Opus Genetics Presents Positive Clinical Data from OPGx-BEST1 and OPGx-LCA5 Gene Therapy Programs at Retina in Rio 2026
Opus Genetics presented positive interim clinical data for its OPGx-BEST1 and OPGx-LCA5 gene therapy programs at Retina in Rio 2026. OPGx-BEST1 showed improved retinal sensitivity in 75% of evaluable low-dose Cohort 1 participants in the Phase 1/2 BIRD-1 trial; the high-dose Cohort 2 has completed enrollment with 11 participants. OPGx-LCA5 demonstrated sustained improvements in visual acuity and light sensitivity through 24 months in adults and encouraging six-month results in pediatric participants, supporting advancement into Phase 3.
Key figures
- Drug Name
- OPGx-BEST1 and OPGx-LCA5
- Phase Of Trial
- Phase 1/2 (OPGx-BEST1 BIRD-1); Phase 1/2/3 (OPGx-LCA5)
- Lca5 duration
- sustained improvements in adults through 24 months; pediatric through 6 months
- Phase3 dosing timing
- Q4 2026
- Best1 cohort1 responders
- 3 of 4 evaluable participants (75%) with improved retinal sensitivity
- Best1 high dose cohort2 enrolled
- 11
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