NY-ESO-1-redirected CRISPR (TCRendo and PD1) Edited T Cells (NYCE T Cells)

Phase 1 Trial of Autologous T Cells Engineered to Express NY-ESO-1 TCR and CRISPR Gene Edited to Eliminate Endogenous TCR and PD-1 (NYCE T Cells)


Principal Investigator: Edward Stadtmauer, M.D., University of Pennsylvania


Study Population:  Adult patients who have relapsed or refractory tumors expressing NY-ESO-1 and HLA A2.  At Penn, patients with myeloma and sarcoma will be screened

Rational:  Recent studies have shown safety and promising efficacy of adoptive transfer of T cells with transgenic TCRs specific for NY-ESO-1. However, in many instances the gene-modified T cells become exhausted.  Here we are investigating a first application in humans of an efficient CRISPR/Cas 9 technology to delete the endogenous TCR and PD1. This offers two potential benefits over the previous work: 1) the T cells have higher expression of the transgenic TCR and 2) are resistant to the checkpoint molecule PDL1/L2.


ClinicalTrial.gov Identifier:  NCT03399448, UPCC 25416



Emerging Medicine