Lead Programs

 

Applying Innovative Technology to Develop Allogeneic CAR T Therapy

The process for manufacturing our off-the-shelf allogeneic CAR T therapy, or AlloCARs™, first involves harvesting healthy, selected, screened and tested T cells from healthy donors. This means that a larger portion of eligible patients, including those who are critically ill and have T cells that are difficult to harvest or expand, can potentially receive treatment, and no eligible patient will have to undergo leukapheresis (a laboratory procedure in which a patient’s white blood cells are separated and the remaining blood cells and plasma are returned to the patient).

Next, the T cells are engineered to express CARs, which recognize certain cell surface proteins that are expressed in hematologic or solid tumors. UCART19, our lead investigational therapy, recognizes CD19, a cell surface protein expressed on B-cells, including cancerous B-cells; it is just the first in a line of AlloCARs™ we plan to develop. The next step in the process involves gene editing to reduce the risk of graft versus host disease (GvHD) and to prevent allogeneic rejection. A T cell receptor gene is knocked out to avoid GvHD. The CD52 gene is knocked out to render the CAR T product resistant to anti-CD52 antibody treatment. Anti-CD52 antibody treatment can therefore be used to suppress the host immune system and allow the CAR T to stay engrafted to achieve full therapeutic impact.

The engineered T cells then undergo a purification step and are ultimately cryopreserved in vials for delivery to patients.