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Ziopharm bets on the HLA-A odds

The A & BCs of stem cell HLA editing

Ziopharm Oncology Inc. is adding gene editing to its repertoire with a strategy to increase the odds of finding a match for hematopoietic stem cell transplants. While the system is still short of an allogeneic supply, the company believes it can go beyond the one donor-one recipient paradigm and employ a single donor's cells to benefit multiple patients.

In a study published last month in Scientific Reports, the company presented its case that by eliminating the HLA-A gene from donor stem cells it could increase patients' chances of finding a match, and showed that engineered stem cells lacking HLA-A could engraft and differentiate into mature blood cells following transplantation in mice.

CEO Laurence Cooper told BioCentury one of the big goals in transplantation is to make off-the-shelf cells that "are made in advance of their need and then deployed on demand." But those cells are often genetically dissimilar between donor and patient, so it becomes

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