OCU410

OCU410 is a novel multifunctional modifier gene therapy candidate being developed by Ocugen for the treatment of geographic atrophy (GA) secondary to dry age-related macular degeneration (dAMD). It utilizes an adeno-associated virus (AAV) platform—specifically AAV5—to deliver the RAR-related orphan receptor alpha (RORA) gene directly to retinal cells. The RORA protein plays a key role in lipid metabolism, reduces lipofuscin deposits and oxidative stress, exhibits anti-inflammatory effects, and inhibits the complement system. By targeting multiple pathways implicated in GA pathophysiology, OCU410 aims to address limitations of current therapies that focus solely on the complement pathway and require frequent injections. Designed as a one-time subretinal injection, OCU410 has completed Phase 2 clinical trial dosing and is positioned as a potential gold standard for GA treatment due to its broad mechanism of action and favorable safety profile[1][4][6][7].