Definition

A preparation of allogeneic, human leukocyte antigen (HLA) matched, cluster of differentiation 34 (CD34)-positive human hematopoietic stem and progenitor cells (HSPCs) that are ex-vivo gene-edited with the clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9 nuclease complex to remove the cluster of differentiation 33 (CD33) antigen, that can potentially be used to improve hematopoietic recovery after hematopoietic cell transplantation (HCT) and CD33-targeting chemotherapy. Upon administration, tremtelectogene empogeditemcel increases and restores the number of HSPCs, which may prevent or decrease infection and other complications of HCT and chemotherapy-induced neutropenia (CIN). The removal of CD33 protects VOR33 from the cytotoxic effects of CD33-targeted agents administered after HCT.