Opus Genetics, Inc. ((IRD)) announced an update on their ongoing clinical study.
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Opus Genetics, Inc. is conducting a clinical study titled ‘A Phase 1b/2a, Open-Label, Dose-Exploration Basket Study to Investigate the Safety and Tolerability of Subretinally Injected OPGx-BEST1 Administered in Patients With Either Autosomal-Dominant BEST1 Disease (Best Vitelliform Macular Dystrophy [BVMD]) or Autosomal-Recessive Bestrophinopathy (ARB)’. The study aims to evaluate the safety, tolerability, and efficacy of the genetic therapy OPGx-BEST1 in treating BVMD and ARB, with a focus on identifying the optimal dose for clinical development.
The intervention being tested is OPGx-BEST1, a genetic therapy administered as a single subretinal injection. It is designed to treat patients with BVMD or ARB by potentially improving vision and reducing disease symptoms.
This interventional study follows a single-group assignment model without masking, focusing on treatment as its primary purpose. The study is open-label, meaning both researchers and participants know the treatment being administered.
The study began on July 17, 2025, with its primary completion and estimated completion dates yet to be announced. The latest update was submitted on September 24, 2025, indicating ongoing recruitment.
The study’s progress could positively influence Opus Genetics’ stock performance and attract investor interest, given the potential breakthrough in treating rare eye conditions. Competitors in the genetic therapy space may also be closely monitoring these developments.
The study is currently recruiting, with further details available on the ClinicalTrials portal.