Gene Therapy for Enduring Protection - Medical / Health Care
We adapted an autologous ex-vivo gene therapy method to load the patient’s own TEMs precursors, that are hematopoietic stem and progenitor cells (HSPCs), with an immunotherapeutic transgene sequence. By re-introducing gene-modified HSPCs into the patient, we seek to take advantage of the self-renewing and multi-differentiation capability of HSPCs to enable durable and potentially long-term effects following a single treatment.
HSPCs are self-renewing cells that are capable of differentiating into all types of blood cells, including white blood cells, red blood cells and platelets. HSPCs can be obtained directly from the bone marrow or from the patient’s peripheral blood with the use of a mobilizing agent that induces HSPCs to relocate from the bone marrow into the peripheral blood, where they may be collected by apheresis. By using HSPCs, we create a biological reservoir of TEMs loaded with the therapeutic, so potentially providing enduring surveillance against future relapses or metastases.