drug_type
RELEVANT_DRUG
intervention_type
Cellular immunotherapy (autologous gene-edited TIL)
drug_description
Autologous, gene-edited tumor-infiltrating lymphocyte (TIL) therapy engineered to inactivate SOCS1 and Regnase-1 (ZC3H12A) to enhance T-cell activation, cytokine production, proliferation, persistence, and tumor killing.
nci_thesaurus_concept_id
C210629
nci_thesaurus_preferred_term
Autologous CRISPR-Cas9 Engineered Regnase-1/SOCS1 Dual-edited Tumor Infiltrating Lymphocytes KSQ-004EX
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 both the endogenous genes suppressor of cytokine signaling 1 (SOCS1) and Regnase-1, with potential immunomodulating and antineoplastic activities. Upon infusion of the autologous CRISPR-Cas9 engineered Regnase-1/SOCS1 dual-edited TILs KSQ-004EX back into the patient, the cells specifically recognize, target and kill the patients tumor cells. Inactivation of endogenous SOCS1 and Regnase-1 in the TILs increases responsiveness to cytokine signals, increases persistence and memory formation of TILs, and enhances anti-tumor responses.
drug_category
TUMOR INFILTRATING LYMPHOCYTES
drug_class
Cellular Therapy
drug_delivery_route
Intravenous
drug_mechanism_of_action
Autologous tumor-infiltrating lymphocytes edited with CRISPR-Cas9 to inactivate SOCS1 and Regnase-1 (ZC3H12A), removing intrinsic brakes on cytokine/JAK-STAT signaling and mRNA stability. This enhances T-cell activation, cytokine production, proliferation, persistence, and cytotoxic killing of tumor cells after infusion.
drug_name
KSQ-004EX
nct_id_drug_ref
NCT06598371