Opus Genetics has unveiled preliminary findings from its Phase I/II clinical trial of the OPGx-BEST1 gene therapy, aimed at treating best vitelliform macular dystrophy (BVMD) and autosomal-recessive bestrophinopathy (ARB). These results were shared at the 49th annual meeting of the Macula Society held in San Diego, California.
Trial Overview
The study highlighted outcomes from a 63-year-old female participant diagnosed with ARB. After a three-month period, OPGx-BEST1 demonstrated a favorable safety profile, with no reported ocular inflammation or adverse events that could limit dosing.
Functional Improvements
Encouraging initial improvements were noted, particularly a 12-letter increase in best corrected visual acuity (BCVA) in the treated eye. Additionally, the central subfield thickness in the study eye saw a significant reduction of 23%, and intraretinal fluid was resolved within a month in areas with minimal atrophy.
Study Design
This open-label, adaptive Phase I/II study involves a single subretinal injection of OPGx-BEST1 per participant’s eye. Participants are divided into two dosing cohorts, focusing on adults affected by BVMD or ARB. The primary goals of the trial include assessing safety and tolerability, identifying the optimal dose for future studies, and monitoring long-term outcomes through both functional and anatomical measurements.
Positive Feedback from Leadership
George Magrath, CEO of Opus Genetics, expressed optimism regarding the results of the initial participant, noting that OPGx-BEST1 was well-tolerated and showed promising early efficacy at the three-month mark. He emphasized that this data represents a crucial milestone for both the OPGx-BEST1 program and patients suffering from BEST1-related retinal conditions.
Ongoing Recruitment
Recruitment for the trial is currently active at two sites in the United States, with plans for additional locations in Cincinnati, Florida, and New York. To date, two participants have been enrolled. The results from the full first cohort are expected to be available by mid-2026.
Future Outlook
In November 2025, the first subject was dosed in this innovative clinical trial, marking a significant step forward in the treatment of Best disease (BEST1). The ongoing research aims to refine the approach, ensuring that future therapies are both safe and effective for patients.
Key Takeaways
- Opus Genetics is conducting a Phase I/II trial of OPGx-BEST1 gene therapy for BVMD and ARB.
- Initial results show good tolerability and early signs of functional improvement in vision.
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The study is open-label and involves a single subretinal injection per participant.
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Recruitment is ongoing, with results from the full cohort expected in mid-2026.
In conclusion, the early findings from Opus Genetics’ trial of OPGx-BEST1 gene therapy indicate a promising avenue for treating debilitating retinal diseases. As the study progresses, it holds potential to significantly enhance the quality of life for patients affected by these conditions.
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