Drug intelligence / Profile preview

CART-45 + CD45BE-HSPC

Development stage
Unknown
Lead developer
University of Pennsylvania
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

CART-45 + CD45BE-HSPC is an experimental combination immunotherapy developed by the University of Pennsylvania for the treatment of various hematologic malignancies. The therapy utilizes base editing technology to target CD45, a pan-hematologic antigen expressed on nearly all white blood cells and most blood cancers. The treatment consists of two components: autologous T cells engineered to express a CD45-binding chimeric antigen receptor (CART-45) and autologous hematopoietic stem and progenitor cells (CD45BE-HSPCs) that have been base-edited to lack the specific CD45 epitope targeted by the CAR. To prevent the CAR T cells from killing each other (fratricide), the CART-45 cells are also epitope-edited. This dual-editing approach allows the CAR T cells to selectively eliminate malignant CD45-positive cells while the edited HSPCs repopulate the patient's blood system with cells that are invisible to the CAR T-cell attack, thereby avoiding the lethal bone marrow aplasia that would otherwise result from targeting a pan-leukocyte marker.

Other names
autologous base edited anti-CD45 CAR T cellsCD45 base edited hematopoietic stem and progenitor cellsCD-45 base edited hematopoietic stem and progenitor cellsCD 45 base edited hematopoietic stem and progenitor cellsanti-CD45 CAR T cellsanti-CD-45 CAR T cellsanti-CD 45 CAR T cells
02

Targets

PTPRC (Receptor-type tyrosine-protein phosphatase C)

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