Beacon Therapeutics’ gene therapy has shown a 12-month benefit in a Phase II study in patients with X-linked retinitis pigmentosa (XLRP), a rare type of inherited progressive eye disease.
In the Phase II DAWN trial (NCT06275620), laruparetigene zovaparvovec (laru-zova) was generally well-tolerated over 12 months, with patients benefitting from sustained improvements across several key measures of visual function, including low luminance visual acuity (LLVA) and mean macular sensitivity as measured by microperimetry, in line with previous nine-month data.
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In those who received the high-dose gene therapy, 50% of patients achieved at least two-line (10+ ETDRS letters) improvement from baseline, with 25% of patients who received a high dose achieving at least three-line (15+ ETDRS letters) improvement from baseline. In the low dose, 67% of achieved at least two-line improvement from baseline.
Data also showed sustained improvement in microperimetry mean sensitivity as compared to baseline.
The data was released during a presentation at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting held 3-7 May 2026 in Denver, Colorado.
Daniel Chung, chief medical officer of Beacon Therapeutics, said: “As we continue to build one of the most significant bodies of evidence for a gene therapy in ocular disease, these findings further support the ongoing clinical development of laru-zova and reinforce its potential as a meaningful treatment option for people living with XLRP.”
XLRP is an inherited retinal disease that leads to progressive vision loss and legal blindness, with no available treatment options. It is caused by mutations to the retinitis pigmentosa GTPase regulator (RPGR) gene.
Laru-zova is a gene therapy designed 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.
Beacon is also evaluating laru-zova in its ongoing pivotal VISTA trial (NCT04850118) in XLRP. Beacon completed enrolment of this trial in June 2025, with topline data expected in H2 2026. Dosing has also been initiated in the LANDSCAPE trial (NCT07174726), which is evaluating the gene therapy in male participants aged between 12 and 50 years who have a confirmed XLRP diagnosis in the US sites.
A GlobalData report predicts cases of RP will increase across the 16 major pharmaceutical markets (16MM: the US, France, Germany, Italy, Spain, the UK, Japan, Australia, Brazil, Canada, China, India, Mexico, Russia, South Africa, and South Korea), from 1.04 million in 2024 to 1.08 million by 2029. GlobalData is the parent company of Clinical Trials Arena.
Cell & Gene therapy coverage on Clinical Trials Arena is supported by Cytiva.
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