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Opus Genetics Reports Positive Gene Therapy Data, Plans Phase 3 Dosing

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Opus Genetics, Inc. (IRD) released positive early clinical trial results for two experimental eye treatments. In a Phase 1/2 study for OPGx-BEST1, three out of four evaluable low-dose participants, or 75%, showed improved retinal sensitivity in the targeted area. Meanwhile, a Phase 1/2 study for OPGx-LCA5 demonstrated sustained improvements in light sensitivity and visual acuity over 24 months in three adult patients, alongside six-month cone-mediated sensitivity gains in three pediatric participants.

The trial findings offer evidence of durable vision improvements and provide functional guidance for future treatment. Structural analyses linking retinal tissue layers to visual functionality helped refine target regions and endpoints for the BEST1 therapy. These clinical results support advancing OPGx-LCA5 into late-stage testing to treat rare genetic retinal disorders.

Looking ahead, Opus Genetics plans to initiate Phase 3 patient dosing for OPGx-LCA5 in the fourth quarter of 2026. The upcoming Phase 3 trial will evaluate central retinal sensitivity across 16 specific locations as its main measure of effectiveness. Shares of Opus Genetics, Inc. fell 1.22% to $4.05 in recent trading.

Key points

  • Three of four evaluable low-dose participants in the OPGx-BEST1 trial showed improved retinal sensitivity.
  • Adults receiving OPGx-LCA5 experienced visual gains lasting through 24 months of observation.
  • Three pediatric patients in the OPGx-LCA5 trial showed cone-mediated sensitivity gains through six months.
  • Opus Genetics plans to begin Phase 3 trial dosing for OPGx-LCA5 in the fourth quarter of 2026.

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Questions and answers

What were the results of the Opus Genetics OPGx-BEST1 trial?

In the Phase 1/2 low-dose trial cohort, 75% of evaluable participants (3 out of 4) showed improved retinal sensitivity in the treated transitional zone.

When will Phase 3 dosing begin for OPGx-LCA5?

Opus Genetics expects to start dosing participants in the Phase 3 clinical trial of OPGx-LCA5 during the fourth quarter of 2026.

How long did vision improvements last in adult OPGx-LCA5 patients?

Adult participants in the Phase 1/2 trial experienced sustained gains in visual acuity and light sensitivity through 24 months.