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Review of gene therapies for age-related macular degeneration
Open accessAbstract
Gene therapies aim to deliver a therapeutic payload to specified tissues with underlying protein deficiency. Since the 1990s, gene therapies have been explored as potential treatments for chronic conditions requiring lifetime care and medical management. Ocular gene therapies target a range of ocular disorders, but retinal diseases are of particular importance due to the prevalence of retinal disease and the current treatment burden of such diseases on affected patients, as well as the challenge of properly delivering these therapies to the target tissue. The purpose of this review is to provide an update on the most current data available for five different retinal gene therapies currently undergoing clinical trials for use against age-related macular degeneration (AMD) and the development of novel delivery routes for the administration of such therapies. Research has been performed and compiled from PubMed and the select authors of this manuscript on the treatment and effectiveness of five current retinal gene therapies: Luxturna, ADVM-022, RGX-314, GT-005, and HMR59. We present the available data of current clinical trials for the treatment of neovascular and dry age-related macular degeneration with different AAV-based gene therapies. We also present current research on the progress of developing novel routes of administration for ocular gene therapies. Retinal gene therapies offer the potential for life-changing treatment for chronic conditions like age-related macular degeneration with a single administration. In doing so, gene therapies change the landscape of treatment options for these chronic conditions for both patient and provider.
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“Gene therapy, successfully used in rare, monogenetic disorders, may prove to be a durable management approach for common, polygenetic conditions, including neovascular age-related macular degeneration (nAMD).”
From Safety and efficacy of ixoberogene soroparvovec in neovascular age-related macular degeneration in the United States (OPTIC): a prospective, two-year, multicentre phase 1 study (2024) · cites this paper as [3]
Experimental — accuracy not yet measuredThe source backs part of the claim, not all of it — or backs it for a different population, condition or comparison.
In the citing paper
“Gene therapy for retinal disease is especially promising since most inherited retinal disorders (IRDs) are monogenetic, cells are post-mitotic, and the imaging modalities used for diagnosis and monitoring are well-described and non-invasive.2,3”
From Safety and efficacy of ixoberogene soroparvovec in neovascular age-related macular degeneration in the United States (OPTIC): a prospective, two-year, multicentre phase 1 study (2024) · cites this paper as [3]
Experimental — accuracy not yet measuredThe source backs part of the claim, not all of it — or backs it for a different population, condition or comparison.
In the citing paper
“Due to its relative immune privilege, the eye represents an ideal environment to deliver viral vector-based in vivo gene therapy via intravitreal, subretinal, or suprachoroidal delivery.2,3”
From Safety and efficacy of ixoberogene soroparvovec in neovascular age-related macular degeneration in the United States (OPTIC): a prospective, two-year, multicentre phase 1 study (2024) · cites this paper as [3]
Experimental — accuracy not yet measuredThe source backs part of the claim, not all of it — or backs it for a different population, condition or comparison.
In the citing paper
“Aflibercept is an anti-VEGF recombinant fusion protein, which was FDA approved for nAMD in 2011 and has been administered intravitreally over 20 million times worldwide to treat VEGF-mediated retinal disorders.3,7”
From Safety and efficacy of ixoberogene soroparvovec in neovascular age-related macular degeneration in the United States (OPTIC): a prospective, two-year, multicentre phase 1 study (2024) · cites this paper as [3]
Experimental — accuracy not yet measuredIn the citing paper
“Several ocular gene therapies are in development for disorders affecting individuals older than 65 years (e.g., age-related macular degeneration [AMD]).27,28”
From Characterization of the ocular inflammatory response to AAV reveals divergence by sex and age (2025) · cites this paper as [28]
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Citations
Cites
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“In 1999, gene therapy for the treatment of severe combined immunodeficiency-X1 ultimately resulted in the death of one patient from unforeseen oncogene activation [4].”
BackgroundT Lymphocyte-Directed Gene Therapy for ADA − SCID: Initial Trial Results After 4 Years
Blaese, R. M., Culver, K. W., Miller, A. D., Carter, C. S., Fleisher, T. A., Clerici, M., Shearer, G. M., Chang, L., Chiang, Y. L., Tolstoshev, P., et al. (1995) · Science · doi:10.1126/science.270.5235.475 · Cited by 1,546
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Research works printed but not linked: 2
- Khanani AM, Kiss S, Turpcu A, Hoang C, Osborne A; Phase 1 Study of Intravitreal Gene Therapy ADVM-022 for neovascular AMD (OPTIC Trial). Invest. Ophthalmol. Vis. Sci. 2020;61:1154.
- Busbee BG, Boyer DS, Khanani AM, et al; Phase 1 Study of Intravitreal Gene Therapy with ADVM-022 for neovascular AMD (OPTIC Trial). Invest. Ophthalmol. Vis. Sci. 2021;62:352.
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Metadata
- Authors
- Khanani, A. M., Thomas, M. J., Aziz, A. A., Weng, C. Y., Danzig, C. J., Yiu, G., Kiss, S., Waheed, N. K., & Kaiser, P. K.
- Affiliations
- University of Nevada, Reno (US)
- Baylor College of Medicine (US)
- Florida Atlantic University (US)
- University of California, Davis (US)
- NewYork–Presbyterian Hospital (US)
- Tufts University (US)
- Cleveland Clinic (US)
- Corresponding author
- Khanani, Arshad M.
- ORCID
- Venue
- Eye
- Published
- 2022-01-11
- Volume
- 36
- Issue
- 2
- Pages
- 303-311
- ISSN
- 0078-5334 (ISSN-L)
- 0950-222X
- 1476-5454
- PMC
- PMC8807824
- OpenAlex
- W4206710973
- Cited by
- 119
- References
- 71
- Citations by year
- 2026: 15 · 2025: 29 · 2024: 36 · 2023: 32 · 2022: 7
- FWCI
- 2.71
- Type
- journal article
- Language
- en
- Open access
- bronze
- doi.org
- Licence
- Locations
- Eye (publishedVersion)
- PubMed Central (publishedVersion)
- PubMed (publishedVersion)
- Digital Commons-TMC (Texas Medical Center) (submittedVersion)
- eScholarship (California Digital Library) (submittedVersion)
- Topics
- Health Sciences › Medicine › Ophthalmology › Retinal Development and Disorders
- Health Sciences › Medicine › Ophthalmology › Retinal Diseases and Treatments
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Keywords
- age-related macular degeneration, retinal gene therapy, adeno-associated virus vectors, neovascular age-related macular degeneration, dry age-related macular degeneration, Luxturna, clinical trials
- MeSH terms
- Humans, Macular Degeneration, Genetic Therapy, Geographic Atrophy
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