Opus Genetics Shares Early Pediatric Data from Gene Therapy Trial for Inherited Retinal Disease

In the ongoing effort to address rare retinal conditions, new data from Opus Genetics marks a significant early milestone. The company has announced the first clinical results from a pediatric patient enrolled in its phase 1/2 trial of OPGx-LCA5, an investigational gene therapy targeting LCA5-related Leber congenital amaurosis (LCA)—a severe inherited retinal disease that typically causes profound vision loss from infancy.

The patient, a 16-year-old girl, received a single subretinal injection of OPGx-LCA5. One month after treatment, she experienced clinically meaningful improvements in vision, with no treatment-related adverse events reported. According to Dr. Tomas Aleman, principal investigator at the Scheie Eye Institute at the University of Pennsylvania, the patient reported that objects appeared significantly brighter and that she was able to distinguish letters and navigate independently—capabilities she had not previously had.

These early pediatric results are consistent with previously observed improvements in adult patients. “We believe these findings provide further evidence supporting the potential of OPGx-LCA5 to restore vision in patients with LCA5 mutations,” said Dr. George Magrath, CEO of Opus Genetics. Because this condition worsens over time, intervening early in pediatric cases could help protect or regain vision before more damage occurs.

The therapy is being evaluated in an open-label phase 1/2 clinical trial that includes both adult and pediatric participants with biallelic mutations in the LCA5 gene. Pediatric enrollment began in February 2025, and broader cohort data are expected later this year.

Among adults, early signs of efficacy have been encouraging. The first three participants showed improved vision at six months post-treatment, and new one-year follow-up data—set to be presented at the Association for Research in Vision and Ophthalmology (ARVO) 2025 Annual Meeting—suggest that those gains have been maintained over time.

Regulatory planning is also moving forward. Following a Type D meeting with the FDA, Opus proposed a single-arm, adaptive pivotal study that may enroll as few as 19 patients. The trial would use the Multi-Luminance Orientation and Mobility Test (MLoMT) as its primary endpoint, a real-world functional measure modeled after the Multi-Luminance Mobility Test (MLMT) that supported the approval of a prior gene therapy for retinal disease.

The FDA has provided feedback on the proposed statistical analysis plan as well as on the manufacturing and quality control strategy. Opus plans to submit additional documentation and is targeting the launch of the pivotal trial in early 2026.

While these are preliminary findings from a single pediatric case, they may represent a meaningful step forward in the treatment of LCA5-associated vision loss. For this patient—and potentially others—gene therapy is offering a new lens through which the future can be seen, quite literally, in a different light.

Source

“Opus Genetics, Inc.” 2025. Opus Genetics, Inc. April 8, 2025. https://ir.opusgtx.com/press-releases/detail/483/opus-genetics-announces-one-month-clinical-data-from-pediatric-patient-in-phase-12-trial-of-opgx-lca5-gene-therapy-in-inherited-retinal-diseases.

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