Columbia University

Technology Ventures

Generation of T Cells for Therapeutic Use

Technology #2871

“Lead Inventor: Megan Sykes, M.D.

T Cells Production Critical for Proper Immune System Function Many pathological conditions and clinical situations can lead to immunodeficiency in patients related to problems with the functioning of the thymus. As a consequence, the body is not able to produce enough T cells for the immune system to function properly. Current treatment strategies for thymic insufficiency include administration of cytokines and growth factors to enhance thymus function and ex vivo production of T cells for infusion. However, these methods are cumbersome and inefficient. Improved technology in this field would have numerous therapeutic applications for patients with thymic insufficiency due to advanced age, chemo/radiotherapy treatment, immunosuppressive drug treatment, graft-vs-host disease, T cell-depleted hematopoietic stem cell transplant and HIV infection.

Large Scale Production of Diverse, Functional, Naive T Cells for Patients with Thymic Insufficiency The invention details a new method for producing large quantities of diverse, functional, naive T cells for infusion into patients with thymic insufficiency. The in vivo generated T cells arise from bone marrow taken from the patient and become genetically compatible immune cells that are tolerant of the donor. In addition to being immunologically functional, the T cells are also rejuvenated – an increased percentage of the cells are ”“naive”“, which means that these cells are capable of mounting immune defenses against new targets once they are transferred into the donor patient. With additional techniques done prior to collection and transfer, the T cells could also be made to recognize specific targets, altered to resist viruses (e.g. HIV resistance) or directed to become regulatory T cells that may ameliorate autoimmune disease and prevent/treat rejection of transplanted tissues.

Applications: • Treatment of patients with immunodeficiency arising from thymic insufficiency caused by advanced age, chemo/radiotherapy treatment, immunosuppressive drug treatment, graft-vs-host disease, T cell-depleted hematopoietic stem cell transplant and HIV infection • Produce T cells with desired specificities (e.g. against tumors, pathogens) • Produce T cells that are resistant to viruses present in the patient (e.g. HIV) • Produce a specific type of T cells, such as regulatory cells, that can treat autoimmune disease and treat/prevent transplant rejection and graft-versus-host disease • Produce mixed populations of T cells from two donor sources that are tolerant of one another, for use in rapid immune reconstitution in patients undergoing hematopoietic cell transplantation

Advantages: • Efficient in vivo development of large numbers of functional T cells for clinical use • Generates diverse naive T cells • Possible to produce T cells with desired specificities, virus resistance or of a regulatory subtype

Patent Status: Patent Pending

Licensing Status: Available for Sponsored Research Support ”