# Novel Cell Free Therapy for Glaucoma

> **NIH NIH R21** · UNIVERSITY OF ILLINOIS AT CHICAGO · 2020 · $249,661

## Abstract

A principal unmet need in glaucoma is that medical or surgical intraocular pressure reduction is the only clin-
ically-approved treatment. But vision cannot be restored because retinal ganglion cell (RGC) loss is irre-
versible. Glaucoma, the 2nd leading cause of worldwide blindness, afflicting greater than 60M, is character-
ized by RGC loss, and optic nerve defects. Knowledge gaps to improved therapy include how to achieve
neuroprotection, i.e., preventing RGCs from dying, or, to use cell-based therapy to re-grow or replace. Ad-
ministration route, dosage, and adverse effects limit clinical application of neuroprotection and cell trans-
plantation. Studying a new treatment using extracellular vesicles (EVs), biologically-active 50-150 nm di-
ameter nanoparticles derived from stem cells, the proposal fills a gap and urgent need in the development of
new treatments to prevent RGC death and vision loss for glaucoma patients. Their stability, biocompatibility,
biological barrier permeability, and low toxicity render EVs attractive vehicles as delivery therapies to the
eye. EVs represent a potential clinically applicable means to prevent RGC, axonal, and visual functional loss
and decreasing the excitotoxic and inflammatory component of glaucoma. Our central hypothesis is that
EVs can be designed and optimized to specifically target RGCs as a basis for precision treatment of glau-
coma and, ultimately, other retinal diseases. We propose to test engineered EVs as a novel cell-free means
to specifically target neuroprotection to RGCs and to fill the knowledge gaps that presently prevent clinical
translation of EVs for retinal disease. Our specific aims are Aim 1: Determine the time course and factors
regulating the distribution of EVs in the vitreous and retina, and optimize EV delivery to retina. Aim
2: Develop and optimize novel engineered EVs to specifically target RGCs. Successful completion of
Aim 1 will optimize delivery of EVs to the retina following intravitreal injection. Aim 2 will guide administration
of EVs to produce innovative, specific, targeted delivery into RGCs, allowing specificity for treatment at the
major pathophysiological site of glaucoma. Fulfillment of these objectives will set the stage to develop glau-
coma therapeutics using EVs by optimizing administration, and by specific RGC-targeted EVs. The study of-
fers promise to save sight via development of safe, effective, and cost-efficient therapy to restore or prevent
loss of sight in patients with glaucoma. This contribution is expected to be significant because these studies
will provide a basis to develop EVs as a therapy for glaucoma, either primarily restoring RGC function and
axonal growth, or optimizing existing therapies such as RGC transplants. Innovative features of this work
are cell-free therapy of glaucoma, novel targeted EVs binding RGC-specific receptors for specific RGC ac-
tion, and novel delivery materials for EVs. RO1 applications are expected to follow, to...

## Key facts

- **NIH application ID:** 9853796
- **Project number:** 5R21EY028690-02
- **Recipient organization:** UNIVERSITY OF ILLINOIS AT CHICAGO
- **Principal Investigator:** STEVEN ROTH
- **Activity code:** R21 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $249,661
- **Award type:** 5
- **Project period:** 2019-02-01 → 2022-01-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/9853796

## Citation

> US National Institutes of Health, RePORTER application 9853796, Novel Cell Free Therapy for Glaucoma (5R21EY028690-02). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/9853796. Licensed CC0.

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