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. 2023 Nov 23;11(12):3124.
doi: 10.3390/biomedicines11123124.

Frequency and Pattern of Worldwide Ocular Gene Therapy Clinical Trials up to 2022

Hossein Ameri  1 , Niranjana Kesavamoorthy  1 , Dara N Bruce  1
Affiliations

Frequency and Pattern of Worldwide Ocular Gene Therapy Clinical Trials up to 2022

Hossein Ameri et al. Biomedicines. .

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.

Keywords: Leber congenital amaurosis; age-related macular degeneration; clinical trials; gene augmentation; gene therapy; inherited retinal diseases; ocular; ophthalmic; retinitis pigmentosa; viral vectors.

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Conflict of interest statement

The authors declare no conflict of interest.

Figures

Figure 1
Figure 1
Distribution of ocular gene therapy clinical trials by start year.
Figure 2
Figure 2
Phases of ocular gene therapy clinical trials (top) and their distribution by start year (bottom).
Figure 3
Figure 3
Geographical distribution of ocular gene therapy clinical trials in single-country trials (top) and multinational trials (bottom).
Figure 4
Figure 4
Categories of ocular gene therapy clinical trials (top) and their distribution by start year (bottom).
Figure 5
Figure 5
Vectors used in ocular gene augmentation trials (top) and their distribution by start year (bottom).
Figure 6
Figure 6
Diseases targeted in ocular gene augmentation trials (top) and their distribution by start year (bottom). The separated piece of pie indicates acquired conditions (top).
See this image and copyright information in PMC

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