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A Phase 1b/2a, Open-Label, Dose-Exploration Basket Study to Investigate the Safety and Tolerability of Subretinally Injected OPGx-BEST1 Administered in Patients With Either Autosomal-Dominant BEST1 Disease (Best Vitelliform Macular Dystrophy [BVMD]) or Autosomal-Recessive Bestrophinopathy (ARB)

Trial Profile

A Phase 1b/2a, Open-Label, Dose-Exploration Basket Study to Investigate the Safety and Tolerability of Subretinally Injected OPGx-BEST1 Administered in Patients With Either Autosomal-Dominant BEST1 Disease (Best Vitelliform Macular Dystrophy [BVMD]) or Autosomal-Recessive Bestrophinopathy (ARB)

Status: Recruiting
Phase of Trial: Phase I/II

Latest Information Update: 10 Mar 2026

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At a glance

  • Drugs OPGx BEST1 (Primary)
  • Indications Vitelliform macular dystrophy
  • Focus Adverse reactions
  • Acronyms BIRD-1
  • Sponsors Opus Genetics

Most Recent Events

  • 27 Feb 2026 According to an Opus Genetics media release, Two participants have been enrolled to date, with 3-month results from the full Cohort 1 expected in mid-year 2026.
  • 27 Feb 2026 According to an Opus Genetics media release, data from this trial was presented at the 49th Annual Meeting of the Macula Society, in San Diego, California.
  • 27 Feb 2026 Results presented in the Opus Genetics media release.

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