T-Cell Therapy

Truly personalized cell therapy for solid tumors

T-Cell receptors and neoantigens

Advances in genetic sequencing have empowered researchers to identify new therapeutic approaches to target neoantigens, the new or “neo” genetic mutations that give rise to cancer cells. These neoantigens are typically unique to each patient’s cancer and not present on any healthy cells or tissue. Ziopharm is using Sleeping Beauty to genetically modify TCRs to target these neoantigens to make a truly personalized cell therapy.

“With our technology, we can rapidly generate your own T cells that express TCR and when they’re put back into your body, they can identify neoantigens that are unique to your own cancer, go after it and kill it.”

Collaboration with NCI

Ziopharm, Precigen and the researchers at the National Cancer Institute are leveraging Sleeping Beauty to develop genetically modified TCR therapies that target neoantigens. In 2017, we entered into a Collaborative Research and Development Agreement with the NCI to evaluate Sleeping Beauty-modified TCRs for patients with advanced cancers. This research is being led by Steven A. Rosenberg, M.D., Ph.D., Chief of the Surgery Branch at the NCI.

The NCI team has used the Sleeping Beauty platform to generate neoantigen-specific T cells and is looking forward to initiating a clinical trial infusing these genetically modified T cells to target solid tumors. The Sleeping Beauty system is able to target the unique mutations that give rise to a patient’s malignancy.

Why Sleeping Beauty?

Sleeping Beauty’s non-viral gene transfer system is well suited for developing genetically modified TCR therapies that target neoantigens because of its very rapid manufacturing capability. For patients receiving genetically modified TCRs targeting their own neoantigens in their cancer cells, there will be a need for a manufacturing process to make one product per patient. That manufacturing process will need to be fast, scalable and cost effective, like Sleeping Beauty. See, “Stable, Nonviral Expression of Mutated Tumor Neoantigen-specific T-cell Receptors Using the Sleeping Beauty Transposon/ Transposase System,” published in the journal Molecular Therapy (5 March 2016, doi:10.1038/mt.2016.51).