Trem-cel
Trem-cel achieved rapid neutrophil and platelet engraftment in the treated patients. The study found no evidence that CD33 deletion impaired hematopoiesis, immune reconstitution, or neutrophil oxidative burst function. The median manufactu…
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Trem-cel achieved rapid neutrophil and platelet engraftment in the treated patients. The study found no evidence that CD33 deletion impaired hematopoiesis, immune reconstitution, or neutrophil oxidative burst function. The median manufactured trem-cel dose was 8.01 x 10^6 CD34+ cells per kg and the median CD33 editing efficiency was 90%. Trem-cel is an allogeneic CD34+ hematopoietic stem and progenitor cell product edited with CRISPR-Cas9 to disrupt CD33. Trem-cel manufacturing used CD34+ magnetic selection followed by electroporation with a CD33-targeted CRISPR-Cas9 ribonucleoprotein. Trem-cel is intended to protect donor-derived hematopoiesis while enabling CD33-directed therapy against residual leukemia.