On March 09, 2016 ZIOPHARM Oncology, Inc. (Nasdaq:ZIOP), a biopharmaceutical company focused on new cancer immunotherapies, reported the publication of an article describing the use of Sleeping Beauty non-viral gene transfer technology to modify T cells for the targeting of neoantigens present within solid tumors (Press release, Ziopharm, MAR 9, 2016, View Source [SID:1234509448]). This approach unlocks the potential for rapid and inexpensive personalized T-cell receptor (TCR) gene therapy aimed at the unique mutations within a patient’s cancer cells. The article, titled "Stable, non-viral expression of mutated tumor neoantigen-specific T-cell receptors using the Sleeping Beauty transposon/transposase system," was published in the journal Molecular Therapy (5 March 2016, doi:10.1038/mt.2016.51), and is available online.
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"The DNA plasmids from the Sleeping Beauty platform provide a customizable solution to personalized TCR gene therapy, which is needed to open the door to safely target solid tumors," said Laurence Cooper, M.D., Ph.D., Chief Executive Officer of ZIOPHARM and an author of the paper. "There is growing consensus that a non-viral approach to gene therapy will be essential to targeting individual neoantigens. In Sleeping Beauty, we have the only clinically validated non-viral gene transfer platform. This compels us to bring this important technology to patients."
Neoantigens unique to each patient’s tumor can be recognized by autologous T cells through their T-cell receptors, but the low frequency and/or terminal differentiation of mutation-specific T cells may limit their utility as adoptive T-cell therapies. The publication describes how the Sleeping Beauty platform can be used to transfer and stably express TCR genes into T cells while retaining their proliferative potential. Indeed, the Sleeping Beauty gene transfer occurs without the need for cell division thus facilitating the genetic modification of minimally-differentiated T cells. The authors note that the Sleeping Beauty platform "can facilitate the use of personalized T cell therapy targeting unique neoantigens."
The Sleeping Beauty transposon-transposase system was exclusively licensed by Intrexon Corporation (NYSE:XON) through the University of Texas MD Anderson Cancer Center and accessed as part of ZIOPHARM’s collaboration with Intrexon.