Epidermolysis Bullosa – Allogeneic
We are recruiting patients with recessive dystrophic Epidermolysis bullosa (RDEB) for an international multicentric phase I/IIa clinical trial.
The aim of the trial is to examine the efficacy and safety of allogeneic ABCB5-positive mesenchymal stem cells (ABCB5+ MSCs) in patients suffering from RDEB. Primary endpoints are the overall improvement of EB symptoms (as measured by percentage change of the EBDASI score) and the occurrence of adverse events.
For this trial 18 patients will be recruited in the USA, UK, Austria, France, Italy and Germany. The trial has been approved by the U.S. Food and Drug Administration (FDA) and the other respective competent national regulatory authorities. The European Medicines Agency (EMA) and the FDA in the USA has granted RHEACELL’s ABCB5+ MSC product Orphan Drug Designation for the treatment of RDEB.
For the manufacturing process of the allogeneic ABCB5+ MSCs we are using a patent hold by the Brigham and Women’s Hospital, Boston/USA, and the Children’s Hospital, Boston/USA, in cooperation with our scientific partner Prof. Dr. Markus Frank.
Background information about the use of ABCB5+ MSCs in EB can be found HERE.