Sangamo Biosciences Inc (SGMO)

[AndiA1]

"The study, published in Nature Medicine, advances the development of novel, cell-based therapies for the treatment of a broad range of cancers."...

"The importance of the work has been recognized with the Van Bekkum Award, the most prestigious EBMT award for the best abstract submitted to the physician's program and is selected by the EBMT Board."...

"These data represent a significant advance in maximizing the potential safety and efficacy of adoptive T-cell therapies for cancer," said Dr. Bonini. ..."ZFN-based genome editing enables us to specifically modify these redirected cells and disrupt genes that interfere with their potency and specificity, thus generating a safer, more efficacious therapeutic product."

Cancer immunotherapy uses the immune system, specifically CD8+ T-cells, that have been "redirected" to seek out and destroy tumors. These redirected T-cells are genetically engineered to express new cell surface receptors that specifically recognize tumor cells. However, CD8+ T-cells have a natural specificity for different (non-tumor) targets and the resulting competition between the natural and the tumor-targeting gene products limits the potency of this cell therapy. More importantly, it can also make the cells "self-reactive," leading to graft versus host disease (GvHD).

In this study, ZFNs were used to specifically disrupt the native T-cell receptor (TCR) genes in these tumor-directed CD8+ T-cells resulting in an enhanced immunotherapeutic product with potent anti-cancer activity coupled with the elimination of GvHD in a mouse model. The data are described in a Nature Medicine paper entitled "Editing T-cell Specificity Towards Leukemia by Zinc Finger Nucleases and Lentiviral Gene Transfer" which appears as an Advance Online Publication on the Nature Medicine website http://dx.doi.org/10.1038/nm.2700 .

All this sounds like the beginning of novel working (functional) cancer therapies!