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BPX-501 (rivogenlecleucel) is a genetically modified T-cell therapy developed by Bellicum Pharmaceuticals for use after allogeneic hematopoietic stem cell transplantation (HSCT), particularly in patients with blood cancers and inherited blood disorders. The therapy consists of donor-derived T cells engineered to express an inducible caspase 9 (iCasp9) "suicide gene" that can be activated by the small molecule rimiducid. This safety switch allows for selective elimination of the infused T cells if severe graft-versus-host disease (GvHD) or other complications arise. Rimiducid acts as a dimerizer that activates iCasp9, triggering apoptosis in the modified T cells to control GvHD while preserving immune reconstitution and anti-leukemic effects. The combination has received orphan drug designation from both FDA and EMA for treatment of immunodeficiency and GvHD following HSCT[1][4][6][7].
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