Ocugen's OCU410 gene therapy has demonstrated encouraging safety and efficacy signals in early-stage clinical trials for geographic atrophy, a severe form of dry age-related macular degeneration. The investigational treatment, which delivers the RORA gene through a single subretinal injection, showed meaningful reductions in disease progression without serious safety concerns.
Phase 1/2 Trial Results Show Disease Modification
In the ArMaDa study, a phase 1/2 open-label dose-escalation trial, nine patients received subretinal injections of OCU410 at varying dose levels. After six months of follow-up, patients in the low-dose cohort experienced a 21.4% reduction in geographic atrophy lesion growth in treated eyes compared to their untreated fellow eyes, according to Jay Chhablani, MD, principal investigator and professor of ophthalmology at University of Pittsburgh School of Medicine.
The 12-month follow-up data presented at the American Society of Retina Specialists meeting revealed even more substantial benefits. Researchers observed more than a 40% decrease in lesion size and a four-line improvement in low luminance visual acuity among treated patients.
Single-Dose Approach Offers Treatment Convenience
Unlike current therapies that require repeated monthly injections, OCU410 represents what Chhablani described as an "almost one-and-done therapy" delivered through a single surgical intervention. The subretinal delivery method involves introducing the gene therapy in the peripheral retinal area, targeting multiple mechanisms involved in geographic atrophy progression.
"The treatment was delivered subretinally, requiring a single surgical intervention rather than repeated monthly injections," Chhablani explained during his presentation at the 2025 ASRS meeting in Long Beach, California.
Safety Profile Supports Continued Development
The safety profile remained favorable throughout the study period, with no serious adverse events reported during the 12-month follow-up. While some pigmentary changes were observed in the initial follow-up period, these changes were not expected to significantly impact patient vision due to the treatment's specific peripheral retinal location.
Patients experienced no serious side effects related to the therapy, which targets multiple mechanisms involved in geographic atrophy progression, according to Chhablani's presentation of the preliminary data at the ARVO meeting.
Clinical Development Advances Toward Phase 3
With phase 2 enrollment already completed, Ocugen is preparing to initiate phase 3 trials in the first quarter of 2026. The progression to late-stage development reflects the promising therapeutic potential demonstrated in early clinical testing.
"We really look forward for long-term data, and they aim to start their phase 3 studies in the first quarter of next year," Chhablani noted, expressing optimism about the therapy's continued development trajectory.
The gene therapy approach represents a potentially transformative method for managing dry macular degeneration by offering patients a single surgical intervention that could provide long-term benefits, presenting an alternative to ongoing repetitive treatments currently available for this degenerative eye condition.
