Scalable in vitro production of red cells for clinical transfusion.

"Creating the ability to manufacture cultured red blood cells (cRBC) from stem cell lines is an important step towards solving supply shortages and reducing the risks of mismatched blood and transfusion transmitted infection in many countries. There is also increasing evidence that red cells stored for longer periods may have harmful effects in ill patients. cRBC are likely to contain a higher proportion of younger cells than standard donated red cell concentrates and may, therefore, provide clinical advantages by surviving longer and performing better. Over the past 4 years with the support of the Trust, the Organisation has established a clinical grade Good Manufacturing Practice protocol for using stem cell lines to manufacture red blood cells. We will build on this work to address 3 key issues: (i) improve and perfect this protocol, (ii) expand the manufacturing capacity (iii) conduct preclinical assessment and gain regulatory approval for initial studies in human volunteers. This work will provide the basis to attract investment for the development of large-scale manufacturing capability and clinical studies. The first clinical and commercial target will be treatment of chronic transfusion-dependent beta thalassaemia, where the complications associated with regular transfusion of red cells cause significant premature death world-wide."