#United States #Salt Lake City #XLRP #laru-zova #Beacon Therapeutics

Introduction

Beacon Therapeutics Holdings Limited, a pioneering clinical-stage biotechnology company, has recently announced an important upcoming presentation at the Association for Research in Vision and Ophthalmology (ARVO) 2025 Annual Meeting. Scheduled to take place from May 4-8, 2025, in Salt Lake City, this event will showcase preliminary six-month data from the Phase 2 DAWN trial focused on laru-zova, a promising new gene therapy developed for patients suffering from X-linked retinitis pigmentosa (XLRP).

Presentation Details

The spotlight will be on a presentation titled "Subretinal gene therapy laru-zova (AGTC-501) for X-linked retinitis pigmentosa (XLRP) Phase 2 DAWN preliminary month 6 results.” This insightful session is part of a broader agenda covering clinical advances in cell and gene therapy, scheduled for May 6, 2025. The presentation will be delivered by Dr. Mark Pennesi, a renowned expert in the field and Director of Ophthalmic Genetics at the Retina Foundation in Dallas, Texas. He also serves as a Professor of Ophthalmology and Molecular and Medical Genetics at the Casey Eye Institute, Oregon Health and Science University.

About laru-zova

Laru-zova (laruparetigene zovaparvovec) aims to establish itself as a best-in-class gene therapy targeting XLRP, an inherited retinal disease that predominantly afflicts males. This innovative therapy has the potential to restore the functionality of retinal cells (rods and cones) by delivering a valid copy of the RPGRORF15 gene, essential for producing the full-length protein necessary for normal vision. The treatment has gained significant regulatory momentum, holding various designations such as Regenerative Medicine Advanced Therapy (RMAT) and Fast Track from the U.S. FDA, Priority Medicines (PRIME) from the EMA, and Orphan Drug Designation from both the FDA and EMA.

Study Overview

The DAWN study is an open-label Phase 2 clinical trial designed to assess the efficacy, safety, and tolerability of laru-zova in male patients previously treated with another AAV vector-based gene therapy. This study is essential for establishing dosing regimens while evaluating various aspects of visual function, including low luminance visual acuity (LLVA) data. The VISTA trial, a pivotal Phase 2/3 randomized controlled study, is in progress to systematically evaluate the treatment's effects in a larger cohort, comparing it against untreated participants.

The Impact of XLRP

X-linked retinitis pigmentosa, which impacts approximately 1 in 25,000 males primarily, is characterized by mutations in the RPGR gene. This genetic defect leads to gradual visual loss often starting in childhood and can affect quality of life dramatically as it leads to complete blindness. With limited approved treatments available, laru-zova represents a hopeful leap toward reversing the condition's debilitating effects.

Conclusion

Beacon Therapeutics is committed to forging ahead in the treatment landscape for XLRP. With the Phase 2 DAWN trial providing vital safety and efficacy data, the upcoming presentation at ARVO 2025 marks a critical step in their mission to innovate potential therapies for vision restoration. The ongoing enrollment for the VISTA trial further indicates the company’s dedication to advancing research against retinal diseases, holding great promise for the future of gene therapies. By drawing attention to the challenges presented by XLRP and showcasing potential solutions, Beacon Therapeutics continues to be a key player within the biotechnology sector.