Research Portfolio
Key
Gene therapy and 
Gene editing
Cell therapy
Drug therapy
Protein therapy
EB cancer therapy
Research
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
NanoCol
A nanoneedle bandage for topical intracellular COL7A1 gene therapy to accelerate wound healing in Recessive Dystrophic Epidermolysis Bullosa
Exon skipping approach to RDEB
Exon skipping approach to Recessive Dystrophic Epidermolysis Bullosa
FIBRX Derm
Development of a topical gel formulation of decorin to reduce fibrosis