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Frequency and Pattern of Worldwide Ocular Gene Therapy Clinical Trials up to 2022
Hossein Ameri1, Niranjana Kesavamoorthy1, Dara N Bruce1
1Department of Ophthalmology, USC Roski Eye Institute, Keck School of Medicine, University of Southern California, Los Angeles, CA 90033, USA.
Abstract:
The purpose of this study is to describe worldwide gene therapy clinical trials aimed at treating ophthalmic disorders. Information regarding all worldwide clinical trials was collected through 15 different sources, including ClinicalTrials.gov. There were 159 gene therapy clinical trials on ophthalmic diseases up until 2022. Phase 1/2 trials had the highest frequency (50-32%), followed by phase 2 (33-21%); 107 trials (67%) were conducted in a single country, and 50 trials (31%) were multinational. Overall, the USA was the site of 113 (71%) single or multinational trials. Of the trials, 153 (96%) targeted retina and optic nerve disorders, 3 (2%) glaucoma, 2 (1%) uveitis, and 1 (1%) cornea; 104 trials (65%) employed gene augmentation using viral vectors, and the remaining employed other methods such as inhibitory RNA (18-11%) and cell-based gene therapy using encapsulated cell technology (18-11%). For gene augmentation trials, adeno-associated virus was used for transgene delivery in 87% of cases. The most common conditions targeted by gene augmentation included inherited retinal (74%) and age-related macular degeneration (wet, 14%; dry, 7%). Overall, a large number of gene therapy clinical trials have been conducted in the eye, and so far, one has led to regulatory approval.
Insights
Worldwide gene therapy clinical trials for eye diseases are numerous, with most targeting retinal disorders using viral vectors. One trial has achieved regulatory approval, highlighting progress in ophthalmic gene therapy.
Area of Science:
- Ophthalmology
- Gene Therapy
- Clinical Trials
Background:
- Gene therapy offers a promising avenue for treating various ophthalmic disorders.
- Significant research efforts are focused on developing gene-based treatments for eye conditions.
Purpose of the Study:
- To comprehensively review and describe global gene therapy clinical trials for ophthalmic diseases.
- To analyze the landscape of these trials up to 2022, including their phases, locations, targets, and methodologies.
Main Methods:
- Systematic data collection from 15 sources, including ClinicalTrials.gov.
- Analysis of 159 gene therapy clinical trials conducted worldwide up to 2022.
- Categorization based on trial phase, geographical distribution, targeted diseases, and therapeutic approaches.
Main Results:
- 159 gene therapy trials for ophthalmic diseases were identified up to 2022.
- Phase 1/2 and Phase 2 trials were most frequent; the USA led in trial conduct.
- Retina and optic nerve disorders were the primary targets (96%), with inherited retinal diseases and age-related macular degeneration being most common.
- Gene augmentation using viral vectors, particularly adeno-associated virus, was the predominant method (65%).
Conclusions:
- A substantial number of gene therapy clinical trials have been initiated for ophthalmic conditions.
- The field shows progress, with one trial having received regulatory approval.
- Gene augmentation via viral vectors is a leading strategy for treating retinal disorders.
