Clinical Read2026-09-09

OPGx-BEST1 - (BIRD-1)

Opus Genetics Inc.IRD
Indication
Bestrophin-1 (BEST1)-related IRD.
Stage
phase1
Event
Topline Data
Details

September 2, 2026: Opus Genetics, Inc. announced that topline clinical data from Cohort 1 of its Phase 1/2 OPGx-BEST1 gene therapy study will be presented on an investor conference call/webcast on September 9, 2026, at 8:00 a.m. ET. OPGx-BEST1 is an investigational AAV gene therapy for BEST1-related inherited retinal diseases, including Best vitelliform macular dystrophy (BVMD) and autosomal recessive bestrophinopathy (ARB).

The company said the full clinical dataset and additional visual function analyses will be presented at upcoming medical meetings. Listed presentations include:

  • Euretina Innovation Spotlight, September 30, 2026: company clinical development update.
  • Euretina 2026, October 4, 2026: initial results from Cohort 1 of the Phase 1b/2a study of OPGx-BEST1 in BVMD and ARB due to BEST1 mutations.
  • Retina in Rio, October 14, 2026: BIRD-1 Phase 1/2 results on structure-function correlation in BEST1-related retinopathy; also a separate presentation on near normalization of cone sensitivity using gene augmentation in children with LCA5.
  • FLORetina: Phase 1/2 Cohort 1 three-month results of OPGx-BEST1 in BVMD and ARB.

The ongoing BIRD-1 trial is an adaptive, open-label Phase 1/2 study evaluating single-eye subretinal OPGx-BEST1 in adults with BVMD or ARB.