causing progressive loss of vision. People with XLRP often experience night blindness (nyctalopia) and narrowing of their visual field (tunnel vision) before progressing to severe sight loss.

There are currently no approved treatments that can slow or stop the progression of XLRP.

What is laru-zova?

Laru-zova (laruparetigene zovaparvovec) is an experimental gene therapy designed to deliver a healthy copy of the RPGR gene to retinal cells. Researchers hope this will enable the retina to produce working RPGR protein and thereby improve visual function.

The treatment is delivered as a single injection beneath the retina and is intended to restore the function of both rod and cone photoreceptor cells.

The VISTA trial

The VISTA trial enrolled 85 male participants aged 12 to 48 years with XLRP caused by RPGR mutations. Participants were assigned to receive either a high or low dose of laru-zova, or to an untreated control group. The study assessed how safe and effective the treatment was over 12 months.

The trial’s primary endpoint measured changes in low luminance visual acuity (LLVA), which tests how well a person can see in dim light. Difficulty seeing in these conditions is one of the earliest and most challenging symptoms experienced by people with XLRP.

Findings

Beacon reported that the VISTA trial met its primary endpoint, making it the first XLRP trial in the world to do so. At 12 months, about 1 in 3 participants receiving the higher dose of laru-zova and about 1 in 4 participants receiving the lower dose could read at least 15 more letters on an eye chart in dim lighting. No participants in the untreated control group achieved this level of improvement.

The company also reported encouraging results from additional measures of vision, including the sensitivity of their retinas to light. The therapy demonstrated a favourable safety profile, with most side effects reported as mild to moderate and related to the surgical procedure used to deliver the treatment.

What happens next?

Based on these results, Beacon Therapeutics is beginning discussions with regulatory authorities and expects to start a rolling application for approval later this year. More data from the VISTA trial will be presented at the American Academy of Ophthalmology Annual Meeting on the 10^th of October 2026.

If regulatory submissions are successful, the review and approval process is likely to take several months. Even after a regulatory approval, more assessments would be needed before the treatment could become routinely available on the NHS. Retina UK will continue to monitor developments and keep our community updated as more information becomes available.

While laru-zova hasn’t yet been approved for use, these findings represent an important step forward for the XLRP community and provide further evidence that gene therapy could become a viable treatment option.

Learn more

To learn more about Beacon Therapeutics and the VISTA trial, visit the company’s website at beacontx.com or search for NCT04850118 on ClinicalTrials.gov.