iXgene iPS cell-derived neural stem cell therapy

iXgene's stroke and brain injury program utilizes a dual gene and cell therapy approach centered on genome-edited, induced pluripotent stem cell (iPSC)-derived neural stem cells (NSCs). These NSCs are engineered to express a therapeutic fusion gene, **CD-UPRT**, which combines cytosine deaminase (CD) and uracil phosphoribosyltransferase (UPRT). This platform technology is designed to be administered alongside the prodrug 5-fluorocytosine (5-FC). The CD-UPRT enzyme locally converts the non-toxic 5-FC into the potent cytotoxic agent 5-fluorouracil (5-FU) at the site of injury. While this "suicide gene" system is traditionally used to eliminate cancer cells (as in iXgene's glioblastoma program), in the context of ischemic stroke and brain injury, it is explored for its potential to modulate the microenvironment or manage the transplanted cell population, while the NSCs themselves contribute to neural repair and neuroprotection.

Other names

iXgene-CD-UPRTgenome-edited iPS cell-derived neural stem cells expressing CD-UPRTiPS-NSC-CD-UPRT