Successful blood and marrow transplantation (BMT) requires the infusion of a sufficient number of hematopoietic stem/progenitor cells (HSPCs), capable of both homing to the bone marrow and regenerating a full array of hematopoietic cell lineages with early and late repopulating ability in a timely fashion.
Omidubicel is a stem/progenitor cell-based product composed of ex vivo expanded allogeneic cells from one entire unit of umbilical cord blood consisting of mature myeloid and lymphoid cells as follows:
1. Ex vivo expanded, umbilical cord blood-derived hematopoietic CD34+ progenitor cells (cultured fraction (CF)), containing a minimum of 8.0 × 10\^8 total viable cells of which a minimum of 8.7% is CD34+ cells and a minimum of 9.2 × 10\^7 CD34+ cells, and
2. the non-cultured cell fraction of the same Cord Blood Unit (CBU) (Non-cultured Fraction (NF)), containing a minimum of 4.0 × 10\^8 total viable cells with a minimum of 2.4 × 10\^7 CD3+ cells
Omidubicel utilizes the small molecule nicotinamide (NAM), as an epigenetic approach to inhibit differentiation and to increase the migration, bone marrow (BM) homing and engraftment efficiency of hematopoietic progenitor cells (HPC) expanded in ex vivo cultures.
The overall study objectives are to provide access to omidubicel for transplantation in patients with hematological malignancies and to collect additional safety and efficacy data.
