# Cell Therapy Program with Scale-up cGMP Manufacturing of Human Corneal Stromal Stem Cells

> **NIH NIH U01** · UNIVERSITY OF PITTSBURGH AT PITTSBURGH · 2024 · $507,420

## Abstract

RFA-HL-23-019 RMIP II-Collaborative U01. Contact PI - Gary Yam
Project Abstract
Corneal blindness due to corneal scarring affects millions of people worldwide. Corneal transplantation is
effective but has limitations. This Multi-PI project is aimed to establish a new stem cell-based approach to
reduce the need for corneal allograft transplantation. The scopes of work in this project are to validate and
confirm procedures to generate Good Manufacturing Practice (GMP) stem cells, verify the cell safety, and
analyze clinical data towards a cell-based therapy for corneal scarring disorders. The Yam Lab and the
Corneal Task Force at the University of Pittsburgh, following the innovative work by the late James L.
Funderburgh, have confirmed the regenerative effects of human corneal stromal stem cells (CSSC) in animal
models of corneal scarring. The Hsu Lab and the Immunologic Monitoring and Cellular Products Laboratory
(IMCPL) at UPMC Hillman Cancer Center have immense experience in establishing Investigation New Drugs
(IND) manufacturing procedures, quality assurance systems and documentation to support GMP cell product
manufacturing and processing for FDA-registered clinical trials. This proposal is a joint force of the two
expertise to pave a route to stem cell-based therapy in future clinical trials and clinical applications treating
corneal scarring. Specific Aim 1 will establish Chemistry and Manufacturing Control (CMC) for GMP grade
CSSC for allocation towards Investigation New Drugs (IND) in FDA. We will characterize and compare the
quality of GMP raised CSSC and lab-generated cells, and establish a complete GMP production protocol.
Specific Aim 2 will elucidate the pharmacological and toxicological effects of GMP-CSSC. We will study cell
safety and efficacy using our established mouse corneal injury model and will determine CSSC distribution,
metabolism, absorption, excretion, metabolism, and tumorigenicity. Specific Aim 3 will run clinical data mining
and analysis that will direct future clinical trial design. We will do retrospective data analysis using corneal
keratitis and scarring patient database at the UMPC Eye Center and obtain statistical data to direct future
clinical trial design on patient eligibility and treatment outcome analysis. This project is established based on
our solid basic and translational studies and data, and will lead to establish GMP-grade CSSC ready for clinical
trials and to design clinical trials for treating corneal blindness.

## Key facts

- **NIH application ID:** 10913577
- **Project number:** 5U01EY035252-02
- **Recipient organization:** UNIVERSITY OF PITTSBURGH AT PITTSBURGH
- **Principal Investigator:** Devanjan Sikder
- **Activity code:** U01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2024
- **Award amount:** $507,420
- **Award type:** 5
- **Project period:** 2023-09-01 → 2026-06-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10913577

## Citation

> US National Institutes of Health, RePORTER application 10913577, Cell Therapy Program with Scale-up cGMP Manufacturing of Human Corneal Stromal Stem Cells (5U01EY035252-02). Retrieved via AI Analytics 2026-05-29 from https://api.ai-analytics.org/grant/nih/10913577. Licensed CC0.

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