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Beacon Therapeutics reveals promising interim results for laru-zova, a gene therapy showing potential to improve vision in XLRP patients.
(Image credit: AdobeStock/vivekFx)
Beacon Therapeutics has released 6-month interim safety and efficacy results from the Phase 2 DAWN (NCT06275620) trial for the pharmaceutical candidate laru-zova (laruparetigene zovaparvovec). This candidate is being evaluated for treatment of patients with X-linked retinitis pigmentosa (XLRP). According to the data, laru-zova was generally well-tolerated by all DAWN participants evaluated at 6 months or beyond and initial data showed promising improvements in visual function across several key measures.1
Lance Baldo, MD, Chief Executive Officer of Beacon Therapeutics spoke to these interim results in a quote to Ophthalmology Times and Modern Retina, he said, “The 6-month interim results from the Phase 2 DAWN trial further highlight laru-zova's potential to improve vision in patients with XLRP with the findings to-date demonstrating advancements in multiple visual function measures. We are committed to advancing laru-zova to offer new hope for those affected by XLRP who are currently without treatment options, and we eagerly await further data from the trial.”
DAWN is a phase 2, open-label study of laru-zova in the fellow eye of male participants with XLRP who have previously been treated with an AAV vector-based gene therapy delivering the full-length RPGR protein. The objective of DAWN is to assess 2 different dose levels of laru-zova for efficacy, safety and tolerability in the target population.1
The interim study results were presented at the 2025 ARVO meeting held in Salt Lake City, Utah. Highlights from this presentation include:
Beacon Therapeutics continues to enroll patients for its pivotal Phase 2/3 VISTA trial of laru-zova for patients with XLRP. VISTA (NCT04850118) is a Phase 2/3, randomized, controlled, masked, multi-center pivotal study evaluating the efficacy, safety and tolerability of laru-zova in two study groups compared to an untreated control group. The study will evaluate the proportion of participants with a 15 or more letter increase from baseline in LLVA and additional measures of functional vision.1
Laru-zova (laruparetigene zovaparvovec) is a potential best-in-class gene therapy currently being investigated for the treatment of patients with X-linked retinitis pigmentosa (XLRP). Laru-zova has the potential to restore the natural function of both rods and cones in XLRP by delivering a functional copy of the RPGRORF15 gene designed to produce the full-length protein.1
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