Preferred Label : Autologous WT1-directed CRISPR/Cas9-engineered TCR-T Cells NTLA-5001;
NCIt synonyms : Autologous WT1-directed CRISPR-Cas9-engineered TCR T Cells NTLA-5001; Autologous Anti-WT1 CRISPR-Cas9-engineered TCR T-cells NTLA-5001;
NCIt definition : A preparation of human autologous T-lymphocytes gene-edited with the clustered regularly
interspaced short palindromic repeats (CRISPR)-Cas9 nuclease complex to disrupt expression
of endogenous T-cell receptor (TCR) and modified to express a TCR specific for the
tumor-associated antigen (TAA) Wilms tumor 1 (WT1) epitope, WT1 37-45, and human leukocyte
antigen (HLA)-A*02:01, with potential immunostimulating and antineoplastic activities.
Upon isolation, transduction, expansion ex vivo, and reintroduction into the patient,
the autologous WT1-directed CRISPR/Cas9-engineered TCR-T cells NTLA-5001 recognize
and bind to WT1-expressing tumor cells. This may result in a specific cytotoxic T-lymphocyte
(CTL)-mediated killing of WT1-expressing tumor cells. WT1 protein, a zinc finger DNA-binding
transcriptional regulator, is overexpressed in various leukemias and solid tumors,
while expression in normal, healthy tissues is very limited; its expression is correlated
with aggressiveness and poor prognosis. The removal of endogenous TCR reduces TCR
competition for expression, increases the persistence and function of the expressed
transgenic TCR, enhances resistance to T-cell exhaustion and increases T-cell activity.;
Molecule name : NTLA 5001; NTLA-5001;
NCI Metathesaurus CUI : CL1773475;
Origin ID : C185126;
UMLS CUI : C5666943;
Semantic type(s)
- Cell [UMLS semantic type]
- chemical_or_drug_affects_cell_type_or_tissue
- chemical_or_drug_has_mechanism_of_action
- chemical_or_drug_has_physiologic_effect
- concept_is_in_subset
- has_target
