Opus Genetics Announces 1-Month Clinical Data from Pediatric Patient in Phase 1/2 Trial of OPGx-LCA5 Gene Therapy in Inherited Retinal Diseases
Opus Genetics announced 1-month clinical data from the first pediatric patient treated with its investigational gene therapy, OPGx-LCA5, in an ongoing phase 1/2 open-label trial. The therapy is aimed at treating LCA5-related inherited retinal disease.
The patient, aged 16 at the time of consent, received a single subretinal injection of OPGx-LCA5. One month post-treatment, clinically meaningful improvement in vision was observed, with no drug-related adverse events reported to date.
“The preliminary results observed in our pediatric patient are encouraging and are consistent with the improvements we previously observed in adults,” Dr. Tomas Aleman, Principal Investigator at Scheie Eye Institute, Perelman School of Medicine, said in a company news release. “She noticed that objects were significantly brighter and was able to distinguish letters and navigate with an independence she had never had before—after only 1 month of treatment.”
“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. “Given the progressive nature of this disease, early intervention in pediatric patients may be especially beneficial.”
Promising Early Results and Ongoing Study
OPGx-LCA5 is currently being evaluated in a phase 1/2 clinical trial involving both adult and pediatric patients with inherited retinal degeneration caused by biallelic mutations in the LCA5 gene. Pediatric patient enrollment began in February 2025, and initial data from this cohort are expected in Q3 2025.
Adult patient data from the same study previously demonstrated visual improvement at 6 months in all three patients treated. New 1-year follow-up data, to be presented at the Association for Research in Vision and Ophthalmology (ARVO) 2025 Annual Meeting on May 4, shows sustained functional and subjective improvements, further validating the therapy’s long-term potential.
Following a recent FDA Type D meeting, Opus Genetics proposed a single-arm, adaptive pivotal study that may enroll as few as 19 patients. The study would utilize the Multi-Luminance Orientation and Mobility Test (MLoMT) as its primary endpoint—a functional assessment designed to measure real-world vision and mobility under various lighting conditions. This innovative test is modeled after the Multi-Luminance Mobility Test (MLMT), which previously supported an FDA approval for another gene therapy.
The FDA provided constructive feedback on the trial’s proposed statistical analysis plan, as well as the chemistry, manufacturing, and controls (CMC) approach. Opus plans to submit additional information in response and anticipates launching the pivotal trial in Q1 2026.