ZIOPHARM Oncology Inc. (NASDAQ:ZIOP), a biopharmaceutical company focused on new cancer immunotherapies, today announced the publication of a study in Scientific Reports, a journal of the Nature Publishing Group, describing the genetic editing of human leukocyte antigen (HLA) in hematopoietic stem cells as a means of broadening the human application of these and other cell therapies. The article, titled “Genetic editing of HLA expression in hematopoietic stem cells to broaden their human application”, is available online first at http://www.nature.com/srep/.

Laurence Cooper, M.D., Ph.D., Chief Executive Officer of ZIOPHARM and senior author of the journal article, noted: “Genetic editing of HLA expression is a step towards generating universal biological products, where one donor’s cells may become suitable for sustained engraftment in multiple unrelated recipients. Unlocking the method by which HLA repertoire can be modified is one key to achieving this goal and fully harnessing the potential for off-the-shelf (OTS) therapies in immuno-oncology applications. Together with our partners at Intrexon and MD Anderson, we are bringing to bear multiple technologies to advance the findings of these studies and achieve this objective, with the goal of deploying it across our T-cell and natural killer (NK)-cell therapy platforms.”

Transplantation of allogeneic, or donor-derived, hematopoietic stem cells (HSCs) into recipients with hematologic disorders is used to restore bone marrow function, termed hematopoiesis. Finding a suitable donor can be challenging due to the need to match the constellation of HLA with the recipient. For the study, researchers at The University of Texas MD Anderson Cancer Center eliminated expression of one set of HLA molecules, termed HLA-A, on donor HSC using artificial zinc finger nucleases. Other HLA molecules, such as HLA B and C remained expressed to help prevent elimination by resident NK cells. Following genetic editing, the HSCs maintained their ability to engraft and reconstitute hematopoiesis. This paper reveals that genetically altered HSC harvested from a small pool of donors will then match with a large number of unrelated recipients, which has two implications. First, it broadens the number of recipients who might benefit from bone marrow transplantation, which has particular appeal for racial minorities underserved by the current genetic makeup of unrelated donors. Second, it paves the way for generating OTS cells that are HLA matched with multiple recipients, even though they were obtained from one donor.

ZIOPHARM is developing various cell-based immuno-oncology programs, including CAR-T, TCR and NK adoptive cell-based therapies, in collaboration with its partner Intrexon Corporation (NYSE:XON) and MD Anderson.(Original Source)

Shares of Ziopharm closed yesterday at $6.38. ZIOP has a 1-year high of $14.93 and a 1-year low of $4.56. The stock’s 50-day moving average is $5.89 and its 200-day moving average is $9.48.

On the ratings front, Ziopharm has been the subject of a number of recent research reports. In a report issued on January 25, Mizuho analyst Eric Criscuolo reiterated a Hold rating on ZIOP, with a price target of $6, which represents a potential downside of 6.0% from where the stock is currently trading. Separately, on January 14, J.P. Morgan’s Whitney Ijem reiterated a Hold rating on the stock .

According to TipRanks.com, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Eric Criscuolo and Whitney Ijem have a total average return of -14.3% and -16.5% respectively. Criscuolo has a success rate of 25.0% and is ranked #3256 out of 3638 analysts, while Ijem has a success rate of 20.0% and is ranked #3187.

ZIOPHARM Oncology Inc is a biopharmaceutical company. The Company is engaged in the acquiring, developing and commercializing portfolio of cancer therapies that can address unmet medical needs through synthetic biology.