FDA Clears IND for Opus Genetics’ OPGx-BEST1 Gene Therapy Candidate

Opus Genetics announced that the FDA has accepted its investigational new drug (IND) application for OPGx-BEST1, a gene therapy for the treatment of bestrophin-1 (BEST1)-related inherited retinal diseases (IRDs).

The company now plans to initiate a Phase 1/2 clinical trial in the second half of 2025, advised Opus Genetics.

According to the company, the multicenter, 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. The trial will also explore biological activity through functional and anatomical endpoints, including changes in visual function and retinal structure.

OPGx-BEST1 leverages Opus Genetics’ adeno-associated virus-based gene therapy platform, designed to deliver a functional copy of the BEST1 gene directly to the retinal pigment epithelium cells where the defective gene resides. The program builds on extensive preclinical work demonstrating restoration of BEST1 protein expression and improved retinal function in relevant disease models, stated the company.