Clinical trials, using ex vivo grown cells as therapeutic means, are currently on their way to treat devastating conditions, including cancer. However, cell-safety is a major concern in most cases, which holds back the full utilisation of these promising new treatments. We introduced a concept (FailSafeTM) and showed a genome editing strategy that addresses this concern.
Building on the FailSafeTM technology, we came up with a solution for the next hurdle faced by cell therapies; the allograft tolerance. We found that the transgenic expression of eight local-acting, immune-modulatory transgenes is sufficient to protect cells against rejection and achieve induced Allogeneic Cell Tolerance (iACT) in fully immune-competent recipients. Allografts survived long-term, in different MHC-mismatched recipients, and without the use of immunosuppressive drugs.
The combination of the FailSafeTM and iACT genome editing allows the generation a “single” pluripotent cell line as a source of off-the-shelf available therapeutic cell products for all humankind. We are in the process of characterising the therapeutic-cell differentiation potential of these cells equipped with FailSafeTM and iACT technologies.