Our research focuses on the clinical development of novel therapeutics and clinical trials for rare monogenic diseases: Epidermolysis Bullosa and Basal Cell Nevus Syndrome. We use pre-clinical mouse models, epidemiology, genomics and investigator-initiated clinical trials to identify new treatments for our patients.
We collaborate with Dr. Peter Marinkovich, Dr. Albert Chiou, Dr. Joyce Teng, and Dr. Anthony Oro in our work on Epidermolysis Bullosa (EB). We focus on molecular correction of the disease and are currently conducting several clinical trials and research studies. We are also investigating therapeutics for itch associated with EB. We continue to collaborate with EB researchers across the country and internationally to improve the lives of patients with this devastating disease.
Phase 1/2a clinical trial of gene-corrected autologous cell therapy for recessive dystrophic epidermolysis bullosa. Eichstadt, S., Barriga, M. et al. (2019). JCI insight
Inhibition of the hedgehog pathway in patients with basal-cell nevus syndrome: final results from the multicentre, randomised, double-blind, placebo-controlled, phase 2 trial Tang, J. Y., Ally, M. S. et al. (2016). Lancet Oncology
Safety and Wound Outcomes Following Genetically Corrected Autologous Epidermal Grafts in Patients With Recessive Dystrophic Epidermolysis Bullosa. Siprashvili, Z., Nguyen, N.T., et al. (2016) JAMA
Inhibiting the Hedgehog Pathway in Patients with the Basal-Cell Nevus Syndrome Tang, J.Y., Mackay-Wiggan, J.M. et al. (2012). New England Journal of Medicine