mRNA vaccine shows promise for treating age-related macular degeneration
An mRNA vaccine developed by researchers from Japan suppressed abnormal blood vessel growth or neovascularization in the retina of mouse models. Neovascularization is a condition that is caused by age-related macular degeneration (AMD), a leading cause of vision loss for elderly people. The vaccine can be delivered intramuscularly and is as effective as current therapies that require frequent eye injections, offering a more comfortable and easier-to-administer alternative for treating AMD and other neovascular eye diseases.
One of the leading causes of vision loss in people over 60 years is age-related macular degeneration (AMD), a condition that affects nearly 200 million people worldwide. One form of the disease, known as wet AMD, is caused by the growth of abnormal vessels in the eye, a condition known as neovascularization.
These blood vessels leak fluid buildup in the retina, which
gradually leads to vision loss, if left untreated. At present, the only way to
slow this process is through regular injections of anti-angiogenic drugs that
stop the formation of blood vessels directly into the eye. However, these must
be taken regularly, and a few patients stop responding to the treatment.
Now, researchers at Institute of Science Tokyo (Science Tokyo), Japan, have developed a novel mRNA vaccine that can be injected intramuscularly, making it easier to administer and less burdensome on patients than current treatments that require direct injections into the eye. The vaccine was tested in mice and shown to strongly suppress retinochoroidal neovascularization, offering hope to millions of patients with AMD.
The research team was led by Professor Satoshi Uchida of the
Department of Advanced Nanomedical Engineering, Science Tokyo, and Visiting
Professor Yasuo Yanagi of the Department of Ophthalmology and Micro-technology,
Yokohama City University, Japan. The findings were made available online on
July 02, 2025, and published in Volume 61 of the journal Vaccine on
August 13, 2025.
“The COVID-19 pandemic highlighted the remarkable potential
of mRNA as a vaccine platform. Inspired by those successes, we sought to expand
its use beyond infectious diseases and cancer to chronic eye conditions. To our
knowledge, this is the first study to show that an mRNA vaccine can suppress
pathological neovascularization in animal models,” says Uchida.
Typically, mRNA vaccines carry instructions that enable the
body to produce antibodies against infectious agents. In this case, the vaccine
delivers mRNA encoding leucine-rich alpha-2-glycoprotein 1 (LRG1), a protein
known to promote angiogenesis and found in elevated levels in patients with
AMD. As a result, the body produces antibodies that specifically bind to and
inhibit LRG1.
The researchers tested the vaccine on two mouse models of
eye disease: one in which choroidal neovascularization (CNV) was induced, and
another that naturally develops CNV. After just two intramuscular injections
given 14 days apart, both models showed strong antibody responses that
significantly reduced abnormal blood vessel growth in the retina. The effects
were visible within a week of the first dose. By the 21st day,
the laser-induced CNV model showed an 85% reduction in leakage and an 82%
reduction in lesion size. In the natural NV model, lesion size decreased by 55%
on the 28th day.
Crucially, the vaccine appeared safe. It did not interfere
with normal blood vessel growth, damage healthy retinal tissue, or trigger
harmful immune reactions in other organs of the mice. At the same time, the
treatment was as effective as standard anti-vascular endothelial growth factor
(VEGF) drugs, yet free from their major drawbacks.
“The effects of LRG1 mRNA vaccination on reducing
endothelial and microglial cells were comparable to those of anti-VEGF antibody
therapy. Unlike conventional treatments requiring repeated intravitreal
injections, this vaccine may provide long-term benefits with a single
intramuscular dose, potentially reducing the treatment burden for patients,”
says Uchida.
If proven effective in humans, this vaccine could replace
the frequent, uncomfortable eye injections that patients with AMD currently
face, with a simple arm injection.