Opus Genetics Receives FDA Clearance for IND Application of Gene Therapy Candidate OPGx-BEST1
Tuesday, August 19, 2025
Opus Genetics (Nasdaq: IRD), a clinical-stage biopharmaceutical company focused on gene therapies for inherited retinal diseases (IRDs) and small molecule treatments for eye disorders, has gained clearance from the U.S. Food and Drug Administration (FDA) for its Investigational New Drug (IND) application of OPGx-BEST1. The therapy is designed for the treatment of BEST1-related IRD.
Best disease, also called vitelliform macular dystrophy, is a rare inherited retinal disorder caused by mutations in the BEST1 gene. It results in macular degeneration, leading to progressive vision loss and, in some cases, complete blindness.
Following the IND approval, Opus Genetics plans to begin a Phase 1/2 clinical trial in the second half of 2025. This multi-centre, open-label study will assess the safety, tolerability, and early efficacy of a single subretinal injection of OPGx-BEST1 in patients with genetically confirmed BEST1-related IRD. The trial will also measure biological activity by evaluating changes in visual function and retinal structure.
OPGx-BEST1 uses the company’s AAV-based gene therapy platform to deliver a functional copy of the BEST1 gene directly to retinal pigment epithelium (RPE) cells, targeting the site of the genetic defect. Preclinical studies have shown restoration of BEST1 protein expression and improvement in retinal function in relevant disease models.
Source: globenewswire.com
