Promethera Biosciences, a Belgian biotechnology company developing Promethera HepaStem, a cell-based therapy for the treatment of liver-based metabolic diseases including Crigler-Najjar Syndrome and Urea Cycle Disorders, has treated its first two patients with its innovative treatment based on allogeneic adult liver stem cell technology (HepaStem).
The trial, a prospective, open label multicentre phase I/II study testing Promethera HepaStem has been designed to evaluate the safety and the preliminary efficacy of Promethera HepaStem in Urea Cycle Disorders and Crigler-Najjar Syndrome patients. “We are delighted to have achieved this important milestone in HepaStem’s development,” said Eric Halioua, Co-founder and Chief Executive Officer, Promethera Biosciences.
Dr Beatrice De Vos, Chief Medical Officer, Promethera Biosciences said, “HepaStem is an innovation in the field of liver-based metabolic diseases. This clinical trial will be the very first to apply allogeneic liver progenitor cells. This treatment with progenitor cells will contribute to restore a deficient enzyme function in Urea Cycle Diseases or in a Crigler-Najjar syndrome.”
The major innovation of Promethera HepaStem resides in the simplicity of the treatment. This consists of a simple injection into the vein leading to the liver, which may make it possible to avoid radical and invasive surgery such as a liver transplant. Promethera HepaStem could be used to treat a wide variety of liver pathologies. Thanks to the industrial development, if this therapy proves to be safe and effective, as many as a hundred patients could be treated from a single liver, thus largely overcoming the organ shortage problem.
Professor Etienne Sokal, Co-founder and Chief Scientific Officer at Promethera Biosciences, paediatric hepatologist at Cliniques Universitaires St Luc and Director of UCL’s cell therapy research lab said, “There is a real need for medical innovation to treat metabolic diseases in children; too many diseases are still intractable. This first study represents a major step in the development of Hepastem towards a therapy that could transform positively the prognosis of these diseases.”
EP News Bureau — Mumbai
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