Bio
My research has focused on rare, monogenetic skin diseases such as Epidermolysis Bullosa (EB) and Gorlin Syndrome (Basal Cell Nevus Syndrome). With my PhD training in basic science, post-doctoral fellowship in clinical research, and residency in dermatology, I am committed to improving the lives of EB patients, to correct their underlying genetic disease and to offer new treatments for their skin and esophageal wounds. I was the PI of the investigator-initiated, Phase 1/2A clinical trial of autologous gene-therapy skin grafts for RDEB (Pz-cel, EB-101, previously called LEAES). I held the IND and have had successful interactions with the FDA Division of Dermatology which led to the successful granting of US orphan drug designation (2016), a Rare Pediatric Disease Designation (2017) and FDA’s Breakthrough Therapy Designation. I have led or co-led the conduct and completion of 10 investigator initiated clinical trials that have been published in high impact journals (NEJM, JAMA, J Clinical Oncology, Lancet Oncology). I have filed multiple INDs and have more than 12 years of clinical/translational experience in rare genetic diseases in dermatology.
