University of Colorado Anschutz Medical School

University of Colorado – Dr. Dennis R. Roop

“Study of the Safety, Tolerability, and Efficacy of an iPS Cell-based Therapy for  

Recessive Dystrophic Epidermolysis Bullosa Delivered with a Spray on Skin Device”

The proposal is for a clinical trial to assess the safety, tolerability and efficacy of delivering genetically corrected iPS cell-derived keratinocytes and fibroblasts with

AVITA Medical’s Spray-on-Skin device, which received approval from the FDA for the treatment of severe burns on September 21, 2018.

This is a multi investigator application, in which Dr. Roop will have primary responsibility for working with staff at the Gate Biomanufacturing Facility to ensure that

cGMP compliant standards will be used for the following: RNA production, gene editing and reprogramming of patient fibroblasts into iPS cells, production of gene

modified keratinocytes and fibroblasts and ultimately, selection and final product formulation. Dr. Anna L. Bruckner will have primary responsibility for designing, implementing,

and conducting the clinical trial at Children’s Hospital Colorado. Her activities include finalizing the study design, overseeing the study start-up process and regulatory compliance,

patient evaluations, patient treatment, and data analysis.

The EBMRF has previously provided partial matching funds for the first two rounds of NIH/NIAMS competitive funding awarded to the University of Colorado team from the

Regenerative Medicine Innovation Project that was made possible through the 21st Century Cures Act. The EBMRF matching funds (along with other Foundations) are required

by the Regenerative Medicine Innovation Project funding mechanism, in order for the Department of Health & Human Services (NIH) to fund the Federal Award #UG3AR082887.