Progress in gene therapy for inherited retinal diseases continues to build momentum. This week, Beacon Therapeutics announced that the first patient has been treated in its LANDSCAPE clinical trial, a new study evaluating bilateral dosing of an investigational gene therapy for X-linked retinitis pigmentosa (XLRP) — one of the most severe forms of RP.
While this trial is not specific to Bardet-Biedl Syndrome (BBS), it represents another meaningful step forward for the broader retinal gene therapy field — and reinforces the pace at which vision-saving science is advancing.
What Is the LANDSCAPE Trial?
The LANDSCAPE trial is an open-label clinical study designed to evaluate the safety of administering gene therapy to both eyes in people with XLRP. Most early retinal gene therapy trials treat only one eye at a time, so studying bilateral dosing is an important milestone.
Key details of the study include:
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Evaluation of laru-zova, an investigational gene therapy
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Subretinal delivery to both eyes
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Enrollment of males ages 12–50 with confirmed XLRP
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Trial sites across the United States
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Focus on safety, with additional evaluation of visual function
Treating both eyes safely could be a critical step toward making gene therapy more practical and impactful for patients with progressive vision loss.
Understanding X-Linked Retinitis Pigmentosa (XLRP)
XLRP is a genetic form of retinitis pigmentosa most often caused by mutations in the RPGR gene. It typically begins in childhood and progresses rapidly, often leading to legal blindness at a young age. There are currently no approved treatments for XLRP.
Although XLRP represents only about 10% of all RP cases, it is among the most aggressive forms — making it a priority target for gene therapy development.
How Laru-zova Works
Laru-zova (laruparetigene zovaparvovec) is designed to deliver a functional copy of the RPGR gene to retinal cells. Its goal is to restore or preserve the function of both rods and cones, the photoreceptors responsible for vision in low light and daylight.
What makes this therapy especially notable is its design to produce a full-length RPGR protein, a technical challenge that has historically complicated XLRP treatment development.
Building on Encouraging Earlier Results
The LANDSCAPE trial builds on earlier Phase 2 studies — including the DAWN and SKYLINE trials — which showed:
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Sustained improvements in certain measures of visual function
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Positive signals in low-luminance visual acuity and microperimetry
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A generally favorable safety profile over extended follow-up
Beacon has also completed enrollment in its pivotal VISTA trial, with topline results expected in the second half of 2026.
Together, these studies form one of the most robust clinical data packages to date for a retinal gene therapy program.
Why Bilateral Treatment Matters
For patients with inherited retinal diseases, vision loss rarely affects just one eye. Studying bilateral dosing helps answer critical questions, such as:
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Can gene therapy be safely delivered to both eyes?
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Does treating both eyes improve functional outcomes?
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What are the risks and benefits of closer dosing intervals?
If proven safe, bilateral treatment could significantly improve quality of life and accelerate real-world adoption of gene therapies.
What This Means for the Broader Rare Disease Community
Every successful step forward in retinal gene therapy — even for a disease outside BBS — strengthens the entire field. These advances help:
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Refine delivery techniques
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Improve regulatory confidence
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Validate trial endpoints
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Attract further investment into rare disease research
For families facing progressive vision loss, including those affected by BBS-related retinal degeneration, this progress matters deeply. It signals that gene therapy is not theoretical — it is happening now, and the pathway to treatment is becoming clearer.
Looking Ahead
Laru-zova remains investigational and is not yet FDA-approved. But treating the first patient in the LANDSCAPE trial marks an important moment — one that reflects the speed, collaboration, and urgency now driving gene therapy for inherited retinal diseases.
At A Race Against Blindness, we continue to watch these developments closely, advocate for faster progress, and support the work needed to bring vision-saving treatments to families before it’s too late.
