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Cancer-Fighting T-Cells Engineered Using CRISPR

Why This Trial

Cell therapy represents a revolution in cancer treatment. The big idea: take cells from the immune system and turn them against a person’s own tumors.

This has worked brilliantly against certain blood cancers, resulting in the 2017 FDA approval of a type of cell therapy called CAR-T. Since then, the immunotherapy field has scrambled to engineer cell therapies that work on solid cancers – not just blood cancers – in a more targeted fashion.

CRISPR/Cas9 gene-editing technology gives us a way to do just that.

By applying CRISPR gene-editing to cell therapy in this trial, we hope to find a novel, safe way to outsmart solid tumors and learn more that can help us as we design cell therapies of the future.

About the Study

This landmark phase 1 clinical trial is the first in the United States to involve CRISPR/Cas9 gene-editing.

Scientists collect T-cells from cancer patients and then gene edit the cells in three ways using CRISPR. The changes are intended to help T-cells stay alive longer and more effectively target and eliminate cancer.

After the modifications, scientists then multiply the cells in culture by the billions and infuse them into the patient to help fight the tumors.

Who’s Eligible

  • Patients with melanoma, sarcoma and multiple myeloma

Treatments Tested

  • T-cells engineered with CRISPR to express a T-cell receptor (TCR) that targets the protein NY-ESO-1 and gene-edited to eliminate the expression of a native T-cell receptor and the protein PD-1.


Findings published in Science in February 2020 showed genetically-edited immune cells can persist, thrive, and function long after a cancer patient receives them. Cells removed from patients and brought back into the lab were able to kill cancer months after their original manufacturing and infusion. Further analysis of these cells confirmed they were successfully edited in three specific ways, marking the first-ever sanctioned investigational use of multiple edits to the human genome.

This follows up on the initial data presentation at the 61st American Society of Hematology Annual Meeting and Exposition that showed researchers were able to use CRISPR/Cas9 technology to successfully edit three cancer patients’ immune cells, the first such successful attempt in a U.S. clinical trial.

Where We’re At Now

The trial began enrolling patients in early 2018 at the University of Pennsylvania, and was completed in February 2020.

Multiple patients with relapsed cancers – including multiple myeloma and sarcoma – were treated.

The University of Pennsylvania presented early evidence at the American Society of Hematology 2019 Annual Meeting suggesting that CRISPR-edited T-cells are safe in cancer patients and published the results in Science in February 2020.

Molecular analysis on patient samples from the trial are underway. The team intends to revise their approach with these learnings in a future study.



Principal Investigators

  • Edward Stadtmauer, MD | University of Pennsylvania


The SINATRA Trial is funded in part by PICI and Tmunity.

For more information on this trial (NCT03399448), visit