Safety, efficacy and quality assessment of cell therapeutic products throughout manufacturing changes

Promethera Biosciences® is a Belgian Biotech company, producing the cell therapy product, HepaStem, to treat serious metabolic liver disorders. This treatment is based on allogeneic human liver progenitors isolated from healthy livers. A phase I/II clinical trial has been successfully accomplished for the treatment of Crigler-Najjar syndrome and Urea Cycle Disorders. As of today, 20 patients aged from 6 weeks to 16 years have been safely treated with HepaStem. Currently, Promethera has received the world’s first approval to start a prospective open-label multicenter phase IIb trial in Belgium for the treatment of Urea Cycle Disorders.