Inherited retinal diseases encompass more than 350 genetic mutations and represent a significant unmet clinical need. Opus Genetics’ portfolio approach prioritises indications with well-characterised biology, validated delivery methods, and measurable functional endpoints, enabling streamlined development timelines and capital efficiency. Two of the company’s seven gene therapy assets are already in clinical trials, with additional programs expected to enter the clinic. The company recently gave a presentation outlining its strategy at the J. P.Morgan Healthcare Conference 2026 in San Francisco.
The most advanced program, OPGx-LCA5, targets Leber congenital amaurosis type 5, a rare and severe retinal degeneration that presents in early childhood. Despite profound visual impairment, LCA5 patients often retain viable photoreceptors into adulthood, supporting the rationale for gene replacement. Phase I/II data demonstrated improvements across multiple functional endpoints, including visual acuity, full-field stimulus testing, and mobility assessments, with effects observed as early as one month and sustained for up to 18 months in adult participants. The therapy was well tolerated, with no ocular serious adverse events reported.
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Graysen Vigneux, Medical Analyst at GlobalData, commented: “Opus Genetics’ portfolio strategy balances long-term gene therapy value drivers with near-term commercial upside from partnered assets. The ophthalmic solution program adds diversification and potential revenue milestones, enhancing the company’s overall risk-adjusted outlook.”
OPGx-LCA5 has received Rare Pediatric Disease, Orphan Drug, and Regenerative Medicine Advanced Therapy designations from the FDA. Opus Genetics has completed a Type B regulatory meeting and is enrolling patients in preparation for a planned adaptive Phase 3 trial, with dosing expected to begin in the second half of 2026.
The company’s second lead program, OPGx-BEST1, is designed to treat bestrophinopathies caused by mutations in the BEST1 gene, which affect approximately 9,000 patients in the U.S. BEST1 disorders are characterised by slow disease progression and long-term photoreceptor viability, providing a broad therapeutic window. The Phase 1/2 BIRD-1 trial initiated dosing in late 2025, with early safety data supporting continued enrollment and initial efficacy data expected in early 2026.
In addition to gene therapy, Opus Genetics benefits from a partnered commercial asset, phentolamine ophthalmic solution 0.75%, which is approved for pharmacologically induced mydriasis and under regulatory review for presbyopia. A Phase III program is also underway for dim-light visual disturbances following keratorefractive surgery, offering potential near-term milestones and royalty streams.
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By GlobalDataWith multiple data readouts anticipated in 2026, a cash runway extending into 2027, and a pipeline addressing a potential multi-billion-dollar U.S. market, Opus Genetics is positioned for a period of elevated clinical and investor interest as rare ophthalmic gene therapy continues to mature.
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