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



Editing of RDEB fibroblasts

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

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Rigosertib for RDEB SCC

Rigosertib for recessive dystrophic epidermolysis bullosa-associated squamous cell carcinoma

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Base editing of RDEB fibroblasts

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

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

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Gene therapy for airway disease

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

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RDEB gene-edited skin

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

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

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Development of a topical gel formulation of decorin to reduce fibrosis

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Mission EB

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

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RNA Derm

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

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