Opus Reports Vision Improvements for Three Pediatric Patients in Phase 1/2 Clinical Trial for LCA5 Gene Therapy

Opus Genetics, a biopharmaceutical company developing gene therapies for inherited retinal diseases (IRDs), has reported positive data for three pediatric patients (16-17 years of age) participating in its Phase 1/2 clinical trial for OPGx-LCA5, a gene therapy for people with Leber congenital amaurosis 5 (LCA5). The company also reported durable vision improvements out to 18 months for the three adult patients in the clinical trial. The company plans to meet with the US Food & Drug Administration (FDA) in the fourth quarter of 2025 to discuss the results and next steps.

Though LCA5 patients have severe vision loss at birth, they have surviving retinal structure, which researchers believe can be harnessed for improved vision using gene therapy.

The company reported the following data on visual acuity improvement for the three pediatric patients in the trial:

• An average visual acuity improvement of 0.3 logMAR was observed for the three pediatric patients. A 0.3 logMAR improvement is considered clinically significant.
• Participant 01-05 had a baseline visual acuity of 2.2 logMAR with an improvement of 0.5 logMAR reported at one month. On the Snellen eye chart, which is often used in a doctor’s office, that’s an improvement from approximately 20/3170 to 20/1002.
• Participant 01-06 had a baseline visual acuity of 0.96 logMAR with an improvement of 0.2 logMAR reported at three months. That’s an improvement from approximately 20/182 to 20/115.
• Participant 01-07 had a baseline visual acuity of 2.3 logMAR with an improvement of 0.7 logMAR reported at one month, which was maintained through three months. That’s an improvement from 20/3990 to 20/796.

All three participants had improvement in cone photoreceptor sensitivity as measured by full-field sensitivity (FST) and object recognition as measured by a Multi-Luminance Orientation and Mobility Test (a virtual reality system).

The LCA5 gene therapy clinical trial is the first launched by Opus, a company founded in 2021 by the Foundation’s RD Fund, its venture philanthropy fund for supporting the development of emerging retinal disease therapies in or nearing early-stage clinical trials.  Opus was acquired by Ocuphire Pharma in 2024, retained the Opus name, and is trading on the Nasdaq under the ticker symbol “IRD.”

The LCA5 gene therapy uses a human-engineered adeno-associated virus (AAV) to deliver healthy copies of the LCA5 gene to the retinas of patients, augmenting the mutated copies leading vision loss. The therapy is administered through a one-time injection underneath the retina. Researchers believe IRD gene therapies may be effective for many years, perhaps for the life of the patient.