Key Takeaways

  • Opus Genetics reported strong early clinical evidence that its OPGx-LCA5 gene therapy may restore cone-mediated daytime vision in children with severe inherited retinal disease, with improvements seen in visual acuity, light response, and daily functional vision
  • Early data from the company’s OPGx-BEST1 program showed meaningful visual improvements and retinal structural gains in treated patients
  • Preclinical findings from Opus Genetics’ RHO programs demonstrated preservation of retinal structure and function across animal models

Opus Genetics unveiled new clinical and preclinical findings at the Association for Research in Vision and Ophthalmology (ARVO) annual meeting, highlighting advances in therapies aimed at restoring vision and preventing blindness.

The company presented data from several programs, including OPGx-LCA5, OPGx-BEST1, and OPGx-RHO, providing evidence that gene therapy may be capable of restoring functional vision in patients with severe, early-onset retinal degeneration.

Pediatric LCA5 Study Shows Robust Vision Recovery

The centerpiece of the presentation was 6-month data from Opus Genetics’ ongoing phase 1/2/3 clinical trial evaluating OPGx-LCA5 in pediatric patients with Leber congenital amaurosis type 5 (LCA5).

Presented by principal investigator Tomas S. Aleman, MD, the findings demonstrated that children receiving a single subretinal injection of OPGx-LCA5 experienced substantial improvements in cone-mediated daytime vision despite having advanced disease at baseline.

According to the company, treated patients achieved more than 30-fold improvements in cone sensitivity, along with measurable gains in visual acuity compared to untreated control eyes. Researchers also reported improvements in pupillary light responses, orientation and mobility testing, and patient-reported daily visual function.

The therapy was reported to be well tolerated, with no dose-limiting toxicities and only mild, expected adverse events that resolved without complication.

“These results indicate that even in severe, early-onset disease, there remains a possible window of opportunity to restore cone function and meaningfully improve vision,” Dr. Aleman said during the presentation.

BEST1 Program Demonstrates Early Clinical Progress

Opus Genetics also shared preliminary results from an adult participant enrolled in its phase 1b/2a study of OPGx-BEST1, a gene therapy targeting retinal diseases caused by BEST1 mutations, including autosomal recessive bestrophinopathy (ARB) and Best vitelliform macular dystrophy (BVMD).

At 3 months post-treatment, the patient experienced up to a 12-letter improvement in visual acuity, alongside a roughly 23% reduction in central retinal thickness—a sign of improved retinal structure. No ocular inflammation or treatment-related adverse events were observed.

The company noted that Cohort 1 enrollment in the ongoing study has been completed, with topline 3-month data expected in September 2026.

In parallel, Opus presented new laboratory assay systems designed to validate gene expression and protein activity for OPGx-BEST1 manufacturing, a step aimed at supporting scalable production and regulatory readiness.

RHO Program Advances Toward Clinical Translation

Additional preclinical presentations focused on Opus Genetics’ mutation-independent approach to treating autosomal-dominant retinitis pigmentosa (RHO-adRP), a genetically diverse retinal disease.

In canine studies evaluating GMP-grade OPGx-RHO delivered via subretinal injection, researchers demonstrated preservation of retinal structure and function while establishing a no-observed-adverse-effect level (NOAEL) to guide future clinical dosing.

Separate swine-model studies evaluating a knockdown-and-replacement strategy showed restoration of rod-driven visual responses, maintenance of cone function, and preservation of photoreceptor structure over time. Researchers also identified a minimal effective dose and observed evidence suggesting interocular vector transfer, which may provide insight into systemic distribution dynamics.

The company stated that presentation materials and posters from ARVO 2026 will be made available through the Opus Genetics website.