We are developing human long-term hematopoietic stem cells (LT-HSCs) for therapeutic uses, in particular, as a cell replacement therapy — in lieu of a bone marrow transplant — for cancer patients whose bone marrow stems cells have been permanently damaged or destroyed by chemotherapy or radiation, for patients suffering from inherited bone marrow failure syndromes, and for patients whose bone marrow no longer produces LT-HSCs, including as a result of old age.

HSCs are rare subsets of hematopoietic cells that are responsible for production of all blood cell lineages, and for the reconstitution of bone marrow (BM) after transplantation into myeloablated recipients. Hematopoietic stem cell transplantation (HSCT) is the therapeutic approach of curing the blood/immune system of patients suffering from malignancies, immunodeficiencies, red blood cell disorders, and inherited bone marrow failure syndromes. We are developing these specialized human cells (LT-HSCs) from induced pluripotent stem cells (iPSCs) using our proprietary HD-DOE™ research and development platform as the basis for a cell replacement-based therapy. We have developed a single-stage cell culture media for substantially increasing the percentage of reconstituting LT-HSC compared to standard protocols.

We are also developing an automated production to scale the production of these cells.