Recessive dystrophic epidermolysis bullosa is a rare genetic condition that can leave children with delicate, blistering skin. It is characterised by the absence of collagen in the skin due to the inheritance of a faulty copy of the COL7A1 gene from both parents.
The autologous, cell-based, gene therapy, Zevaskyn, was originally developed by researchers at Stanford University, California, who used a genetically engineered form of the herpes simplex virus to deliver a functional copy of the COL7A1 gene into skin cells, thereby correcting their ability to properly synthesise collagen (see BioNews 1139).
Abeona Therapeutics in Cleveland, Ohio, who licensed the therapy from Stanford University, has now gained FDA approval based on a Phase 3 VIITAL study, showing that just a single application of could safely heal chronic wounds
For more details, please visit Inside Precision Medicine.