# Chimeric antigen receptor (CAR) Tregs for treatment of ocular inflammatory diseases

> **NIH NIH R21** · UNIV OF MASSACHUSETTS MED SCH WORCESTER · 2024 · $209,375

## Abstract

Project Summary
 Two million uveitis new cases per year occur in the United States. The current treatment strategy is to
suppress the immune system with corticosteroids or other medications that have serious side effects including
an increased susceptibility to infection. After a patient achieves remission for one to two years, a slow taper of
the medications can be attempted, and some patients can be completely tapered off medications and maintain
durable remission. There is a strong correlation between the induction of regulatory T cells (Tregs) and
achieving durable remission. Unfortunately, the current medications used to treat uveitis do not induce Tregs.
Therefore, treatments that target Treg induction or a cell-based approach to delivery Tregs to a patient
represents a novel therapy with lasting benefits. Chimeric antigen (CAR) T cells that target B cells have been
used to treat B cell lymphoma, and more recently for the treatment of B cell mediated autoimmune diseases,
such as systemic lupus erythematosus. CAR Treg cells have been designed to prevent an immune response
against adeno associated virus (AAV), thus allowing for more effective gene therapy. We propose to
demonstrate that CAR Tregs are effective in suppressing autoimmune uveitis. Our central hypothesis is that
CAR Tregs effectively suppresses ocular inflammation. We have a unique opportunity to evaluate and develop
ocular CAR Tregs with the experience in the Lee lab with experimental autoimmune uveitis (EAU), and the
Keeler lab that has experience developing CAR Tregs. The Keeler lab has AAV-specific CAR Tregs on hand
and has shown these are effective at suppressing inflammation. Therefore, we will determine if administration
of AAV-specific CAR Tregs in combination with an intraocular injection of AAV is effective in suppressing EAU
(Aim 1). We will also generate ocular antigen-specific CAR Tregs and evaluate the efficacy of these Tregs to
suppress EAU. Completion of this project will provide the foundation for developing a novel therapeutic for
autoimmune uveitis and could further be applied to any ocular disease with an inflammatory component.

## Key facts

- **NIH application ID:** 10871366
- **Project number:** 1R21EY036212-01
- **Recipient organization:** UNIV OF MASSACHUSETTS MED SCH WORCESTER
- **Principal Investigator:** Darren James Lee
- **Activity code:** R21 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2024
- **Award amount:** $209,375
- **Award type:** 1
- **Project period:** 2024-05-01 → 2026-04-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10871366

## Citation

> US National Institutes of Health, RePORTER application 10871366, Chimeric antigen receptor (CAR) Tregs for treatment of ocular inflammatory diseases (1R21EY036212-01). Retrieved via AI Analytics 2026-05-25 from https://api.ai-analytics.org/grant/nih/10871366. Licensed CC0.

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