Beacon Therapeutics Initiates LANDSCAPE Trial for X-Linked Retinitis Pigmentosa Gene Therapy
核心洞察
Beacon Therapeutics (搜索) has treated the first patient in its LANDSCAPE trial evaluating bilateral administration of laru-zova gene therapy for X-linked retinitis pigmentosa (搜索) (XLRP (搜索)).
The open-label study will assess safety of the therapy when administered in both eyes and examine visual function changes in males aged 12-50 with confirmed XLRP (搜索) diagnosis.
The trial builds on positive interim data from Phase II DAWN and SKYLINE studies showing sustained improvements in key visual function measures including low luminance visual acuity and microperimetry.
Beacon Therapeutics (搜索) has treated the first patient in its open-label LANDSCAPE trial evaluating laru-zova (laruparetigene zovaparvovec), a potential gene therapy for X-linked retinitis pigmentosa (搜索) (XLRP (搜索)). The milestone marks another step forward for the clinical-stage biotechnology company's efforts to address this devastating inherited retinal disease that affects approximately 10,000 patients in the United States.
Trial Design and Objectives
The LANDSCAPE trial (NCT07174726) is designed to evaluate the safety of laru-zova when administered bilaterally via subretinal injection. The study will also assess changes in visual function following treatment in both eyes. The trial is enrolling male participants aged between 12 and 50 years who have a confirmed XLRP (搜索) diagnosis across sites in the United States.
"Following the completion of enrollment in our registrational VISTA trial of laru-zova in XLRP (搜索) earlier this year, the initiation of our LANDSCAPE trial, which will access the safety of bilateral dosing within a short period of time, is another important step toward offering patients a potential new treatment for this devastating inherited retinal disease," said Daniel Chung, Chief Medical Officer of Beacon Therapeutics (搜索).
Building on Previous Clinical Success
The LANDSCAPE trial builds upon encouraging interim data from the Phase II DAWN and SKYLINE studies presented at EURETINA 2025. These trials demonstrated sustained improvements across several key measures of visual function, including low luminance visual acuity and microperimetry, in patients treated with laru-zova.
Safety data from the previous studies showed that laru-zova was generally well-tolerated by participants in the SKYLINE study through month 36 and by those in the DAWN trial at nine months or later.
Understanding XLRP
X-linked retinitis pigmentosa (搜索) is an inherited retinal condition that primarily impacts males and is most often caused by mutations in the retinitis pigmentosa GTPase regulator (搜索) (RPGR (搜索)) gene. The disease leads to progressive vision loss and legal blindness, with no available treatment options. XLRP (搜索) affects about 10% of the 100,000 retinitis pigmentosa (搜索) patients in the United States, with mutations resulting in progressive photoreceptor loss over time and visual dysfunction beginning in childhood.
Gene Therapy Approach
Laru-zova is designed to restore the natural function of both rods and cones in XLRP (搜索) by delivering a functional copy of the RPGRORF15 (搜索) gene designed to produce the full-length protein. The therapy represents a potential best-in-class gene therapy approach to addressing the underlying genetic cause of the disease.
Regulatory Recognition and Future Milestones
The therapy has received several regulatory designations recognizing its potential clinical value. These include fast track and regenerative medicine advanced therapy (RMAT) status from the US Food and Drug Administration (FDA), European Medicines Agency's (EMA) priority medicines (PRIME) designation, and the UK Medicines and Healthcare products Regulatory Agency's innovative licensing and access pathway (ILAP). Laru-zova also holds orphan drug status from both the FDA and EMA.
Beacon Therapeutics (搜索) completed enrollment in its pivotal VISTA trial of laru-zova in July 2025, with topline data anticipated in the second half of 2026.
"This new study builds upon our extensive clinical experience across multiple trials and will contribute to one of the most significant data packages for a gene therapy in ocular diseases," Chung noted.