Opus Genetics says OPGx-LCA5 trial restores cone-mediated vision in pediatric LCA5 patients

Opus Genetics, Inc.

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Opus Genetics released new clinical results already presented at ARVO 2026 from ongoing Phase 1/2/3 study of OPGx-LCA5 in pediatric Leber congenital amaurosis type 5, showing patients regained meaningful daytime visual function following a single treatment.
Separate early clinical findings already presented at ARVO from Phase 1b/2a OPGx-BEST1 study in an adult with BEST1-related disease indicated improved vision with signs of better retinal structure, supporting plans to broaden development in a larger inherited retinal disease population.
Enrollment completed in Cohort 1 of Phase 1/2 OPGx-BEST1 study, with 3-month topline data scheduled for September 2026.

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