Opus Genetics Inc. (IRD), a clinical-stage biopharmaceutical company, on Tuesday provided updates on various gene therapies under development for the treatment of inherited retinal diseases (IRDs). The data was presented at the Research and Development (R&D) Science Forum conducted by the company.
Pipeline Highlights:
OPGx-RDH12 is a gene therapy that delivers functioning enzymes to repair the RDH12 mutations responsible for Leber congenital amaurosis (LCA). The disease affects approximately 30,900 patients worldwide. Preclinical testing was completed, and clinical testing is set to begin in the fourth quarter of this year.
OPGx-MERTX targets MERTK mutations that cause a severe form of autosomal recessive retinitis pigmentosa, resulting in severe vision loss. The therapy was developed to restore function of retinal pigment epithelium metabolism and has shown evidence of retinal preservation in preclinical studies on animal models. Clinical testing is expected to begin in 2027.
OPGx-RHO addresses RHO mutations that cause misfolded or dysfunctional rhodopsin, resulting in progressive rod-cone dystrophy. This autosomal-dominant form of retinitis pigmentosa eventually leads to peripheral and central vision loss, affecting over 30,200 patients worldwide. In preclinical studies, OPGx-RHO retained rod-cone morphology in animal models, and clinical trials are planned for initiation in the second half of 2027.
OPGx-LCA5 targets genes producing a faulty lebercilin protein, responsible for photoreceptor functioning. In the Phase 1/2 trial, OPGx-LCA5 repaired and improved vision for adults over 24 months, and in pediatric patients over 6 months. The full-field stimulation test (FST) yielded durable vision improvement, and microperimetry results showed increased visual sensitivity in treated patients.
OPGx-BEST1 targets retinal pigment epithelium (RPE) cells that produce the bestrophin protein through BEST1 genes. BEST1 mutations (bestrophinopathy) cause RPE dysfunction and retinal degeneration and occur with a frequency of about 21,800 patients worldwide. The Phase 1/2 trial for OPGx-BEST1 completed dosing patients in May 2026.
Upcoming Events:
The company has begun recruitment in the Phase 3 trial for OPGx-LCA5, with dosing expected to begin in the fourth quarter of this year.
Three-month topline data from the Phase 1/2 trial for OPGx-BEST1 is anticipated in September 2026.
IRD closed Tuesday at $3.90, down 0.38%. In the pre-market, shares are trading at $3.81, down 2.19%.
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