June 16, 2026 —
Opus Genetics highlighted progress across its expanding AAV gene therapy pipeline for inherited retinal diseases, or IRDs, during a Research and Development Science Forum. The company said it is advancing five programs, with four clinical data readouts expected in 2027 and three additional programs expected to enter clinical testing over the next 12 to 18 months.
Opus is developing gene therapies designed to restore vision and prevent blindness in patients with severe retinal diseases caused by defined genetic mutations. The company’s pipeline includes programs targeting RDH12, MERTK, RHO, LCA5, and BEST1, spanning several forms of Leber congenital amaurosis, retinitis pigmentosa, and bestrophin-associated retinal disorders.
The company’s OPGx-RDH12 program is designed to treat RDH12-associated Leber congenital amaurosis, a severe inherited retinal disease that often causes rapid vision loss beginning in infancy or childhood. The therapy is intended to restore a key component of the visual cycle. In preclinical studies, OPGx-RDH12 delivered functional RDH12 enzyme in vivo and restored retinal structure and function in a small animal model. Clinical testing in the U.S. is expected to begin in the fourth quarter of 2026.
Opus is also advancing OPGx-MERTK for MERTK-associated autosomal recessive retinitis pigmentosa, an early-onset disease that can lead to rapid vision loss. The therapy is designed to restore critical retinal pigment epithelium metabolic functions. Preclinical studies showed evidence of retinal preservation, including maintenance of structure and function and preserved outer nuclear layer thickness in a small animal model of retinal degeneration. Clinical testing is expected to begin at Cleveland Clinic Abu Dhabi in the first quarter of 2027.
The company’s OPGx-RHO program targets autosomal dominant retinitis pigmentosa caused by RHO mutations. Because RHO mutations often lead to misfolded or dysfunctional rhodopsin that drives photoreceptor stress and degeneration, OPGx-RHO is designed as a “silence and replace” strategy. Clinical testing is expected to begin globally in the second half of 2027.
Opus’ more advanced OPGx-LCA5 program is designed to restore lebercilin function in patients with LCA5-associated Leber congenital amaurosis, a very early-onset retinal disease that severely impairs photoreceptor function. In the ongoing Phase 1/2 trial, visual acuity improved and was maintained in the adult cohort over 24 months, while improvement was also observed in the pediatric cohort over six months. Additional measures, including full-field stimulus testing and microperimetry, showed evidence of durable vision improvement, increased sensitivity, and movement of fixation toward the fovea. Recruitment is ongoing in the run-in period for the pivotal Phase 3 trial, with dosing expected to begin in the fourth quarter of 2026.
The company’s OPGx-BEST1 program is designed to target retinal pigment epithelium cells and restore bestrophin function in patients with BEST1-associated retinal diseases, including best vitelliform macular dystrophy and autosomal recessive bestrophinopathy. In the ongoing Phase 1/2 trial, three BVMD participants and two ARB participants were enrolled in the first cohort, with dosing completed in May 2026. Three-month topline data from Cohort 1 are expected in September 2026.
Opus said its cash runway into 2029 is expected to fund multiple clinical inflection points, potential product approvals, and opportunities for Priority Review Vouchers. The company also emphasized the commercial potential of its pipeline, citing meaningful patient prevalence across select global markets and possible underdiagnosis in certain IRD populations due to limited genetic testing.
