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UM171-expanded cord blood is an ex vivo cell therapy product consisting of hematopoietic stem and progenitor cells (HSPCs) derived from a single umbilical cord blood unit and expanded using the small molecule UM171. Developed by researchers at the Maisonneuve-Rosemont Hospital and commercialized by ExCellThera (as ECT-001), this technology addresses the primary limitation of cord blood transplantation—the low number of stem cells—by inducing a significant expansion of long-term repopulating hematopoietic stem cells (HSCs). UM171 acts as a self-renewal agonist, likely by targeting the CoREST complex, which enhances the lymphoid differentiation potential of the graft while preventing exhaustion. This approach allows for the use of smaller, better HLA-matched cord blood units, leading to rapid engraftment, low non-relapse mortality, and reduced rates of chronic graft-versus-host disease in patients with high-risk hematologic malignancies who lack suitable matched donors.
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