CONGRESS 2023 - Production of platelets and red cell in vitro for human transfusion

The idea of generating blood cells in vitro for transfusion is not new but only now we are reaching the point where the concept is reaching clinical trials. In vitro derived blood cells (namely platelets and red cells at this stage) are complementary to blood donor-derived products but with distinct advantages: biological safety, more resilient supply line and potentially less immunogenicity. We have developed a forward programming approach relying on the overexpression of transcription factors in pluripotent stem cells to produce the platelet mother cells, the megakaryocytes, conferring added efficiency and purity to the culture system. The challenges that remain to be addressed are related to transition to GMP production, optimising platelet release in the culture and quality control of the final product. The power of genome editing has also allowed us to explore the production of platelets with added clinical benefit (immune silent, added thrombotic potential). Red cell production from primary CD34+ progenitors has been demonstrated in academic laboratories about a decade ago. We are now mid-way through a first in human study to look at the potential of using in vitro derived red cells for transfusion. One of the main benefits would be a potentially longer survival of the manufactured red cells in the circulation than their donor-derived counterpart. This would allow spacing out transfusion intervals for patients on chronic transfusion programme, thereby reducing iron overload.

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