OCU410

Drug

Drug Profile

OCU410 is an investigational one-time intravitreal AAV5-RORA modifier gene therapy being developed by Ocugen for geographic atrophy secondary to dry age-related macular degeneration. The program has reported positive preliminary Phase 2 data and is planned for Phase 3 progression with a targeted BLA filing in 2028. It remains unapproved.

Drug Class
Investigational AAV5-based modifier gene therapy (AAV5-RORA)
Approval Status
Investigational; Phase 2 with planned Phase 3 and BLA filing target in 2028
Mechanism of Action
Delivers RORA to regulate multiple retinal homeostasis pathways including oxidative stress response, complement regulation, inflammation, and lipid metabolism
Brand Names
  • OCU410
Indications
  • \Geographic atrophy (GA) secondary to dry age-related macular degeneration (dAMD)\

Related News

Ocular Therapeutix's Axpaxli Outperforms Eylea in Wet AMD Trial

Ocular Therapeutix's experimental drug Axpaxli showed superior vision maintenance compared to Regeneron's Eylea in a late-stage wet AMD trial. Meanwhile, Ocugen reported 12-month data showing its gene therapy OCU410 reduced geographic atrophy lesions by 31%, though less than earlier interim results. Both companies are advancing toward regulatory submissions and further clinical development.

Ocugen Reports Phase 2 Data for Geographic Atrophy Gene Therapy OCU410

Ocugen reported Phase 2 data for its geographic atrophy gene therapy OCU410 showing 31% lesion growth reduction versus control. The company plans Phase 3 initiation in Q3 2026 and maintains its goal of three BLA filings in three years. Shares fell 11.48% following the data release which trailed earlier interim results.