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Breakthroughs in age-related macular degeneration (AMD) treatments have revolutionized the management of this progressive and potentially devastating eye disease. These include treatments such as complement inhibitors and stem cell therapy fordry AMD, and long-acting anti-VEGF drugs and gene therapy for more advancedwet AMD.
These treatments may help slow disease progression and avoid vision loss in people with AMD. Some are available for use now, while others are in differentphases of clinical research.
Verywell / Brianna Gilmartin
Emerging Treatments for Dry AMD
Dry AMD occurs when the macula starts thinning with age and/or becomes covered with protein deposits known asdrusen. Depending on the severity of the damage, dry AMD may be classified as early-stage, intermediate-stage, or late-stage.
Currently, there is no cure for dry AMD, but two therapies appear effective in overcoming the mechanisms that give rise to the disease.
Complement Inhibitors
The drugs are known as complement inhibitors because they block a part of the immune system called the “complement cascade” that instigates the formation of these lesions.
Syfovre and Izervay are both injected directly into the eye every 25 to 60 days. While 80% of people with GA experience improvement with these drugs, 20% do not.
Both drugs are known to cause significant and sometimes severe side effects, including blurred vision,floaters, eye pain, andretinal detachment.
Stem Cell Therapy
Several methods have been proposed to introduce stem cells into the retina. One option is to inject them into the eye where they can migrate on their own to the retina. Another is to surgically implant them onto the retina using a piece of synthetic or natural material, known as scaffolding, that has been infused with stem cells.
Despite promising early results, experts suggest that it may take 10 years or more to perfect the procedure.Human clinical trials are currently underway are underway to assess the safety and effectiveness of the different treatment models.
Emerging Treatment for Wet AMD
Between 10% and 15% of people with dry AMD will progress to a more severe form of the disease, called wet AMD (also known as neovascular or exudate AMD).
Researchers have designed several treatments that may slow the progression of wet AMD and prevent irreversible vision loss and blindness.
Long-Acting Anti-VEGF Drugs
These include:
Anti-VEGF Treatments in the Pipeline
Gene Therapy
One promising alternative to anti-VEGF injections isgene therapyin which the eyes are given the genetic tools to make their own anti-VEGF agents.
There are two candidates currently under investigation:
RGX-314 is currently in phase 1/2 clinical trials. Among other things, the researchers want to determine which delivery system is most effective: either a standard eye injection or the surgical implantation of the viral vector under the retina.
Although both methods have proven beneficial, stimulating the production of anti-VEGF agents for up to two years, the long-term safety and effectiveness of RGX-314 have yet to be proven. It may take several more years for phase 3 human studies to establish this.
If proven effective, gene therapy may displace anti-VEGF injection as the preferred form of treatment for wet AMD.
Summary
With a better understanding of how age-related macular degeneration (AMD) occurs, researchers have been able to develop treatments that can slow and potentially stop this progressive eye disease. These include complement inhibitors and stem cell therapies for dry AMD and anti-VEGF agents and gene therapies for wet AMD.
A Word From VerywellWith the advent of the mRNA vaccines making antibodies to protect us from COVID-19, the treatments in gene therapy are very exciting. Using your body’s antibody factories with new gene instructions for anti-VEGF antibodies is right around the corner and can be game changing for AMD treatment.—ANDREW GREENBERG, MD, MEDICAL EXPERT BOARD
A Word From Verywell
With the advent of the mRNA vaccines making antibodies to protect us from COVID-19, the treatments in gene therapy are very exciting. Using your body’s antibody factories with new gene instructions for anti-VEGF antibodies is right around the corner and can be game changing for AMD treatment.—ANDREW GREENBERG, MD, MEDICAL EXPERT BOARD
With the advent of the mRNA vaccines making antibodies to protect us from COVID-19, the treatments in gene therapy are very exciting. Using your body’s antibody factories with new gene instructions for anti-VEGF antibodies is right around the corner and can be game changing for AMD treatment.
—ANDREW GREENBERG, MD, MEDICAL EXPERT BOARD
12 SourcesVerywell Health uses only high-quality sources, including peer-reviewed studies, to support the facts within our articles. Read oureditorial processto learn more about how we fact-check and keep our content accurate, reliable, and trustworthy.American Academy of Ophthalmology.New treatments for age-related macular degeneration.MedlinePlus.Age-related macular degeneration.Vakharia P, Eichenbaum D.Geographic atrophy: current and future therapeutic agents and practical considerations for retinal specialists.Curr Opin Ophthalmol. 2024 Feb 29. doi: 10.1097/ICU.0000000000001046American Academy of Ophthalmology.Geographic atrophy.Maeda T, Sugita S, Kurimoto Y, Takahashi M.Trends of stem cell therapies in age-related macular degeneration.J Clin Med. 2021 Apr 20;10(8):1785. doi:10.3390/jcm10081785Semeraro F, Morescalchi F, Russo A, et al.Central serous chorioretinopathy: pathogenesis and management.Clin Ophthalmol. 2019;13:2341-2352. doi:10.2147/OPTH.S220845Arepalli S, Kaiser PK.Pipeline therapies for neovascular age related macular degeneration.Int J Retina Vitreous. 2021;7(1):55. doi:10.1186/s40942-021-00325-5Ranade SV, Wieland MR, Tam T, et al.The port delivery system with ranibizumab: a new paradigm for long-acting retinal drug delivery.Drug Deliv. 2022;29(1):1326-1334. doi:10.1080/10717544.2022.2069301Gao Y, Lu F, Li X, et al.Safety and tolerability of oral vorolanib for neovascular (wet) age-related macular degeneration: a phase I, open-label study.Eye.2023;37:3228–3233. doi:10.1038/s41433-023-02496-xShirian JD, Shukla P, Singh RP.Exploring new horizons in neovascular age-related macular degeneration: novel mechanisms of action and future therapeutic avenues.Eye.2025;39:40-44. doi:10.1038/s41433-024-03373-xKhanani AM, Thomas MJ, Aziz AA, et al.Review of gene therapies for age-related macular degeneration.Eye. 2022;36(2):303-311. doi:10.1038/s41433-021-01842-1American Academy of Ophthalmology.Gene therapy for AMD.
12 Sources
Verywell Health uses only high-quality sources, including peer-reviewed studies, to support the facts within our articles. Read oureditorial processto learn more about how we fact-check and keep our content accurate, reliable, and trustworthy.American Academy of Ophthalmology.New treatments for age-related macular degeneration.MedlinePlus.Age-related macular degeneration.Vakharia P, Eichenbaum D.Geographic atrophy: current and future therapeutic agents and practical considerations for retinal specialists.Curr Opin Ophthalmol. 2024 Feb 29. doi: 10.1097/ICU.0000000000001046American Academy of Ophthalmology.Geographic atrophy.Maeda T, Sugita S, Kurimoto Y, Takahashi M.Trends of stem cell therapies in age-related macular degeneration.J Clin Med. 2021 Apr 20;10(8):1785. doi:10.3390/jcm10081785Semeraro F, Morescalchi F, Russo A, et al.Central serous chorioretinopathy: pathogenesis and management.Clin Ophthalmol. 2019;13:2341-2352. doi:10.2147/OPTH.S220845Arepalli S, Kaiser PK.Pipeline therapies for neovascular age related macular degeneration.Int J Retina Vitreous. 2021;7(1):55. doi:10.1186/s40942-021-00325-5Ranade SV, Wieland MR, Tam T, et al.The port delivery system with ranibizumab: a new paradigm for long-acting retinal drug delivery.Drug Deliv. 2022;29(1):1326-1334. doi:10.1080/10717544.2022.2069301Gao Y, Lu F, Li X, et al.Safety and tolerability of oral vorolanib for neovascular (wet) age-related macular degeneration: a phase I, open-label study.Eye.2023;37:3228–3233. doi:10.1038/s41433-023-02496-xShirian JD, Shukla P, Singh RP.Exploring new horizons in neovascular age-related macular degeneration: novel mechanisms of action and future therapeutic avenues.Eye.2025;39:40-44. doi:10.1038/s41433-024-03373-xKhanani AM, Thomas MJ, Aziz AA, et al.Review of gene therapies for age-related macular degeneration.Eye. 2022;36(2):303-311. doi:10.1038/s41433-021-01842-1American Academy of Ophthalmology.Gene therapy for AMD.
Verywell Health uses only high-quality sources, including peer-reviewed studies, to support the facts within our articles. Read oureditorial processto learn more about how we fact-check and keep our content accurate, reliable, and trustworthy.
American Academy of Ophthalmology.New treatments for age-related macular degeneration.MedlinePlus.Age-related macular degeneration.Vakharia P, Eichenbaum D.Geographic atrophy: current and future therapeutic agents and practical considerations for retinal specialists.Curr Opin Ophthalmol. 2024 Feb 29. doi: 10.1097/ICU.0000000000001046American Academy of Ophthalmology.Geographic atrophy.Maeda T, Sugita S, Kurimoto Y, Takahashi M.Trends of stem cell therapies in age-related macular degeneration.J Clin Med. 2021 Apr 20;10(8):1785. doi:10.3390/jcm10081785Semeraro F, Morescalchi F, Russo A, et al.Central serous chorioretinopathy: pathogenesis and management.Clin Ophthalmol. 2019;13:2341-2352. doi:10.2147/OPTH.S220845Arepalli S, Kaiser PK.Pipeline therapies for neovascular age related macular degeneration.Int J Retina Vitreous. 2021;7(1):55. doi:10.1186/s40942-021-00325-5Ranade SV, Wieland MR, Tam T, et al.The port delivery system with ranibizumab: a new paradigm for long-acting retinal drug delivery.Drug Deliv. 2022;29(1):1326-1334. doi:10.1080/10717544.2022.2069301Gao Y, Lu F, Li X, et al.Safety and tolerability of oral vorolanib for neovascular (wet) age-related macular degeneration: a phase I, open-label study.Eye.2023;37:3228–3233. doi:10.1038/s41433-023-02496-xShirian JD, Shukla P, Singh RP.Exploring new horizons in neovascular age-related macular degeneration: novel mechanisms of action and future therapeutic avenues.Eye.2025;39:40-44. doi:10.1038/s41433-024-03373-xKhanani AM, Thomas MJ, Aziz AA, et al.Review of gene therapies for age-related macular degeneration.Eye. 2022;36(2):303-311. doi:10.1038/s41433-021-01842-1American Academy of Ophthalmology.Gene therapy for AMD.
American Academy of Ophthalmology.New treatments for age-related macular degeneration.
MedlinePlus.Age-related macular degeneration.
Vakharia P, Eichenbaum D.Geographic atrophy: current and future therapeutic agents and practical considerations for retinal specialists.Curr Opin Ophthalmol. 2024 Feb 29. doi: 10.1097/ICU.0000000000001046
American Academy of Ophthalmology.Geographic atrophy.
Maeda T, Sugita S, Kurimoto Y, Takahashi M.Trends of stem cell therapies in age-related macular degeneration.J Clin Med. 2021 Apr 20;10(8):1785. doi:10.3390/jcm10081785
Semeraro F, Morescalchi F, Russo A, et al.Central serous chorioretinopathy: pathogenesis and management.Clin Ophthalmol. 2019;13:2341-2352. doi:10.2147/OPTH.S220845
Arepalli S, Kaiser PK.Pipeline therapies for neovascular age related macular degeneration.Int J Retina Vitreous. 2021;7(1):55. doi:10.1186/s40942-021-00325-5
Ranade SV, Wieland MR, Tam T, et al.The port delivery system with ranibizumab: a new paradigm for long-acting retinal drug delivery.Drug Deliv. 2022;29(1):1326-1334. doi:10.1080/10717544.2022.2069301
Gao Y, Lu F, Li X, et al.Safety and tolerability of oral vorolanib for neovascular (wet) age-related macular degeneration: a phase I, open-label study.Eye.2023;37:3228–3233. doi:10.1038/s41433-023-02496-x
Shirian JD, Shukla P, Singh RP.Exploring new horizons in neovascular age-related macular degeneration: novel mechanisms of action and future therapeutic avenues.Eye.2025;39:40-44. doi:10.1038/s41433-024-03373-x
Khanani AM, Thomas MJ, Aziz AA, et al.Review of gene therapies for age-related macular degeneration.Eye. 2022;36(2):303-311. doi:10.1038/s41433-021-01842-1
American Academy of Ophthalmology.Gene therapy for AMD.
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