Research Portfolio

We have been funding research since 2012. Explore our timeline of research projects below, or browse our Current or Completed project pages.

Gene therapy and Gene editing

Cell therapy

Drug therapy

Protein therapy

EB cancer therapy

Research

2023

Testing the in vivo efficacy of base and prime editing in primary fibroblasts and in a 3D skin model of dystrophic epidermolysis bullosa

Rigosertib for recessive dystrophic epidermolysis bullosa-associated squamous cell carcinoma

2022

Testing the efficacy of genomic base editing in dystrophic epidermolysis bullosa primary fibroblasts

Development of a topical gene-editing treatment to restore Collagen VII

2021

Combined respiratory epithelial cell and gene therapy for amelioration of respiratory symptoms in children with Junctional Epidermolysis Bullosa

Pre-clinical and clinical trial for using gene-edited skin in Recessive Dystrophic Epidermolysis Bullosa

A nanoneedle bandage for topical intracellular COL7A1 gene therapy to accelerate wound healing in Recessive Dystrophic Epidermolysis Bullosa

Development of a topical gel formulation of decorin to reduce fibrosis

2020

Double blind placebo controlled study of mesenchymal intravenous stromal cell infusions in children with Recessive Dystrophic Epidermolysis Bullosa

2019

Development of IV-administered siRNAs targeting mutant KRT5 and KRT1 in EB Simplex