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CD33HER2CAR HSPCs

Development stage
Preclinical
Lead developer
University of Pennsylvania
Modality
Gene Therapies, Stem Cell Therapies → Cell Therapies
Administration
Intravenous
01

Overview

CD33HER2CAR HSPCs are an experimental cell therapy consisting of primary human hematopoietic stem/progenitor cells (HSPCs) genetically engineered using non-viral CRISPR/Cas9 technology. The engineering involves a knock-in of a chimeric antigen receptor (CAR) targeting Human Epidermal Growth Factor Receptor 2 (HER2) into the endogenous CD33 locus, effectively replacing the CD33 gene. This approach exploits the dispensability of CD33 in normal myelopoiesis to create a durable source of CAR-expressing myeloid cells (CAR-M). Once engrafted, these HSPCs reconstitute long-term myelopoiesis, producing CAR-M that infiltrate the tumor microenvironment, mediate direct antitumor activity, and promote T-cell recruitment and infiltration. Developed by researchers at the University of Pennsylvania, this platform aims to overcome the limited half-life of traditional CAR-M therapies by providing a continuous supply of engineered myeloid cells.

Other names
CD33-HER2-CAR HSPCsCD-33-HER2-CAR HSPCsCD 33-HER2-CAR HSPCsCD33-targeted HER2-CAR engineered hematopoietic stem/progenitor cells
02

Targets

CD33 (Myeloid cell surface antigen CD33)ERBB2 (Erb-b2 receptor tyrosine kinase 2)

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