Opus Genetics announces FDA clearance of IND application for OPGx-BEST1

Opus Genetics (IRD) 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. With this IND clearance, Opus Genetics plans to initiate a Phase 1/2 clinical trial in the second half of 2025. “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, M.D., Chief Executive Officer, 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.”