tremtelectogene empogeditemcel

Tremtelectogene empogeditemcel (trem-cel) is an allogeneic, human leukocyte antigen-matched, genome-edited hematopoietic stem cell product created by using CRISPR-Cas9 to genetically ablate the myeloid protein CD33 from healthy donor hematopoietic stem and progenitor cells (HSPCs). This product is designed to provide patients with a reconstituted hematopoietic compartment that is resistant to anti-CD33 drug cytotoxicity, enabling the use of CD33-targeted therapies such as gemtuzumab ozogamicin (Mylotarg) at optimal doses and schedules without dose delays or omissions due to on-target, off-tumor toxicity. Trem-cel is intended for administration to hematopoietic cell transplant (HCT)-eligible patients with acute myeloid leukemia (AML) who are at high risk for leukemia relapse and post-transplant mortality.

Brand names

trem-cel

Other names

CD33-deleted CD34+ HSCCD33-null HSPC drug productCD-33-null HSPC drug productCD 33-null HSPC drug product