Grant Award Details

A universally applicable skin sheet for Dystrophic Epidermolysis Bullosa via next-generation gene editing, iPS cell technology and tissue engineering
Grant Number: 
DISC2-12590
Project Objective: 
  • To develop an autologous, gene corrected iPSC-derived skin sheet for treating RDEB patients with any mutation(s) in the COL7A1 gene
Investigator: 
Institution: 
Type: 
PI
Disease Focus: 
Epidermolysis Bullosa
Skin Disease
Human Stem Cell Use: 
iPS Cell
Award Value: 
$1,229,040
Status: 
Pre-Active

Grant Application Details

Application Title: 
  • A universally applicable skin sheet for Dystrophic Epidermolysis Bullosa via next-generation gene editing, iPS cell technology and tissue engineering
Public Abstract: 

Research Objective

We will develop a cell therapy for a rare skin disease. Patient-derived iPS cells will be genetically corrected and differentiated into epithelial sheets to be grafted on skin wounds.

Impact

In this proposal we will develop a universal genetic correction strategy for all COL7A1 which will be a prerequisite for the commercial viability of our iPS cell-based cell therapy.

Major Proposed Activities

  • Replacing a medium size fragment of the COLLAGEN7A1 locus
  • Excision of the entire COLLAGEN7A1 locus
  • Replacement of the entire COLLAGEN7A1 locus with a normal copy
  • Differentiate corrected iPS cells into skin cells
  • Develop a clinical-grade cell purification system for skin cells
  • Verify that manufactured skin cells are functional.
Statement of Benefit to California: 

Our ultimate goal is to bring our iPSC-based therapy into the clinic. Our product will have to be commercially viable to ensure sufficient funding through Phase III clinical testing, FDA approval, and production at scale to provide it to the entire patient community. This therapy will directly benefit DEB patients in California. More Californians will benefit from future therapies based on our platform. Finally, academic and commercial development will benefit California's economy.