Drug intelligence / Profile preview

mCART117

Development stage
Preclinical
Lead developer
Washington University School of Medicine
Modality
CAR-T Cells → Engineered T Cells → Adoptive Cell Transfer → Cell Therapies
Administration
Intravenous
01

Overview

mCART117 is a murine chimeric antigen receptor (CAR) T cell therapy designed to target CD117 (also known as c-kit or stem cell factor receptor), a cell-surface receptor expressed on hematopoietic stem and progenitor cells (HSPCs) and acute myeloid leukemia (AML) cells. Developed as a preclinical tool to study the safety and feasibility of CD117-targeted immunotherapy, mCART117 is intended to function both as a conditioning regimen for hematopoietic stem cell transplantation (HSCT) and as a direct treatment for AML. The construct typically utilizes a single-chain fragment variable (scFv) derived from clones such as 2D1 or ACK2, fused to murine CD28 costimulatory and CD3 zeta signaling domains. Research has shown that while mCART117 can eliminate CD117+ cells in the spleen, its efficacy against bone marrow-resident AML and HSPCs in immunocompetent models is limited by protective mechanisms such as regulatory T cell (Treg) suppression and reactive myelopoiesis following lymphodepletion.

Other names
murine CD117-targeting chimeric antigen receptor T cellsmurine c-kit-targeting CAR T cellsmurine CART117-2D1murine CART117-ACK2
02

Targets

KIT (c-KIT proto-oncogene receptor tyrosine kinase)

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