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Opus Genetics Announces FDA Clearance of IND Application for Gene Therapy Candidate OPGx-BEST1
Globenewswireยท 2025-08-18 12:00
Core Insights - Opus Genetics has received FDA acceptance for its Investigational New Drug (IND) application for OPGx-BEST1, a gene therapy targeting BEST1-related inherited retinal disease (IRD) [1][4] - The Phase 1/2 clinical trial is set to begin in the second half of 2025, focusing on safety, tolerability, and preliminary efficacy of a single subretinal injection [3][4] - BEST1-related IRDs currently lack approved treatments, highlighting the urgency for effective therapies in this area [4] Company Overview - Opus Genetics is a clinical-stage biopharmaceutical company specializing in gene therapies for inherited retinal diseases and small molecule therapies for other ophthalmic disorders [5] - The company's pipeline includes AAV-based gene therapies for conditions such as Leber congenital amaurosis (LCA) and retinitis pigmentosa, with OPGx-LCA5 also in an ongoing Phase 1/2 trial [5] - The company is based in Research Triangle Park, NC, and is committed to advancing multiple therapies for patients with unmet needs [5]