Drug Profile

OPGx BEST1

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

Latest Information Update: 16 Jan 2026

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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

08 Jan 2026 Opus Genetics plans a phase III trial in the second half of 2026
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

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