LONDON and CAMBRIDGE, Mass., April 29, 2026 (GLOBE NEWSWIRE) -- Beacon Therapeutics Holdings Limited (Beacon Therapeutics or the Company), a leading clinical-stage biotechnology company with a mission to save and restore the vision of patients with blinding retinal diseases, today announced that it will deliver five presentations at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting to be held May 3-7, 2026 in Denver, Colorado.
At ARVO, 12-month safety and efficacy data from the Phase 2 DAWN trial of laruparetigene zovaparvovec (laru-zova), the Company's lead asset for the treatment of X-linked retinitis pigmentosa (XLRP), will be featured in an oral presentation. In addition, three posters on laru-zova will be presented, including the design and baseline characteristics of the pivotal VISTA trial; a post-hoc microperimetry analysis from the Phase 2 SKYLINE trial; and 5-year safety results from the the Phase 1/2 HORIZON trial. The Company will also deliver an oral presentation on BTX-001, an adeno-associated virus (AAV)-mediated intravitreal gene therapy candidate being investigated for the treatment of geographic atrophy (GA) secondary to dry age-related macular degeneration (AMD).
Oral Presentation Details:
Presentation title: Subretinal Gene Therapy laru-zova (AGTC-501) for X-linked retinitis pigmentosa (XLRP): Phase 2 DAWN Safety and Efficacy Update
Date: Thursday, May 7, 2026
Presenter: Andreas Lauer, M.D., Director of the OHSU Casey Eye Institute and Chair for the Department of Ophthalmology
Presentation title: Development of BTX-001, a Novel AAV-Mediated Intravitreal Gene Therapy Delivering a C5 Inhibitor for Geographic Atrophy
Date: Monday, May 4, 2026
Presenter: Anna Dreismann, Executive Director, Research, Beacon Therapeutics
Poster Details:
Poster title: Subretinal Gene Therapy laru-zova for X-linked retinitis pigmentosa (XLRP): Pivotal Phase 2/3 VISTA Trial Design and Baseline Demographics
Date: Wednesday, May 6, 2026
Presenter: Ashley Simmons, Executive Medical Director, Beacon Therapeutics
Poster title: Subretinal laru-zova Gene Therapy for XLRP: Phase 2 SKYLINE Trial Post-Hoc Microperimetry Scotomatous vs. Nonscotomatous Analysis
Date: Wednesday, May 6, 2026
Presenter: Ben Rice, Senior Director, Medical Affairs, Beacon Therapeutics
Poster title: Subretinal Gene Therapy laru-zova for X-linked retinitis pigmentosa (XLRP): Phase 1/2 HORIZON 5-year Safety Results
Date: Wednesday, May 6, 2026
Presenter: Paul Yang, M.D., Ph.D. Chief, Paul H. Casey Ophthalmic Genetics Division, OHSU Casey Eye Institute
About laru-zova
Laruparetigene zovaparvovec (laru-zova) is a potential best-in-class gene therapy currently being evaluated 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. Laru-zova has Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations from the U.S. Food and Drug Administration (FDA), Priority Medicines (PRIME) designation from the European Medicines Agency (EMA), Innovative Licensing and Access Pathway (ILAP) from the UK's Medicines and Healthcare products Regulatory Agency (MHRA), as well as Orphan Drug Designation (ODD) from the FDA and EMA.
Laru-zova is investigational and has not been approved by FDA for use.
About the DAWN, SKYLINE, and HORIZON Studies
DAWN (NCT06275620) is an ongoing, fully enrolled, Phase 2, open-label trial 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 two different dose levels of laru-zova for efficacy, safety and tolerability in the target population. DAWN is also evaluating the changes in visual function and functional vision, and is the first trial in the laru-zova clinical development program that is collecting and evaluating low luminance visual acuity (LLVA) data.
SKYLINE (NCT06333249) is an ongoing, fully enrolled, Phase 2, randomized, controlled trial evaluating the safety, efficacy and tolerability of laru-zova in 14 male patients with XLRP caused by mutations in the RPGR gene. The trial's primary endpoint is the proportion of response by microperimetry between the study and fellow eye at month 12.
HORIZON (NCT03316560) is a completed 5-year Phase 1/2, open-label, dose escalation trial of laru-zova in patients with XLRP due to mutations in the RPGR gene. The trial included 29 male participants, who received a single sub-retinal dose of laru-zova in one eye to obtain initial data about safety and potential efficacy of laru-zova gene therapy.
About X-linked Retinitis Pigmentosa
X-linked retinitis pigmentosa (XLRP) is an inherited retinal disease that predominantly affects males, typically caused by mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene. The mutations, which affect approximately 1 in 25,000 males in the U.S., Europe and Australia, result in progressive photoreceptor loss over time and visual dysfunction beginning in childhood, eventually leading to blindness and impacting quality of life with no approved treatments.
About BTX-001
BTX-001 is an adeno-associated virus (AAV)-based gene therapy candidate being evaluated for the treatment of geographic atrophy (GA) secondary to dry age-related macular degeneration (AMD). The therapy is designed to target the clinically-validated complement pathway through delivery of a C5 inhibitor administered as a single intravitreal injection, combining the potential for sustained treatment effect with the convenience of in-office delivery.
BTX-001 is investigational and has not been approved by FDA for use.
About Geographic Atrophy
Geographic atrophy (GA) secondary to dry age-related macular degeneration (AMD) is a leading cause of irreversible vision loss in people over age 60 and represents an advanced stage of dry age-related macular degeneration. GA is characterized by loss of retinal cells including photoreceptors, retinal pigment epithelium and underlying choriocapillaris, forming areas of atrophy in the macula that expand over time. The condition leads to blind spots and progressive central vision loss, significantly impacting quality of life. GA affects over 5 million people globally and remains a significant unmet clinical need.
About Beacon Therapeutics
Beacon Therapeutics is a clinical-stage biotechnology company dedicated to saving and restoring sight for people living with rare and prevalent ocular diseases. The Company is harnessing the transformative power of gene therapy to deliver the most meaningful outcomes for severe ocular diseases. Beacon's pipeline currently targets devastating blinding retinal diseases such as X-linked retinitis pigmentosa (XLRP) and geographic atrophy (GA) secondary to dry age-related macular degeneration (AMD).
Beacon Therapeutics' investors include Advent Life Sciences, Forbion, Life Sciences at Goldman Sachs Alternatives, Oxford Science Enterprises, Retinal Degeneration Fund, Syncona Limited, and TCGX, among others. Learn more about Beacon Therapeutics at beacontx.com and follow on LinkedIn for more updates.
Contact:
info@beacontx.com
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