Drug intelligence / Profile preview

CD123-specific autologous CAR T cells

Development stage
Phase 1
Lead developer
St. Jude Children's Research Hospital
Modality
CAR-T Cells → Engineered T Cells → Adoptive Cell Transfer → Cell Therapies, Gene Therapies
Administration
Intravenous
01

Overview

CD123-specific autologous CAR T cells are an investigational cell-based immunotherapy designed to target the interleukin-3 receptor alpha chain (CD123), which is highly expressed on leukemic blasts and leukemia stem cells in various hematologic malignancies. Developed primarily by St. Jude Children's Research Hospital, the therapy involves the genetic modification of a patient's own T cells using a lentiviral vector to express a chimeric antigen receptor (CAR). The CAR construct typically incorporates a CD123-specific single-chain variable fragment (scFv) linked to costimulatory domains such as CD28 and the CD3ζ signaling domain. The treatment is being evaluated as a bridge to allogeneic hematopoietic stem cell transplant for patients with relapsed or refractory acute myeloid leukemia (AML), B-cell acute lymphoblastic leukemia (B-ALL), and blastic plasmacytoid dendritic cell neoplasm (BPDCN). Additionally, partnerships have explored the use of PCR-produced linear DNA technology for the production of these CAR T cells in clinical trials.

Other names
CD123 CAR-TCD-123 CAR-TCD 123 CAR-Tanti-CD123 CAR-T cellsanti-CD-123 CAR-T cellsanti-CD 123 CAR-T cellsautologous anti-CD123 CAR-T cells
02

Targets

IL3RA (Interleukin 3 Receptor)

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