Ocugen, Inc., a biotechnology company focused on gene and cell therapies, hosted a high-profile Clinical Showcase in New York City to present updates from its pipeline of retinal gene therapy trials. The event, held at the Nasdaq MarketSite in Times Square, brought together leading researchers, clinicians, and patients to discuss the progress of three key clinical trials targeting rare and degenerative eye diseases.

Trial Highlights

OCU400 – liMeliGhT Phase 3 Trial for Retinitis Pigmentosa (RP) and Leber Congenital Amaurosis (LCA)

Ocugen shared new data from its pivotal Phase 3 trial evaluating OCU400, a modifier gene therapy designed to treat RP and LCA. The therapy uses an AAV-based delivery system to introduce a functional copy of the NR2E3 gene, aiming to restore photoreceptor function. Early results suggest stabilization of visual acuity and improvements in retinal sensitivity, with a favorable safety profile.

OCU410 – ArMaDa Phase 1/2 Trial for Geographic Atrophy (GA)

This trial targets GA, a late-stage form of dry age-related macular degeneration. Preliminary data from the ArMaDa study indicate that OCU410 may slow the progression of retinal degeneration, with no serious adverse events reported. The therapy leverages the RORA gene to modulate inflammation and oxidative stress in retinal cells.

OCU410ST – GARDian Phase 1/2 Trial for Stargardt Disease

Stargardt disease, a genetic form of macular degeneration affecting children and young adults, is notoriously difficult to treat. Ocugen’s OCU410ST trial is exploring the same RORA-based platform used in GA, adapted for Stargardt pathology. Investigators reported early signs of photoreceptor preservation, and patient-reported outcomes suggest improved contrast sensitivity and night vision.

Expert Panel & Patient Voices

The showcase featured presentations from prominent ophthalmologists:

• Dr. Benjamin Bakall, University of Arizona
• Dr. Lejla Vajzovic, Duke University
• Dr. Syed M. Shah, Gundersen Health

A patient panel concluded the event, offering firsthand accounts of life with inherited retinal diseases and the impact of participating in gene therapy trials. One participant shared:

What’s Next for Ocugen

Ocugen also announced plans to initiate a Phase 1 trial for OCU200, a biologic candidate targeting diabetic macular edema, by the end of Q4 2024. The company’s expanding pipeline reflects a strategic commitment to addressing unmet needs in ophthalmology through innovative gene and biologic therapies.

Industry Impact

Ocugen’s multi-trial approach underscores the growing momentum in precision ophthalmology. With no current cures for RP, GA, or Stargardt disease, these trials represent a beacon of hope for thousands of patients worldwide. The company’s use of modifier gene therapy—targeting upstream regulators rather than single mutations—could set a new standard for treating genetically heterogeneous conditions.