Drug intelligence / Profile preview

CART33

Development stage
Phase 1
Lead developer
University of Pennsylvania
Modality
CAR-T Cells → Engineered T Cells → Adoptive Cell Transfer → Cell Therapies, Gene Therapies
Administration
Intravenous
01

Overview

CART33 is an investigational cell therapy consisting of autologous or allogeneic T cells genetically modified to express a chimeric antigen receptor (CAR) targeting the human CD33 antigen. The CAR construct typically uses a single-chain variable fragment (scFv) derived from the anti-CD33 antibody clone My96, which is also used in gemtuzumab ozogamicin. CART33 has demonstrated potent antileukemic activity in preclinical models and early clinical studies for acute myeloid leukemia (AML), particularly in relapsed or refractory cases. The therapy works by redirecting patient or donor-derived T cells to recognize and kill CD33-expressing leukemic blasts. However, because CD33 is also expressed on normal myeloid progenitors, there are concerns about hematopoietic toxicity and prolonged cytopenias with persistent expression of the CAR; transient mRNA-based approaches have been explored to mitigate this risk. Clinical trials have shown that CART33 can induce rapid reduction of leukemic burden but may be best suited as a bridge to further therapies such as hematopoietic stem cell transplantation[1][2][3][8].

Other names
CD33-directed CAR T cellsCD-33-directed CAR T cellsCD 33-directed CAR T cellsCD33-specific chimeric antigen receptor T cellsCD-33-specific chimeric antigen receptor T cellsCD 33-specific chimeric antigen receptor T cellsanti-CD33 CAR-Tanti-CD-33 CAR-Tanti-CD 33 CAR-T
02

Targets

CD33 (Myeloid cell surface antigen CD33)

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