On August 18, 2025, Opus Genetics, Inc. announced that the U.S. Food and Drug Administration (FDA) accepted its Investigational New Drug (IND) application for OPGx-BEST1. This gene therapy is designed for the treatment of bestrophin-1 (BEST1)-related inherited retinal disease, also known as Best disease.
With this IND clearance, Opus Genetics plans to initiate a Phase 1/2 clinical trial in the second half of 2025. This multi-center, open-label study will evaluate the safety, tolerability, and preliminary efficacy of a single subretinal injection of OPGx-BEST1 in patients with genetically confirmed BEST1-related IRD.
BEST1-related IRDs currently have no approved treatments, making OPGx-BEST1 a potentially transformative approach. The program leverages Opus Genetics’ proprietary AAV-based gene therapy platform and builds on preclinical work demonstrating restoration of BEST1 protein expression and improved retinal function.
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