Opus Genetics Presents Positive Clinical Data from OPGx-BEST1 and OPGx-LCA5 Gene Therapy Programs at Retina in Rio 2026
Globe Newswire•15/10/2026•16:00 ET
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Key Highlights
- ➤OPGx-BEST1 improved retinal sensitivity in three of four evaluable participants.
- ➤OPGx-LCA5 adults sustained visual-acuity improvements through 24 months.
- ➤OPGx-LCA5 pediatric participants showed cone-vision sensitivity gains through six months.
- ➤OPGx-LCA5 Phase 3 dosing expected in fourth quarter of 2026.
- ➤OPGx-BEST1 high-dose Cohort 2 enrollment completed with 11 participants.
Expert Statements
George Magrath, Chief Executive Officer of Opus Genetics
“The data presented at Retina in Rio highlight meaningful progress across our gene therapy pipeline, with sustained improvements in visual function observed through 24 months in adults treated with OPGx-LCA5 and encouraging early evidence of improved retinal sensitivity in participants receiving OPGx-BEST1”
George Magrath, Chief Executive Officer of Opus Genetics
“Importantly, these findings strengthen our understanding of how to measure treatment response in IRDs and support the continued advancement of both programs.”
George Magrath, Chief Executive Officer of Opus Genetics
“Our OPGx-BEST1 Phase 1/2 trial has completed enrollment in the high-dose Cohort 2 with 11 participants, and with our OPGx-LCA5 Phase 3 trial progressing toward dosing initiation later this year, we remain focused on bringing potentially transformative gene therapies to patients living with rare inherited retinal diseases.”
OPGx-BEST1 showed improved retinal sensitivity in 75% of evaluable participants in the initial low-dose Cohort 1 of the Company’s Phase 1/2 clinical trial, supporting further clinical development
New BEST1 data analyses link retinal structure to visual function, enabling focus on regions most likely to respond to treatment
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