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lentiviral-engineered hematopoietic stem cells with CD68 promoter
- Development stage
- Preclinical
- Lead developer
- Northwestern University
- Modality
- Gene Therapies, Stem Cell Therapies → Cell Therapies
- Administration
- Intravenous
01
Overview
This drug is an autologous cell and gene therapy platform consisting of hematopoietic stem cells (HSCs) engineered with a lentiviral vector. The vector utilizes the human CD68 (hCD68) promoter to drive the lineage-specific expression of therapeutic transgenes within the myeloid lineage, specifically targeting tumor-associated macrophages (TAMs). The platform is designed to overcome the immunosuppressive tumor microenvironment (TME) of glioblastoma (GBM) and other malignancies by leveraging the natural recruitment of HSC-derived monocytes to tumor sites. Once these monocytes differentiate into TAMs within the tumor, the CD68 promoter triggers the localized and durable expression of the engineered payload, potentially enhancing the efficacy of immunotherapies while minimizing systemic toxicity. Preclinical studies have demonstrated high specificity and induction of gene expression within the brain TME compared to peripheral tissues.
- Other names
- hCD68-LV-HSChCD-68-LV-HSChCD 68-LV-HSCmacrophage-specific HSC-based gene delivery platform
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