LONDON and CAMBRIDGE, Mass., Aug. 21, 2025 (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 announces upcoming presentations on the preliminary 9+-month data from the Phase 2 DAWN trial and 36-month results of the randomized, controlled Phase 2 SKYLINE trial of the Company's lead program, laru-zova (laruparetigene zovaparvovec), in patients with X-linked retinitis pigmentosa (XLRP) at the EURETINA 2025 Conference being held September 4-7, 2025 in Paris, France.
Presentation details:
Presentation title: Subretinal gene therapy laru-zova for X-linked retinitis pigmentosa (XLRP): Phase 2 DAWN trial, preliminary month 9+ results
Date: Thursday, September 4, 2025
Presenter: Rajiv Anand, M.D., FRCS, FRCOphth, Texas Retina Associates and Retina Foundation of the Southwest
Presentation title: Subretinal laru-zova gene therapy for XLRP: 36-month results of the randomized, controlled, multicenter Phase 2 SKYLINE trial
Date: Thursday, September 4, 2025
Presenter: Paul Yang, M.D., Ph.D. Chief, Paul H. Casey Ophthalmic Genetics Division, Casey Eye Institute, OHSU
Contact:
info@beacontx.com
Media & Investors:
beacon@icrhealthcare.com
About laru-zova
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. 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 SKYLINE and DAWN Studies
SKYLINE is an ongoing, fully enrolled, Phase 2, randomized, controlled study evaluating the safety, efficacy and tolerability of laru-zova in 14 male patients with XLRP caused by mutations in the RPGR gene. The study's primary endpoint is the proportion of response by microperimetry between the study and fellow eye at Month 12. NCT06333249.
DAWN (NCT06275620) is an ongoing, fully enrolled, 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 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.
About XLRP
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 Beacon Therapeutics
Beacon Therapeutics is a clinical-stage biotechnology company with a mission to save and restore the vision of patients with blinding retinal diseases. The Company has an established scientific foundation that includes a late-stage clinical development candidate to treat XLRP and two preclinical programs targeting dry age-related macular degeneration (AMD) and an inherited cone-rod dystrophy (CRD).
Beacon Therapeutics' investors include Syncona Limited, Forbion, Oxford Science Enterprises, TCGX and Advent Life Sciences, among others. Learn more about Beacon Therapeutics at beacontx.com.
