New Product: Strimvelis (autologous CD34+ enriched cell fraction that contains CD34+ cells transduced with retroviral vector that encodes for the human adenosine deaminase cDNA sequence from human haematopoietic stem/progenitor (CD34+) cells)

This is the first approved stem cell gene therapy for an individual patient, for this condition. The patient must be able to donate adequate CD34+ cells to deliver the minimum 4 million purified CD34+ cells/kg, required for manufacture of Strimvelis.