Gene Therapies in Clinical Development to Treat Retinal Disorders

Journal article : Review

Gene therapies have emerged as promising treatments in clinical development for various retinal disorders, offering hope to patients with inherited degenerative eye conditions. Several gene therapies have already shown remarkable success in clinical trials, with significant improvements observed in visual acuity and the preservation of retinal function. A multitude of gene therapies have now been delivered safely in human clinical trials for a wide range of inherited retinal disorders but there are some gaps in the reported trial data. Some of the most exciting treatment options are not under peer review and information is only available in press release form. Whilst many trials appear to have delivered good outcomes of safety, others have failed to meet primary endpoints and therefore not proceeded to phase III. Despite this, such trials have enabled researchers to learn how best to assess and monitor patient outcomes, which will guide future trials to greater success. In this review, we consider recent and ongoing clinical trials for a variety of potential retinal gene therapy treatments and discuss the positive and negative issues related to these trials. We discuss the treatment potential following clinical trials as well as the potential risks of some treatments under investigation. As these therapies continue to advance through rigorous testing and regulatory approval processes, they hold the potential to revolutionise the landscape of retinal disorder treatments, providing renewed vision and enhancing the quality of life for countless individuals worldwide.

Authors: McClements, ME; Elsayed, MEAA; Major, L; de la Camara, CM; MacLaren, RE

• Publication status: Published
• Peer review status: Peer reviewed
• Publisher: Springer
• Journal: Molecular Diagnosis & Therapy
• Volume: 28
• Issue: 5
• Pages: 575-591
• Publication date: 2024-07-02
• Acceptance date: 2024-05-30
• DOI: 10.1007/s40291-024-00722-0
• EISSN: 1179-2000
• ISSN: 1177-1062
• Language: English
• Subtype: Review