Opus Genetics publishes investor presentation on gene therapy pipeline for rare inherited retinal diseases

Opus Genetics, Inc.

Opus Genetics, Inc.

IRD

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  • Opus Genetics outlined a seven-asset AAV gene therapy portfolio in inherited retinal diseases, with two programs in clinical trials.
  • OPGx-BEST1 Phase 1/2 enrollment completed; final participant scheduled for dosing in May 2026; Cohort 1 3-month data expected September 2026.
  • Sentinel OPGx-BEST1 participant showed no ocular inflammation or dose-limiting toxicities through 3 months, with a 12-letter BCVA gain.
  • The same patient showed a 23% decrease in central subfield thickness in the treated eye over 3 months, indicating an early structural response.
  • OPGx-LCA5 program cited Phase 1/2 safety and efficacy in adults and children, with RMAT, Orphan Drug, Rare Pediatric Disease designations.


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