Research Portfolio
Key
Gene therapy and 
Gene editing
Cell therapy
Drug therapy
Protein therapy
EB cancer therapy
Research
Nonsense Tx
Nonsense Tx reading through disease causing nonsense codons
PASTE gene therapy in DEB
PASTE-Mediated Superexon Replacement of COL7A1 as a Treatment for Dystrophic Epidermolysis Bullosa
Spray-on-Skin
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
siRNA interference in EBS
siRNA-mediated allele-specific inhibition of mutant KRT5 and KRT14 transcripts carrying frequent dominant mutations in Epidermolysis Bullosa Simplex
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
Rigosertib for RDEB SCC
Rigosertib for recessive dystrophic epidermolysis bullosa-associated squamous cell carcinoma
Base editing of RDEB fibroblasts
Testing the efficacy of genomic base editing in dystrophic epidermolysis bullosa primary fibroblasts
BrB-101
Development of a topical gene-editing treatment to restore Collagen VII
Gene therapy for airway disease
Combined respiratory epithelial cell and gene therapy for amelioration of respiratory symptoms in children with Junctional Epidermolysis Bullosa
RDEB gene-edited skin
Pre-clinical and clinical trial for using gene-edited skin in Recessive Dystrophic Epidermolysis Bullosa