Ocugen CSO to Present Modifier Gene Therapy at ISCT 2024

Ocugen, Inc. (NASDAQ: OCGN), a biotechnology firm dedicated to the development of novel gene and cell therapies, announced that its Chief Scientific Officer, Dr. Arun Upadhyay, will present OCU400 data at the International Society for Cell & Gene Therapy (ISCT) Annual Meeting in Vancouver, Canada, from May 29 to June 1, 2024. Dr. Upadhyay will share insights into the safety and efficacy results from a Phase 1/2 clinical trial of OCU400, a modifier gene therapy aimed at treating retinitis pigmentosa (RP).

Expressing his enthusiasm, Dr. Upadhyay stated, "I’m very pleased to present OCU400 data among an organization dedicated to translating cell and gene therapies into safe and effective treatments to improve patients’ lives." He highlighted OCU400 as a groundbreaking approach for potentially treating RP and Leber congenital amaurosis (LCA), diseases characterized by over 100 mutations.

The ISCT Annual Meeting serves as a platform for cutting-edge translational content and facilitates connections among global experts from academic institutions, regulatory bodies, and the healthcare industry. Participants will engage in comprehensive discussions, collaborate on developing sector solutions, and advance the field of cell and gene therapy.

Details for Dr. Upadhyay’s presentation are as follows:
- Session: Late-Breaking Oral Abstract Session: Safety and Efficacy Results from a Phase 1/2 Clinical Trial of OCU400 Modifier Gene Therapy for Treatment of Retinitis Pigmentosa
- Date: Thursday, May 30, 2024
- Time: 5-6 p.m. PDT
- Location: Room 202-204, Vancouver Convention Centre, West Building

Additionally, Dr. Upadhyay’s poster will be displayed during Poster Networking Session #1 on Wednesday, May 29, 2024, from 7-8:30 p.m. PDT.

The OCU400 Phase 3 liMeliGhT clinical trial is progressing well and aims to meet the Company’s 2026 target for Biologics License Application (BLA) and Marketing Authorization Application (MAA) approvals. Notably, OCU400 is the first gene therapy to enter Phase 3 trials for a broad RP indication.

About OCU400

OCU400 is a gene-agnostic modifier gene therapy leveraging the nuclear hormone receptor (NHR) gene, NR2E3. This gene regulates various physiological functions within the retina, such as photoreceptor development, metabolism, phototransduction, inflammation, and cell survival. OCU400 seeks to restore balance within altered cellular gene networks, potentially improving retinal health and function in RP patients. Approximately 300,000 people in the U.S. and EU are affected by RP.

About Modifier Gene Therapy

Modifier gene therapy addresses unmet medical needs related to retinal diseases, including inherited retinal diseases (IRDs) like RP, LCA, and Stargardt disease, as well as complex diseases like dry age-related macular degeneration (dAMD). This therapy uses NHRs, which can potentially restore homeostasis in the retina. Unlike single-gene replacement therapies targeting one genetic mutation, modifier gene therapy addresses multiple retinal diseases caused by various gene mutations with a single product. Ocugen has three modifier gene therapy programs in clinical development: OCU400, OCU410, and OCU410ST. Besides the OCU400 Phase 3 liMeliGhT trial, the OCU410 Phase 1/2 ArMaDa trial for geographic atrophy secondary to dAMD and the OCU410ST Phase 1/2 GARDian trial for Stargardt disease are ongoing. Geographic atrophy affects around two to three million people in the U.S. and EU, and Stargardt disease affects nearly 100,000 people in these regions.

About Ocugen, Inc.

Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines that aim to improve health and provide hope for patients worldwide. The company is committed to innovative scientific research that harnesses unique intellectual and human resources. Their modifier gene therapy platform has the potential to treat multiple retinal diseases with a single product. Additionally, Ocugen is advancing research in infectious diseases to support public health and orthopedic diseases to meet unmet medical needs.

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