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Clinical Research in Regenerative Ophthalmology

Researchers around the world are searching for answers to eye diseases that still have no cure. This page connects each condition to the official trial registry — and to our plain-language pages explaining the condition, the science being tested and the biology behind it — so you can understand what a trial is studying before you talk to your doctor.

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.

Where science and technology come together

Regenerative ophthalmology sits where several fields meet. Stem cell biology lets laboratories grow retinal cells such as RPE cells from induced pluripotent stem cells. Research on extracellular vesicles and exosomes studies how cells send signals to one another. High-resolution retinal imaging lets researchers measure small structural changes, and surgical engineering makes it possible to place cells precisely in the eye.

Each of these advances is tested step by step: first in the laboratory, then in animal studies, then in small human safety studies, and only then in larger controlled trials. Most approaches do not complete that path. How we grade the evidence →

Trials by condition

For each condition: what it is, the research topics and biological mechanisms researchers are investigating, and live searches of ClinicalTrials.gov. Each title links to the full page.

Age-Related Macular Degeneration Retina

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.

Why researchers are studying it: 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 topics
  • Stem-Cell-Derived RPE Cells — Retinal pigment epithelial cells grown from pluripotent stem cells — one of the most studied cell-replacement approaches in retinal research.
  • Induced Pluripotent Stem Cells (iPSCs) — Adult cells reprogrammed to a pluripotent state, used in research to derive retinal cells and to model inherited eye disease.
  • Mesenchymal Stromal/Stem Cells (MSCs) — What MSCs are, where they come from, and why researchers study their signaling and immune-modulating activity in the eye.
  • Extracellular Vesicles — Membrane-bound particles released by cells that carry proteins, lipids and RNA — studied as messengers in cell-to-cell signaling.
Biology being studied
  • Cell Replacement — Research on replacing lost cells, such as RPE or photoreceptors, with laboratory-derived cells.
  • Angiogenesis — The growth of new blood vessels — central to wet AMD and diabetic eye disease.
  • Oxidative Stress — Cellular damage from reactive oxygen species, studied in retinal aging and degeneration.
  • Ocular Inflammation — How immune signaling contributes to eye disease, and research on modulating it.
Search registered trials (ClinicalTrials.gov): Macular Degeneration + stem cell · Macular Degeneration + retinal pigment epithelium
Why we link here: it is the official U.S. registry, so results are always current. A listing is not an endorsement.

Stargardt Disease Retina

Stargardt disease is an inherited macular dystrophy, most often caused by variants in the ABCA4 gene. It typically begins in childhood or early adulthood and causes progressive loss of central vision.

Why researchers are studying it: Because Stargardt disease involves loss of RPE cells and photoreceptors, it has been included in cell-replacement research alongside AMD.

Research topics
  • Stem-Cell-Derived RPE Cells — Retinal pigment epithelial cells grown from pluripotent stem cells — one of the most studied cell-replacement approaches in retinal research.
  • Stem Cell Research — An overview of the stem and progenitor cell types researchers study in vision science — what they are, how they differ, and how far the research has progressed.
Biology being studied
  • Cell Replacement — Research on replacing lost cells, such as RPE or photoreceptors, with laboratory-derived cells.
  • Oxidative Stress — Cellular damage from reactive oxygen species, studied in retinal aging and degeneration.
Search registered trials (ClinicalTrials.gov): Stargardt Disease + stem cell
Why we link here: it is the official U.S. registry, so results are always current. A listing is not an endorsement.

Retinitis Pigmentosa & Inherited Retinal Diseases Retina

Retinitis pigmentosa (RP) is a group of inherited retinal diseases in which photoreceptors gradually degenerate, typically starting with night vision and peripheral vision.

Why researchers are studying it: Because photoreceptors are lost, researchers are investigating whether progenitor cells could support remaining photoreceptors or replace lost ones, and how to protect remaining cells.

Research topics
Biology being studied
  • Neuroprotection — Research on protecting retinal neurons and retinal ganglion cells from degeneration.
  • Cell Replacement — Research on replacing lost cells, such as RPE or photoreceptors, with laboratory-derived cells.
Search registered trials (ClinicalTrials.gov): Retinitis Pigmentosa + retinal progenitor · Retinitis Pigmentosa + stem cell
Why we link here: it is the official U.S. registry, so results are always current. A listing is not an endorsement.

Diabetic Retinopathy & Diabetic Macular Edema Retina

Diabetic retinopathy is damage to the retina's blood vessels caused by diabetes. Diabetic macular edema (DME) is swelling of the macula from leaking vessels and is a common cause of vision loss in people with diabetes.

Why researchers are studying it: Researchers are interested in whether cell-derived signals could reduce inflammation or support damaged retinal vessels and neurons.

Research topics
  • Mesenchymal Stromal/Stem Cells (MSCs) — What MSCs are, where they come from, and why researchers study their signaling and immune-modulating activity in the eye.
  • Extracellular Vesicles — Membrane-bound particles released by cells that carry proteins, lipids and RNA — studied as messengers in cell-to-cell signaling.
Biology being studied
  • Angiogenesis — The growth of new blood vessels — central to wet AMD and diabetic eye disease.
  • Ocular Inflammation — How immune signaling contributes to eye disease, and research on modulating it.
  • Oxidative Stress — Cellular damage from reactive oxygen species, studied in retinal aging and degeneration.
Search registered trials (ClinicalTrials.gov): Diabetic Retinopathy + stem cell · Diabetic Retinopathy + exosome
Why we link here: it is the official U.S. registry, so results are always current. A listing is not an endorsement.

Glaucoma Optic Nerve

Glaucoma is a group of diseases that damage the optic nerve, often — but not always — associated with elevated eye pressure. Vision loss usually begins in the periphery and is permanent.

Why researchers are studying it: Because glaucoma vision loss comes from the death of retinal ganglion cells, researchers study ways to protect these cells (neuroprotection) and, much further off, to replace them.

Research topics
  • Mesenchymal Stromal/Stem Cells (MSCs) — What MSCs are, where they come from, and why researchers study their signaling and immune-modulating activity in the eye.
  • Extracellular Vesicles — Membrane-bound particles released by cells that carry proteins, lipids and RNA — studied as messengers in cell-to-cell signaling.
  • Stem Cell Research — An overview of the stem and progenitor cell types researchers study in vision science — what they are, how they differ, and how far the research has progressed.
Biology being studied
  • Neuroprotection — Research on protecting retinal neurons and retinal ganglion cells from degeneration.
  • Cell Replacement — Research on replacing lost cells, such as RPE or photoreceptors, with laboratory-derived cells.
Search registered trials (ClinicalTrials.gov): Glaucoma + stem cell
Why we link here: it is the official U.S. registry, so results are always current. A listing is not an endorsement.

Optic Nerve Damage & Optic Neuropathy Optic Nerve

Optic neuropathy is damage to the optic nerve, which carries visual information from the eye to the brain. Causes include reduced blood flow (ischemic optic neuropathy), inflammation (optic neuritis), trauma, compression and inherited conditions.

Why researchers are studying it: Optic nerve regeneration is one of the most difficult problems in neuroscience; researchers study neuroprotection and axon regeneration in laboratory and animal models.

Research topics
  • Mesenchymal Stromal/Stem Cells (MSCs) — What MSCs are, where they come from, and why researchers study their signaling and immune-modulating activity in the eye.
  • Extracellular Vesicles — Membrane-bound particles released by cells that carry proteins, lipids and RNA — studied as messengers in cell-to-cell signaling.
Biology being studied
  • Neuroprotection — Research on protecting retinal neurons and retinal ganglion cells from degeneration.
Search registered trials (ClinicalTrials.gov): Optic Neuropathy + stem cell
Why we link here: it is the official U.S. registry, so results are always current. A listing is not an endorsement.

Limbal Stem Cell Deficiency & Corneal Damage Cornea & Ocular Surface

Limbal stem cell deficiency (LSCD) occurs when the stem cells at the edge of the cornea (the limbus) are damaged or lost, so the corneal surface can no longer renew itself normally. Chemical and thermal burns are common causes.

Why researchers are studying it: Limbal stem cells are the eye's own corneal-surface stem cells — one of the few areas where stem-cell-based approaches have reached established clinical use in some jurisdictions.

Research topics
  • Limbal Stem Cells — The stem cells that maintain the corneal surface; the basis of established surgical approaches for limbal stem cell deficiency.
  • Mesenchymal Stromal/Stem Cells (MSCs) — What MSCs are, where they come from, and why researchers study their signaling and immune-modulating activity in the eye.
Biology being studied
  • Cell Replacement — Research on replacing lost cells, such as RPE or photoreceptors, with laboratory-derived cells.
  • Ocular Inflammation — How immune signaling contributes to eye disease, and research on modulating it.
Search registered trials (ClinicalTrials.gov): Limbal Stem Cell Deficiency + limbal
Why we link here: it is the official U.S. registry, so results are always current. A listing is not an endorsement.

Dry Eye Disease Cornea & Ocular Surface

Dry eye disease is a common condition in which the tear film does not adequately lubricate the ocular surface, causing discomfort, irritation and fluctuating vision.

Why researchers are studying it: Because inflammation drives many forms of dry eye, researchers have studied the immune-modulating properties of MSCs and their secreted factors, including in dry eye related to graft-versus-host disease.

Research topics
  • Mesenchymal Stromal/Stem Cells (MSCs) — What MSCs are, where they come from, and why researchers study their signaling and immune-modulating activity in the eye.
  • Extracellular Vesicles — Membrane-bound particles released by cells that carry proteins, lipids and RNA — studied as messengers in cell-to-cell signaling.
Biology being studied
  • Ocular Inflammation — How immune signaling contributes to eye disease, and research on modulating it.
  • Cell Signaling — Paracrine signaling, growth factors, cytokines and microRNA — how cells communicate.
Search registered trials (ClinicalTrials.gov): Dry Eye + mesenchymal · Dry Eye + exosome
Why we link here: it is the official U.S. registry, so results are always current. A listing is not an endorsement.

Uveitis & Ocular Inflammation Inflammation

Uveitis is inflammation inside the eye, affecting the uvea and sometimes the retina. It can be associated with autoimmune disease, infection, or occur without an identified cause, and can threaten vision.

Why researchers are studying it: MSCs are studied for their immune-modulating signaling, which has led to animal research in models of autoimmune uveitis.

Research topics
Biology being studied
  • Ocular Inflammation — How immune signaling contributes to eye disease, and research on modulating it.
Search registered trials (ClinicalTrials.gov): Uveitis + mesenchymal
Why we link here: it is the official U.S. registry, so results are always current. A listing is not an endorsement.

The research topics behind these trials

What each approach is, explained in plain language, and why it appears on EyeCyte.

The biology researchers are targeting

Legitimate trial or unapproved clinic?

  • A legitimate trial is registered (for example on ClinicalTrials.gov), overseen by an institutional review board, and — for most investigational products in the U.S. — conducted under FDA oversight.
  • Trials do not usually charge participants for the investigational product. Be cautious of clinics charging large fees for unapproved procedures.
  • Registration alone does not guarantee quality; discuss any trial with your own eye-care professional.
  • The FDA has warned the public about unapproved stem cell and exosome products. Read our medical disclaimer →

Frequently asked questions about clinical trials

What is a clinical trial?

A clinical trial is a research study in people that tests whether an investigational approach is safe and whether it works. Trials move in phases, from small safety studies to larger studies that compare an approach with standard care. Most approaches studied in early trials never become approved treatments.

Does a registered trial mean a treatment works?

No. Registration on ClinicalTrials.gov means a study has been publicly described, not that the approach has been shown to be safe or effective. A registered trial is not an endorsement by EyeCyte or by the FDA.

How can I tell a legitimate trial from an unapproved clinic?

Legitimate trials are registered, reviewed by an institutional review board, and — for most investigational products in the U.S. — run under FDA oversight. They do not usually charge participants for the investigational product. Be cautious of any clinic charging large fees for stem cell or exosome procedures for the eye.

Why does EyeCyte link to ClinicalTrials.gov instead of listing trials?

Trials open, close and change constantly. ClinicalTrials.gov, run by the U.S. National Library of Medicine, is the official public registry, so linking to a live search there is more accurate than any list we could maintain.

Should I join a clinical trial?

That is a decision to make with your own eye-care professional, who knows your diagnosis and can help you weigh the possible benefits and risks of a specific study.

All frequently asked questions → · Browse every answer → · Find an eye-care professional →

Clinical Research in Regenerative Ophthalmology | EyeCyte