OPGx-BEST1 showed improved retinal sensitivity in 75% of evaluable participants in the initial low-dose Cohort 1 of the Company’s Phase 1/2 clinical trial, supporting further clinical development
New BEST1 data analyses link retinal structure to visual function, enabling focus on regions most likely to respond to treatment
OPGx-LCA5 demonstrated sustained improvements in visual acuity and light sensitivity through 24 months in adults, with encouraging results in pediatric participants through six months in the Company’s Phase 1/2/3 clinical trial
Phase 3 clinical trial dosing for OPGx-LCA5 expected in the fourth quarter of 2026
RESEARCH TRIANGLE PARK, N.C., Oct. 15, 2026 (GLOBE NEWSWIRE) -- Opus Genetics, Inc. (NASDAQ:IRD) ("Opus Genetics" or the "Company"), a clinical-stage biopharmaceutical company developing gene therapies to restore vision and prevent blindness in patients with inherited retinal diseases (IRDs), presented clinical findings from its OPGx-BEST1 and OPGx-LCA5 gene therapy programs at Retina in Rio 2026 in Rio de Janeiro, Brazil.
The Company presented findings from the low-dose Cohort 1 of its Phase 1/2 BIRD-1 trial of OPGx-BEST1, which showed improved retinal sensitivity in three of four evaluable participants (75%). Additional analyses identified retinal structural and imaging characteristics associated with visual function and treatment response, providing insights that may help guide future clinical development. Additional findings from the ongoing Phase 1/2 trial of OPGx-BEST1 demonstrated encouraging improvements in retinal sensitivity in participants with BEST1-associated retinal diseases, along with new insights into imaging markers that may help predict treatment response.
The Company also presented findings from the Phase 1/2 clinical trial of OPGx-LCA5, which demonstrated sustained improvements in visual function among adults and children treated with OPGx-LCA5 for Leber congenital amaurosis type 5 (LCA5), a rare inherited retinal disease that causes severe vision loss beginning in childhood. In the OPGx-LCA5 Phase 1/2 study, improvements in visual acuity and light sensitivity were observed in three adult participants through 24 months and three pediatric participants through six months. Participants also reported improvements in everyday activities, including navigating independently, recognizing family members, and identifying objects and seeing colors.