Beacon's Gene Therapy Shows Promise in Rare Eye Disease, While J&J Faces Setback

Beacon Therapeutics has reported encouraging interim results from its phase 2 trial of laruparetigene zovaparvovec (laru-zova), a gene therapy for X-linked retinitis pigmentosa (XLRP). The data, announced on May 6, 2025, indicate improvements in visual function for patients with this rare inherited retinal disease. Meanwhile, Johnson & Johnson's gene therapy for the same condition failed to meet its primary endpoint in a late-stage trial.

Beacon's Laru-Zova Demonstrates Early Success

Beacon's phase 2 Dawn study, evaluating laru-zova in patients with XLRP, has shown promising results at the six-month interim analysis. The therapy, designed to restore natural function of both rods and cones in patients' eyes, demonstrated improvements across several key measures of visual function, including low luminance visual acuity and eye sensitivity.

CEO Lance Baldo, M.D., stated, "This update continues to demonstrate laru-zova's potential to enhance vision in patients with XLRP." The company has built a "compelling body" of safety and efficacy data over five years through three clinical studies.

Laru-zova works by delivering a functional copy of the retinitis pigmentosa GTPase regulator (RPGR) gene to affected cells. Beacon describes it as a "potential best-in-class" candidate for XLRP, a condition affecting 1 in 25,000 males across the U.S., Europe, and Australia, with no currently available targeted treatments.

Contrasting Outcomes in XLRP Gene Therapy Landscape

While Beacon celebrates its early success, Johnson & Johnson recently reported disappointing results for its XLRP gene therapy, botaretigene sparoparvovec (bota-vec). On May 2, 2025, J&J announced that bota-vec failed to meet its primary endpoint in the phase 3 Lumeos trial, which aimed to improve patients' ability to navigate through a virtual maze.

Despite J&J's setback, the company noted improvements in several secondary endpoints, including visual acuity. However, these were not claimed as statistically significant. A J&J spokesperson stated they are "working to understand the totality of the data."

The XLRP gene therapy field has faced other challenges, with 4DMT scrapping its program earlier this year. These developments underscore the complexity of treating rare genetic eye disorders and highlight the potential significance of Beacon's early positive results.

Next Steps and Ongoing Research

Beacon Therapeutics is advancing its XLRP gene therapy program with a $170 million series B funding round secured in July 2024. The company plans to combine results from the phase 2 Dawn study and a phase 1/2 trial to support regulatory applications in the U.S. and Europe.

A larger, more definitive trial called Vista is currently recruiting patients. This phase 2/3 study is designed as a randomized, controlled trial to evaluate the efficacy, safety, and tolerability of laru-zova in two study groups compared to an untreated control group.

As the landscape of gene therapies for rare eye diseases continues to evolve, the pharmaceutical industry watches closely to see if Beacon's laru-zova can maintain its early promise and potentially become the first approved treatment for XLRP.