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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)

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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: 05 May 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 Apr 2026 According to an Opus Genetics media release, data from this study will be presented at the Association for Research in Vision and Opthalmology (ARVO) Meeting.
  • 10 Mar 2026 According to an Opus Genetics media release, recruitment is ongoing at multiple U.S. sites with two participants treated to date in the Phase 1/2 trial (BIRD-1) that includes patients with both dominant and recessive forms of BEST disease.
  • 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.

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