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RDH12 mutations cause a severe form of Leber congenital amaurosis (LCA), which leads to early, rapid vision loss in infancy or childhood, often
resulting in legal blindness before the third decade of life.
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Estimated global prevalence is 30,900 patients, including 2,500 patients in the U.S. and 17,500 patients in the Middle East / North Africa.*
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OPGx-RDH12 is designed to restore a key component of the visual cycle.
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In preclinical studies, OPGx-RDH12 delivered a functional RDH12 enzyme in vivo and restored structure and function in a small animal model.
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This program is expected to initiate clinical testing in the U.S. in the fourth quarter of 2026. Funding is supported by the RDH12 Alliance.
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MERTK mutations cause a severe form of autosomal recessive retinitis pigmentosa, which has an early-onset (childhood or adolescence) and
results in rapid vision loss.
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Estimated global prevalence is 21,960 patients, including 2,600 patients in the U.S. and 14,300 patients in the Middle East / North Africa.*
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OPGx-MERTK is designed to restore critical retinal pigment epithelium metabolic functions.
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In preclinical studies, OPGx-MERTK provided evidence of retinal preservation of structure/ function and preserved outer nuclear layer thickness
in a small animal model of retinal degeneration.
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This program is expected to initiate clinical testing at the Cleveland Clinic Abu Dhabi in the first quarter of 2027. Funding is supported by a
consortium led by Abu Dhabi’s Healthcare Research and Innovation Fund.
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RHO mutations cause autosomal-dominant retinitis pigmentosa, a highly variable, often slowly progressive rod-cone dystrophy characterized by
misfolded or dysfunctional rhodopsin that drives photoreceptor stress and progressive peripheral field loss followed by central vision decline.
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Estimated global prevalence is 30,200 patients, including 8,800 patients in the U.S.*
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OPGx-RHO is designed to “silence and replace” in autosomal dominant RHO.
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In preclinical studies, OPGx-MERTK maintained rod morphology in the retinas in two large animal models.
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This program is expected to initiate clinical testing globally in the second half of 2027.
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LCA5 mutations cause a very early onset, severe disease typically presenting in infancy or early childhood.
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Estimated global prevalence is 3,240 patients, including 170 patients in the U.S.*
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OPGx-LCA5 is designed to restore the lack of functioning lebercilin, a key protein of the visual cycle, which severely impairs photoreceptor
function.
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In the ongoing Phase 1/2 clinical trial, visual acuity was improved and maintained in the adult cohort over 24 months and improved in the
pediatric cohort over 6 months. In addition, the full-field stimulus test (FST) demonstrated durable vision improvement and microperimetry data provided evidence of increased sensitivity and movement of fixation toward the fovea.
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Recruitment is ongoing in the run-in period for the pivotal Phase 3 trial. In parallel, the clinical and commercial batch of drug product is being
manufactured. Dosing with OPGx-LCA5 is expected to start in the fourth quarter of 2026.
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BEST1 mutations disrupt cellular ion and fluid homeostasis resulting in electrophysiological abnormalities, RPE dysfunction, and retinal
degeneration, with a range of onset from childhood to adulthood and a slow rate of progression.
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Estimated global prevalence is 21,800 patients, including 8,400 patients in the U.S. comprised of approximately 8,000 best vitelliform macular
dystrophy (BVMD) and approximately 400 autosomal recessive bestrophinopathy (ARB).*
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OPGx-BEST1 is designed to target retinal pigment epithelium (RPE) cells and restore bestrophin function.
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In the ongoing Phase 1/2 clinical trial, three BVMD and two ARB participants were enrolled in the first cohort, with dosing completed in May
2026.
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Three-month topline data from Cohort 1 is expected in September 2026.
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IRD patient prevalence across select global markets provides significant commercial market opportunity across Opus’ pipeline.
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IRD prevalence may be higher than current estimates in some mutations. For example, patients may not undergo genetic testing when clinicians
diagnose BEST1 disease based on the presence of a characteristic vitelliform (“egg-yolk”) lesion beneath the macula.
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