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OPGx BEST1

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Drug Profile

OPGx BEST1

Alternative Names: IC-200 - IVERIC Bio; OPGx-BEST1

Latest Information Update: 29 Sep 2025

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At a glance

  • Originator University of Florida Research Foundation; University of Pennsylvania
  • Developer Opus Genetics
  • Class Eye disorder therapies; Gene therapies
  • Mechanism of Action Bestrophin replacements; Gene transference
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    No
  • New Molecular Entity No
  • Available For Licensing Yes

Highest Development Phases

  • Phase I/II Vitelliform macular dystrophy

Most Recent Events

  • 22 Sep 2025 Phase-I/II clinical trials in Vitelliform macular dystrophy in USA (Subretinal) (NCT07185256)
  • 18 Aug 2025 US FDA approves IND application for OPGx BEST1 in BEST1-related in Opus Genetics
  • 18 Aug 2025 Opus Genetics plans a phase I/II trial for Vitelliform macular dystrophy in USA in the second half of 2025

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