Drug intelligence / Profile preview

CAR123

Development stage
Unknown
Lead developer
Institute of Hematology and Blood Transfusion
Modality
Gene Addition/Replacement → Gene Therapies, Gene Silencing → Gene Therapies, Gene Editing → Gene Therapies, CAR-T Cells → Engineered T Cells → Adoptive Cell Transfer → Cell Therapies
Administration
Intravenous
01

Overview

CAR123 (UHKT-CAR123-01) is an investigational autologous chimeric antigen receptor (CAR) T-cell therapy targeting the CD123 antigen (interleukin-3 receptor subunit alpha), which is highly expressed in various hematological malignancies. Developed by the Institute of Hematology and Blood Transfusion (ÚHKT) in Prague and Charles University, this therapy is distinguished by its non-viral manufacturing process. It utilizes a linear DNA-based piggyBac transposon system (incorporating Applied DNA Sciences' Linea DNA technology) for gene transfer, which is designed to be more cost-effective and rapid than traditional viral vector methods. The CAR construct includes a 4-1BB-zeta intracellular signaling domain to enhance T-cell persistence and activation. It is currently being evaluated in Phase 1 clinical trials for relapsed or refractory acute myeloid leukemia (AML) and other CD123-positive hematologic disorders.

Other names
CD123-specific CAR-T cellsCD-123-specific CAR-T cellsCD 123-specific CAR-T cellsUHKT-CAR123UHKT-CAR-123UHKT-CAR 123
02

Targets

IL3RA (Interleukin 3 Receptor)

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