OCU400 Gene Therapy Data Showcased at 2024 Retinal Summit

Ocugen, Inc., a company dedicated to the innovation of gene and cell therapies and vaccines, has announced that Dr. Benjamin Bakall will present findings from the OCU400 Phase 1/2 clinical trial at the upcoming Retinal Cell and Gene Therapy Innovation Summit. Dr. Bakall, a Clinical Assistant Professor at the University of Arizona, is eager to discuss the novel gene therapy for retinitis pigmentosa (RP), a condition that currently lacks effective treatments.

The summit, co-hosted by the Foundation Fighting Blindness and the Casey Eye Institute of Oregon Health and Science University, aims to unite professionals from various sectors to discuss advancements in ocular gene and cell therapies and strategize on the clinical application of these therapies.

Dr. Bakall's presentation will delve into the safety and efficacy of the nuclear hormone receptor-based gene modifier therapy for RP. The OCU400 Phase 3 clinical trial is progressing with the goal of meeting approval targets by 2026. RP, a genetic disorder leading to vision loss, affects nearly 300,000 people in the U.S. and EU.

OCU400 is a gene therapy product that targets the NR2E3 gene, which plays a crucial role in the retina's physiological functions. This therapy aims to restore balance and potentially improve retinal health for RP patients. Unlike gene replacement therapies, which are limited to a single mutation, OCU400's gene-agnostic approach offers hope for a broader range of the disease.

Ocugen, Inc. is committed to developing innovative therapies that can make a significant impact on global health. Their modifier gene therapy platform has the potential to treat multiple retinal diseases, and they are also advancing research in infectious and orthopedic diseases to address unmet needs.