Opus Genetics , a Research Triangle Park, North Carolina-based clinical-stage biopharmaceutical company developing gene therapies for inherited retinal diseases (IRDs), presented clinical findings from its OPGx-BEST1 and OPGx-LCA5 programs at Retina in Rio 2026 in Rio de Janeiro, Brazil, the company announced on October 15, 2026.
“The data presented at Retina in Rio highlight meaningful progress across our gene therapy pipeline, with sustained improvements in visual function observed through 24 months in adults treated with OPGx-LCA5 and encouraging early evidence of improved retinal sensitivity in participants receiving OPGx-BEST1,” said George Magrath, M.D., CEO of Opus Genetics. “Importantly, these findings strengthen our understanding of how to measure treatment response in IRDs and support the continued advancement of both programs. Our OPGx-BEST1 Phase 1/2 trial has completed enrollment in the high-dose Cohort 2 with 11 participants, and with our OPGx-LCA5 Phase 3 trial progressing toward dosing initiation later this year, we remain focused on bringing potentially transformative gene therapies to patients living with rare inherited retinal diseases.”
OPGx-LCA5: Sustained Gains in Vision
OPGx-LCA5 is an investigational AAV8-based gene therapy that delivers a functional copy of the LCA5 gene to retinal photoreceptors. Mutations in LCA5 disrupt production of lebercilin, a protein critical for normal retinal function, causing Leber congenital amaurosis type 5 (LCA5), a rare disease that causes severe vision loss beginning in childhood.
Rodrigo Jorge, M.D., of Ribeirão Preto Medical School at the University of São Paulo, presented data from six participants treated at the low dose: three adults and three adolescents. Adults showed sustained improvements in visual acuity through 24 months. Pediatric participants showed restoration of cone-mediated vision, with sensitivity improvements reaching normal ranges through six months. Both groups showed improved light sensitivity compared with their untreated eyes. Participants also reported improvements in everyday life, including recognizing family members, navigating independently without a cane, and identifying objects and colors.
Based on these findings, Opus advanced OPGx-LCA5 into Phase 3 at the low dose without further dose escalation. The Phase 3 trial’s natural history and treatment cohorts are fully enrolled, with dosing expected to begin in the fourth quarter of 2026. Its primary efficacy endpoint will assess improvements in retinal sensitivity across 16 central retinal test locations.
OPGx-BEST1: Early Signals and Better Measurement
OPGx-BEST1 is an investigational gene therapy designed to restore bestrophin-1 function in patients with IRDs caused by mutations in the BEST1 gene, including Best vitelliform macular dystrophy and autosomal recessive bestrophinopathy. In the low-dose Cohort 1 of the Phase 1/2 BIRD-1 trial, five participants were treated, with follow-up ranging from three to six months. Among four who could complete retinal sensitivity testing, three (75%) showed improvements in the treated “transitional zone,” the area around damaged retinal tissue where light-sensitive cells remain and may respond to treatment. In responders, those gains were also associated with reductions in intraretinal fluid characteristic of BEST1-associated disease.
Additional analyses suggested that optical coherence tomography and other imaging may help identify the retinal regions most likely to respond to treatment. Natural history findings also showed that retinal sensitivity declines most noticeably in the transitional zone, even when whole-retina measurements appear stable. Together, these insights are shaping more targeted assessments for future development. High-dose Cohort 2 has completed enrollment with 11 participants.
About Opus Genetics
Opus Genetics is a clinical-stage biopharmaceutical company developing one-time gene therapies that address the underlying genetic causes of severe retinal disorders. Its pipeline includes seven AAV-based programs, led by OPGx-LCA5 and OPGx-BEST1, with additional candidates targeting RDH12, MERTK, RHO, CNGB1, and NMNAT1.
