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 may be a durable management approach for common, polygenetic conditions, including neovascular age-related macular degeneration (nAMD).
“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]
The Abstract and Discussion describe gene therapy as a potential single-administration treatment for AMD, and the nAMD therapy section discusses approaches intended to reduce repeated injections. The Retinal gene therapy section notes that many disorders are polygenic, but the review does not identify nAMD as polygenic or establish the broader claim for common polygenic conditions. No page numbers are available in our copy. (LLM, conf=0.80)
Experimental — accuracy not yet measuredGene therapy for retinal disease is especially promising because most inherited retinal disorders are monogenetic, cells are post-mitotic, and imaging modalities used for diagnosis and monitoring are well-described and non-invasive.
“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]
The Retinal gene therapy section says that individual genes are responsible for a variety of ocular conditions, supporting the appeal of single-gene replacement, but it does not establish that most inherited retinal disorders are monogenetic. The Viral vector gene therapy section discusses nondividing cells generally, and the trial discussion mentions optical coherence tomography as an outcome measure; neither states that retinal cells are post-mitotic or that diagnostic and monitoring imaging is well-described and non-invasive. No page numbers were available in our copy. (LLM, conf=0.86)
Experimental — accuracy not yet measuredDue to its relative immune privilege, the eye is an ideal environment to deliver viral vector-based in vivo gene therapy via intravitreal, subretinal, or suprachoroidal delivery.
“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]
The Routes of delivery section (page unavailable) identifies intravitreal, subretinal, and suprachoroidal routes for ocular genetic therapies. The Subretinal injections section describes an immune-privileged location, but the review does not extend that immune advantage to all three routes; it notes potential immune responses with intravitreal and suprachoroidal delivery. (LLM, conf=0.85)
Experimental — accuracy not yet measuredAflibercept is an anti-VEGF recombinant fusion protein, FDA approved for nAMD in 2011 and administered intravitreally over 20 million times worldwide to treat VEGF-mediated retinal disorders.
“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]
In the ADVM-022 section (page unavailable), the review calls aflibercept an anti-VEGF protein used for retinal disease and says it has been FDA-approved since 2011; it also identifies Eylea as an aflibercept recombinant-protein injection. Our copy does not specify that it is a fusion protein or state that aflibercept has been administered intravitreally over 20 million times worldwide. (LLM, conf=0.88)
Experimental — accuracy not yet measuredSeveral ocular gene therapies are in development for disorders affecting individuals older than 65 years, such as age-related macular degeneration.
“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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- 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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Age-related macular degeneration
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Biomaterials used for tissue engineering of barrier-forming cell monolayers in the eye
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AAV NRF2 gene therapy preserves retinal structure and function in rodent models of oxidative damage
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Review of Gene Therapy Clinical Trials for Retinal Diseases
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Inhibitory effect of RGD peptide hydrogel on inflammation and angiogenesis in vitro
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Toward Precision Medicine: Gene Therapy Applications in the Management of Uveal Melanoma
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mRNA therapy: A novel approach for retinal neurodegenerative diseases
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Tratamiento actual de la degeneración macular relacionada con la edad
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CRISPR‐based gene therapy for wet age‐related macular degeneration in mouse model
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Self-citationWhat is on the horizon for dry age-related macular degeneration drug treatment?
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Self-citationEmergent therapeutics for the treatment of diabetic macular edema
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Immune Privilege of the Eye: A Delicate Balance Between Clarity and Chaos
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Ocular Gene Therapy: Literature Review
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Immunological and Inflammatory Side Effects of Treatments for Age-Related Macular Degeneration
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Neovaskuläre altersabhängige Makuladegeneration
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Updates on Gene Therapy for Neovascular Age-related Macular Degeneration: A Review
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- 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
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › 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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