Room:

Genome Engineering of a Safety Switch and Immunoprotection in Stem Cells

Andras Nagy, Canada

Senior Investigator and Professor
Lunenfeld-Tanenbaum Research Institute, Sinai Health System
Sinai Health and University of Toronto

Overview

The solutions for cell therapy safety and allogeneic cell acceptance are critical for getting therapies, including those that targeting T1D, into the clinic. Our work in these areas has led to the development of two platform technologies; FailSafeTM cells and induced Allogeneic Cell Tolerance (iACT). We feel strongly that both could become the standard of care in many cell therapy applications and allow us to envision a single, pluripotent cell line that could be used as a source of safe and off-the-shelf therapeutic cells to serve all humankind. 

We foresee several complementary approaches for our technologies towards the treatment of T1D.  First, we showed that we could generate a subcutaneous immune-privileged hosting tissue site for transplanted islets, which protects from allogeneic or even xenogeneic rejection and autoimmune attack. In addition to creating a subcutaneous tissue as a protective site for grafts, we also envision that our FailSafeTM and cloaked pluripotent cells could be used to make therapeutic cells via directed differentiation, such as insulin-producing, beta cells.



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