Ocugen's CSO to Present on Modifier Gene Therapy at Retina World Congress

MALVERN, Pa., May 02, 2024 - Ocugen, Inc. (NASDAQ: OCGN), a biotechnology firm dedicated to the innovation of gene and cell therapies and vaccines, has announced that its Chief Scientific Officer, Dr. Arun Upadhyay, will be presenting at the Retina World Congress (RWC) from May 9-12, 2024 in Ft. Lauderdale, FL.

Dr. Upadhyay expressed his enthusiasm for the event, emphasizing the importance of such gatherings for sharing the latest advancements in modifier gene therapy and learning from fellow researchers in the field of retinal diseases. He highlighted the significance of these interactions in addressing the unmet medical needs of patients with blindness-causing diseases.

The RWC is one of the premier global conferences focusing on retinal health, attracting specialists worldwide to foster a comprehensive exchange of scientific and clinical knowledge.

Dr. Upadhyay will be part of the RWC Unplugged Panel Session titled "Gene Therapy, Pharmacotherapy, and Optogenetics for Inherited Retinal Diseases (IRD)" on Thursday, May 9, 2024, from 9:05 to 9:35 a.m. (ET) at the Marriott Harbor Beach Resort.

Additionally, Ocugen's research abstract on "Safety and Efficacy Results from a Phase 1/2 Clinical Trial of OCU400 Modifier Gene Therapy for Treatment of Retinitis Pigmentosa" has been selected for e-poster/video presentation and will be displayed during the conference.

The company is progressing with its OCU400 Phase 3 liMeliGhT clinical trial, which aims to meet the 2026 Biologics License Application (BLA) and Marketing Authorization Application (MAA) approval targets. OCU400 is notable for being the first gene therapy to advance to Phase 3 clinical trials with a broad application for retinitis pigmentosa (RP).

OCU400 leverages the NR2E3 nuclear hormone receptor gene, which plays a crucial role in various retinal functions, including photoreceptor development, metabolism, and cell survival. By resetting disrupted cellular gene networks, OCU400 aims to restore balance and improve retinal health in RP patients. An estimated 300,000 individuals in the U.S. and EU are affected by RP.

Modifier gene therapy, which Ocugen focuses on, addresses unmet medical needs in retinal diseases like RP, Leber congenital amaurosis, and Stargardt disease, as well as multifactorial conditions such as dry age-related macular degeneration (dAMD). This therapy utilizes nuclear hormone receptors (NHRs) to restore the retina's homeostasis, potentially treating multiple diseases with different genetic mutations using a single product.

Ocugen's pipeline includes three clinical programs: OCU400, OCU410, and OCU410ST. Besides the OCU400 Phase 3 trial, the company is conducting the OCU410 Phase 1/2 ArMaDa clinical trial for geographic atrophy secondary to dAMD and the OCU410ST Phase 1/2 GARDian clinical trial for Stargardt disease. Geographic atrophy affects approximately two to three million individuals in the U.S. and EU, while Stargardt disease impacts nearly 100,000 people in these regions.

Ocugen, Inc. is dedicated to discovering, developing, and commercializing innovative gene and cell therapies and vaccines. The company's breakthrough modifier gene therapy platform aims to treat multiple retinal diseases with a single product, while also advancing research in infectious and orthopedic diseases to address public health needs.