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BE CAR-33

Development stage
Phase 1
Lead developer
Great Ormond Street Hospital
Modality
CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, CAR-T Cells → Engineered T Cells → Adoptive Cell Transfer → Cell Therapies
Administration
Intravenous
01

Overview

BE CAR-33 is an investigational allogeneic, "off-the-shelf" chimeric antigen receptor (CAR) T-cell therapy being developed for the treatment of relapsed or refractory acute myeloid leukemia (AML), particularly in pediatric populations. The therapy utilizes healthy donor-derived T-cells that are engineered to express a CAR targeting the **CD33** antigen, which is highly expressed on AML blasts. To overcome challenges inherent in targeting myeloid antigens and using allogeneic cells, BE CAR-33 is manufactured using CRISPR-mediated base editing. This precision genome editing technology is used to perform multiplex modifications, including the knockout of the **TRAC** gene (to prevent graft-versus-host disease) and the disruption of the endogenous **CD33** gene on the T-cells themselves (to prevent fratricide, or self-killing, of the CAR T-cells). This approach aims to provide a readily available cell product for patients who may not have time for autologous manufacturing, potentially serving as a bridge to a definitive stem cell transplant.

Other names
Base-Edited CD33 CAR T-cellsBase Edited CAR T Cells Against AML
02

Targets

CD33 (Myeloid cell surface antigen CD33)

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