In summary, this is the first ever, human clinical trial to demonstrate multiplex CRISPR gene editing of T cells from patients with advanced, refractory cancer, resulting in a durable, safe and effective TCR immunotherapy. Therapeutic T cells, modified with the MaxCyte ExPERT platform, engrafted and survived for months in the human body, a significant improvement over many previous approaches where these cells lost their function within days.
In addition to having sustained ability to attack and kill tumors, the infusions were well tolerated, with no cytokine release syndrome or detected reaction to Cas9 protein. The result is a treatment that is not only more effective but safer for the patient.
The study presented here is an important milestone towards the ultimate goal of helping the body’s own immune system recognize and attack cancer, but the promise of the approach extends far beyond this. In fact, there are thousands of cell and gene therapy clinical trials ongoing worldwide to investigate modification of immune and stem cells for disease treatment (2). With the demonstrated safety and efficiency of multiplex gene editing and MaxCyte enabling technology, we now have not only a strong foundation for genome engineering but the potential for future improvement of innovative medicines.