# Exploring Alternative iPS Cell Therapies for Recessive Dystrophic Epidermolysis Bullosa

> **NIH NIH U01** · UNIVERSITY OF COLORADO DENVER · 2020 · $419,231

## Abstract

Project Summary
 No effective treatments are available for epidermolysis bullosa (EB), a group of rare inherited skin
blistering disorders that can be devastating, and in some cases lethal. The ability to reprogram adult skin cells
into induced pluripotent stem cells (iPSC) now offers the possibility of developing a permanent corrective
therapy for EB. Our advances in developing safer and more efficient reprogramming, gene editing and iPSC
differentiation protocols allowed us to establish the “EB iPS Cell Consortium” comprised of the University of
Colorado (Drs. Roop, Bilousova, Kogut and Bruckner), Stanford University (led by Dr. Oro) and Columbia
University (led by Dr. Christiano). The Consortium is currently developing an iPSC-based therapy for the
severe recessive dystrophic form of EB (RDEB) that is comprised of gene-corrected epidermal sheets and
composite skin grafts. The current application will explore two novel iPSC therapies for RDEB, and the non-
profit foundations that currently sponsor the Consortium, the EB Research Partnership, the EB Medical
Research Foundation and the Cure EB Charity, have agreed to provide the required 1 to 1 matching funds for
this RFA submission. Although skin grafts may be the fastest path forward to demonstrate safety and efficacy
of an iPSC-based therapy for RDEB patients, the time required to generate these grafts from genetically
corrected iPSCs is lengthy and consequently expensive. Therefore, we are proposing to evaluate a “spray-on-
skin” delivery system developed by Avita Medical, for delivering skin cells differentiated from gene-edited
RDEB iPSCs as a more straightforward alternative to skin grafts. If successful, the “spray-on-skin” delivery
system would decrease the time to patient application vs. the time and cost it takes to grow epidermal and
composite grafts and would potentially produce superior outcomes for EB patients due to the lower risk of
inflammation and scarring. In addition, while gene-corrected iPSC-derived keratinocytes may correct the
cutaneous phenotype in RDEB patients, these cells will not be effective in treating the severe gastrointestinal
manifestations associated with RDEB, which often require the use of feeding tubes. Therefore, we will also
assess if the systemic delivery of gene-corrected-iPSC-derived mesenchymal stem cells (MSCs) can facilitate
wound healing in both internal epithelia and the skin. If effective, the systemic delivery of MSCs would not only
revolutionize how we treat EB patients, but also potentially represent a novel approach to treat many systemic
multifocal diseases and injuries that affect internal organs and tissues. The following aims are proposed: Aim 1
will generate gene-corrected iPSC-derived fibroblasts and MSCs under cGMP-compliant conditions. Aim 2 will
explore the feasibility of using the “spray on skin” device to deliver gene-corrected iPSC-derived keratinocytes
and fibroblasts. Aim 3 will assess the ability of systemically delivered MSCs ...

## Key facts

- **NIH application ID:** 9986660
- **Project number:** 5U01AR075932-02
- **Recipient organization:** UNIVERSITY OF COLORADO DENVER
- **Principal Investigator:** Ganna Bilousova
- **Activity code:** U01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $419,231
- **Award type:** 5
- **Project period:** 2019-08-01 → 2023-06-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/9986660

## Citation

> US National Institutes of Health, RePORTER application 9986660, Exploring Alternative iPS Cell Therapies for Recessive Dystrophic Epidermolysis Bullosa (5U01AR075932-02). Retrieved via AI Analytics 2026-05-26 from https://api.ai-analytics.org/grant/nih/9986660. Licensed CC0.

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