– Phase 1/2 trial expected to initiate in 2H 2025 in patients with BEST1 inherited retinal disease
Opus Genetics, a clinical-stage biopharmaceutical company developing gene therapies for the treatment of inherited retinal diseases (IRDs) and small molecule therapies for other ophthalmic disorders, announced that the U.S. Food and Drug Administration (FDA) has accepted its Investigational New Drug (IND) application for OPGx-BEST1, a gene therapy for the treatment of bestrophin-1 (BEST1)-related IRD.
Best disease, or vitelliform macular dystrophy, is a rare, inherited retinal condition causing macular degeneration by mutations in the BEST1 gene, leading to progressive vision loss and, in some cases, blindness.
With this IND clearance, Opus Genetics plans to initiate a phase 1/2 clinical trial in the second half of 2025.
The 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.
The trial will also explore biological activity through functional and anatomical endpoints, including changes in visual function and retinal structure.
“The FDA’s clearance of our BEST1 IND is a significant step forward for the IRD community and for our mission at Opus Genetics focused on restoring vision for patients,” said George Magrath, MD, CEO, Opus Genetics. “BEST1-related IRDs have no approved treatments today, leaving patients and families with uncertainty about the future of their vision. The OPGx-BEST1 trial will be our third ongoing clinical program, reflecting the depth of our pipeline and our commitment to advancing multiple therapies in parallel for patients with urgent, unmet needs.”
OPGx-BEST1 leverages Opus Genetics’ proprietary AAV-based gene therapy platform, designed to deliver a functional copy of the BEST1 gene directly to the retinal pigment epithelium (RPE) 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.