June 23 2026 —
A Korean research consortium is aiming to begin clinical studies by 2029 for a domestically developed gene therapy for inherited retinal diseases, a group of rare genetic disorders that can cause progressive vision loss and blindness.
The effort is part of the Gene and Cell Therapy Research Center project led by the Korea Research Institute of Bioscience and Biotechnology, or KRIBB, under the Korean government’s Global TOP Strategic Research Group initiative. The project has been underway since May 2024 and is focused on developing new gene and cell therapy technologies with domestic scientific and clinical capabilities.
Inherited retinal diseases are rare genetic disorders that often begin in childhood or adolescence. Symptoms can include night blindness, progressive loss of peripheral vision, tunnel vision, and, in some cases, blindness. More than 300 genes have been linked to inherited retinal disorders, and an estimated 15,000 to 20,000 people in Korea are affected.
Professor Park Tae-kwan of the Department of Ophthalmology at Soon Chun Hyang University Hospital Bucheon is participating in the project. His team is conducting efficacy studies in mouse models using gene therapy candidates developed by KRIBB and is helping prepare for potential first-in-human clinical studies targeted for 2029.
Among the programs under development, the most advanced candidate targets X-linked juvenile retinoschisis, an inherited retinal disease that primarily affects school-age boys. The condition causes splitting of the retinal layers, disrupting communication between retinal cells and gradually impairing vision. Many patients eventually become legally blind.
Although clinical studies for X-linked juvenile retinoschisis are already underway in the United States and China, the Korean research team aims to develop a therapy with improved efficacy and safety and with relevance to domestic patient populations. As part of the project, Professor Park is studying mechanisms associated with retinal splitting and vision loss in retinoschisis mouse models and supporting selection of the lead clinical candidate from multiple KRIBB-developed programs.
Once preclinical toxicology studies confirm safety, the team plans to seek approval from Korea’s Ministry of Food and Drug Safety to begin human testing. If approved, patient studies could begin in the first half of 2029.
The research team is also building a patient registry for individuals who may be interested in future clinical study participation. Patients can register through the ophthalmology department at Soon Chun Hyang University Hospital Bucheon.
Globally, only a limited number of retinal gene therapies have reached the market, underscoring both the complexity and potential significance of the Korean effort. If successful, the program could help expand access to gene therapy for inherited retinal diseases and support the development of Korea-based genetic medicines for rare ophthalmic disorders.
The project also reflects growing collaboration among industry, academia, hospitals, and government-backed research institutions in Korea. With multiple inherited retinal disease programs expected to move toward clinical testing over the next five years, domestically developed retinal gene therapies may become an increasingly important part of Korea’s rare disease innovation landscape.
