Age-Related Macular Degeneration and Regenerative Medicine Research

Last medically reviewed: October 7, 2026

⚠ Important — educational information only, not medical advice

EyeCyte provides educational and scientific information and does not provide medical advice, diagnosis, or treatment. Emerging regenerative and biologic technologies discussed on this website may be experimental, investigational, unapproved, or supported only by preclinical evidence. Patients should discuss medical decisions with appropriately licensed healthcare professionals.
Not an FDA-approved treatment unless explicitly stated. Except where a page names an FDA-approved product, the stem-cell, MSC, exosome, extracellular-vesicle, Wharton’s jelly and other biologic approaches described here are not FDA-approved for any eye condition. Research findings — especially laboratory and animal studies — are not proof that anything works or is safe in people.
Research-Use-Only (RUO): Research-Use-Only (RUO) materials referenced on EyeCyte are for laboratory research by qualified professionals only. They are not drugs, biologics or devices approved for human use, are not intended for administration to people, and are not intended to diagnose, treat, cure, mitigate or prevent any disease.
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What is age-related macular degeneration?

Age-related macular degeneration (AMD) affects the macula, the central part of the retina responsible for sharp, detailed central vision. It is a leading cause of vision loss in older adults. AMD is usually described as dry (atrophic) — including its advanced form, geographic atrophy — or wet (neovascular), in which abnormal blood vessels grow beneath the retina.

What causes age-related macular degeneration?

AMD develops from a combination of age, genetics and environmental factors such as smoking. Researchers study the roles of the retinal pigment epithelium (RPE), the complement system, chronic inflammation, oxidative stress and abnormal blood-vessel growth.

What structures or cells are affected?

The retinal pigment epithelium (a supporting cell layer), the photoreceptors that sense light, Bruch's membrane, and — in wet AMD — the choroidal blood vessels.

How is age-related macular degeneration currently treated or managed?

● Established / Approved

Regular monitoring by an eye-care professional, including home monitoring of central vision when advised.
For intermediate AMD, specific nutritional supplement formulations studied by the National Eye Institute (AREDS/AREDS2) may be recommended by a clinician.
For wet AMD, intravitreal anti-VEGF injections are established care.
Low-vision rehabilitation and assistive devices.

Are these approaches FDA approved for age-related macular degeneration?

● Established / Approved Anti-VEGF medicines are approved for wet AMD.^[1]

● Established / Approved In 2023 the FDA approved the first treatment for geographic atrophy (pegcetacoplan); a second complement inhibitor (avacincaptad pegol) was also approved that year.^[2]

No stem cell, MSC, exosome or Wharton’s jelly product is FDA-approved to treat AMD, to our knowledge as of our last review. Confirm current status on the FDA's list of approved cellular and gene therapy products.^[9]

Why are researchers studying regenerative approaches?

Because AMD involves the loss of RPE cells and, later, photoreceptors, researchers are investigating whether laboratory-grown RPE cells could replace lost cells, and whether cell-derived signals could support the remaining retina.

Research is not treatment. The studies summarized below are investigational. They are presented for education only, do not show that any approach is safe or effective in patients, and are not a recommendation. Unless stated as FDA-approved, none of these approaches is approved for this condition. RUO materials are not for human use.

Stem Cell Research and Age-Related Macular Degeneration

● Early Human ResearchStem-Cell-Derived RPE Cells
Early-phase human studies have transplanted RPE cells derived from human embryonic stem cells in people with advanced AMD. These studies were designed mainly to evaluate safety and feasibility in small numbers of participants.^{[3, 4, 5]}
● Early Human ResearchInduced Pluripotent Stem Cells (iPSCs)
A widely cited report described transplantation of RPE cells derived from a patient's own induced pluripotent stem cells in a single person with neovascular AMD. It is a case report, not evidence of benefit in a population.^[6]

MSC Research and Age-Related Macular Degeneration

● Insufficient EvidenceMesenchymal Stromal/Stem Cells (MSCs)
There is no established evidence that injecting MSCs or other "stem cell" preparations into the eye benefits people with AMD. A published case series described severe, permanent vision loss after intravitreal injection of autologous cell preparations at a clinic outside a regulated trial.^{[7, 8]}

Extracellular Vesicle / Exosome Research and Age-Related Macular Degeneration

● Laboratory / In Vitro EvidenceExtracellular Vesicles
Laboratory and animal studies have explored extracellular vesicles in models of retinal degeneration and RPE stress. This research is preliminary and has not established a clinical use.
EVIDENCE VERIFICATION REQUIRED. Specific studies have not yet been verified by our reviewers. Search the literature: PubMed search: extracellular vesicles retinal pigment epithelium age-related macular degeneration

Other Cell-Based Research

We have not identified research in this category for age-related macular degeneration that meets our standards for inclusion.

What Has Been Demonstrated in Laboratory Research?

● Laboratory / In Vitro EvidenceExtracellular Vesicles
Laboratory and animal studies have explored extracellular vesicles in models of retinal degeneration and RPE stress. This research is preliminary and has not established a clinical use.
EVIDENCE VERIFICATION REQUIRED. Specific studies have not yet been verified by our reviewers. Search the literature: PubMed search: extracellular vesicles retinal pigment epithelium age-related macular degeneration

What Has Been Demonstrated in Animal Research?

We have not identified research in this category for age-related macular degeneration that meets our standards for inclusion.

What Human Research Exists?

● Early Human ResearchStem-Cell-Derived RPE Cells
Early-phase human studies have transplanted RPE cells derived from human embryonic stem cells in people with advanced AMD. These studies were designed mainly to evaluate safety and feasibility in small numbers of participants.^{[3, 4, 5]}
● Early Human ResearchInduced Pluripotent Stem Cells (iPSCs)
A widely cited report described transplantation of RPE cells derived from a patient's own induced pluripotent stem cells in a single person with neovascular AMD. It is a case report, not evidence of benefit in a population.^[6]

Are Clinical Trials Underway?

Trial status changes often. Search the official U.S. registry directly: Macular Degeneration + stem cell, Macular Degeneration + retinal pigment epithelium. A registered trial is not an endorsement, and registration does not mean a treatment works.

What Has NOT Yet Been Proven

● Insufficient Evidence

That any cell or biologic approach improves vision in people with AMD.
The long-term safety and durability of transplanted RPE cells.
That exosome or extracellular-vesicle products have any benefit in AMD.

Potential Biological Mechanisms Researchers Are Investigating

● Mechanistic / Theoretical

Cell Replacement Angiogenesis Oxidative Stress Ocular Inflammation

Risks and Scientific Unknowns

Unapproved cell and biologic procedures offered outside clinical trials have been associated with serious harm, including permanent vision loss.
Products sold as "stem cells" or "exosomes" vary widely in what they actually contain; composition, purity and potency are often not independently verified.
Results in cells and animals frequently do not carry over to people; many approaches that look promising in the laboratory never reach approval.

Questions to Ask a Qualified Eye Doctor

What type and stage of this condition do I have, and how is it likely to progress?
Which treatments are established for my situation, and what are their benefits and risks?
Are there clinical trials I might qualify for, and how would I verify a trial is legitimate?
If I have seen a clinic offering stem cell or exosome procedures for my eyes, what questions should I ask?

Find an Eye-Care Professional

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Information for Physicians and Researchers

Professional education, research summaries and research-use-only materials are on our professional pages — kept separate from patient information.

Professional resources For Providers — Get More Information →

Explore Age-Related Macular Degeneration

Research topics

Stem-Cell-Derived RPE Cells Induced Pluripotent Stem Cells (iPSCs)Mesenchymal Stromal/Stem Cells (MSCs)Extracellular Vesicles

Mechanisms

Cell Replacement Angiogenesis Oxidative Stress Ocular Inflammation

Answers

Frequently Asked Questions

Are stem cells being studied for macular degeneration?

Yes. Early-phase human studies have tested laboratory-grown RPE cells, mostly to assess safety. This is research, not an established treatment.

Is any stem cell treatment FDA approved for macular degeneration?

Not to our knowledge as of our last review. Established care includes anti-VEGF injections for wet AMD and, since 2023, approved complement inhibitors for geographic atrophy.

What is the difference between a clinical trial and a clinic offering stem cells?

A legitimate trial is registered, overseen by an ethics board and regulators, and does not typically charge participants for the investigational product. Clinics selling unapproved procedures may not have these protections.

Is any stem cell or exosome product FDA approved for age-related macular degeneration?

References

Eye Conditions and Diseases. National Eye Institute (NIH). [Government health agency · citation details pending reviewer confirmation]
FDA Approves First Treatment for Geographic Atrophy, a Leading Cause of Blindness (pegcetacoplan). U.S. Food and Drug Administration, 2023. [Regulator · citation details pending reviewer confirmation]
Schwartz SD, et al. Human embryonic stem cell-derived retinal pigment epithelium in patients with age-related macular degeneration and Stargardt's macular dystrophy: follow-up of two open-label phase 1/2 studies. The Lancet, 2015. [Peer-reviewed study · citation details pending reviewer confirmation]
da Cruz L, et al. Phase 1 clinical study of an embryonic stem cell–derived retinal pigment epithelium patch in age-related macular degeneration. Nature Biotechnology, 2018. [Peer-reviewed study · citation details pending reviewer confirmation]
Kashani AH, et al. A bioengineered retinal pigment epithelial monolayer for advanced, dry age-related macular degeneration. Science Translational Medicine, 2018. [Peer-reviewed study · citation details pending reviewer confirmation]
Mandai M, et al. Autologous Induced Stem-Cell–Derived Retinal Cells for Macular Degeneration. New England Journal of Medicine, 2017. [Peer-reviewed study · citation details pending reviewer confirmation]
Kuriyama AE, Albini TA, et al. Vision Loss after Intravitreal Injection of Autologous "Stem Cells" for AMD. New England Journal of Medicine, 2017. [Peer-reviewed study · citation details pending reviewer confirmation]
FDA Warns About Stem Cell Therapies (consumer update). U.S. Food and Drug Administration. [Regulator · citation details pending reviewer confirmation]
Approved Cellular and Gene Therapy Products. U.S. Food and Drug Administration. [Regulator · citation details pending reviewer confirmation]

Live searches (always current)

ClinicalTrials.gov: Macular Degeneration + stem cell — ClinicalTrials.gov (NIH National Library of Medicine)
ClinicalTrials.gov: Macular Degeneration + retinal pigment epithelium — ClinicalTrials.gov (NIH National Library of Medicine)
PubMed search: extracellular vesicles retinal pigment epithelium age-related macular degeneration — PubMed (NIH National Library of Medicine)