First-in-Human CRISPR Immunotherapy Would Target PD-1


“At a time when PD-1 inhibitors are dominating the immunotherapy field, a team of researchers is seeking to use groundbreaking CRISPR gene editing technology for the first time in human beings to create an engineered T-cell agent that would knock out the gene that controls the immune checkpoint’s activity.

“Plans for the complex therapy call for taking NY-ESO-1, a peptide-based form of adoptive immunotherapy already in clinical trials, and disrupting the activity of the PDCD1 gene that encodes PD-1 as well as the endogenous TRAC and TRBC genes, which control T-cell receptor (TCR) alpha and beta, respectively.

“The goal would be to enhance the immune response with NY-ESO-1 by eliminating genomic drivers that hamper T-cell proliferation and function, University of Pennsylvania (UPenn) researchers said in describing the proposed study before a National Institutes of Health (NIH) panel on June 21.”

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