drug_type
RELEVANT_DRUG
intervention_type
Cellular therapy (gene-edited autologous TIL)
drug_description
Autologous, gene-edited tumor-infiltrating lymphocyte (TIL) therapy with SOCS1 inactivation to enhance JAK-STAT signaling, boosting T-cell activation, proliferation, persistence, and cytotoxicity against tumor antigens.
nci_thesaurus_concept_id
C204100
nci_thesaurus_preferred_term
Autologous CRISPR-Cas9 Engineered Tumor Infiltrating Lymphocytes KSQ-001EX
nci_thesaurus_definition
A preparation of autologous tumor infiltrating lymphocytes (TILs) gene-edited with the clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9 nuclease complex to inactivate the endogenous gene suppressor of cytokine signaling 1 (SOCS1), with potential immunomodulating and antineoplastic activities. Upon infusion of the autologous CRISPR-Cas9 engineered TILs KSQ-001EX back into the patient, the cells specifically recognize, target and kill the patients tumor cells. SOCS1 serves as a negative regulator of cytokine signaling in T-cells and negatively influences the survival, differentiation, and function of T-cells. Inactivation of endogenous SOCS1 in TILs increases responsiveness to cytokine signals, and enhances anti-tumor potency, persistence and memory formation of TILs.
drug_category
TUMOR INFILTRATING LYMPHOCYTES
drug_class
Cellular Therapy
drug_delivery_route
Intravenous
drug_mechanism_of_action
Autologous TILs engineered with CRISPR-Cas9 to inactivate SOCS1, removing negative regulation of cytokine/JAK-STAT signaling and thereby enhancing T-cell activation, proliferation, persistence, and cytotoxic killing of tumor cells.
drug_name
KSQ-001EX
nct_id_drug_ref
NCT06237881