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CDUPRT-engineered MSC
- Development stage
- Preclinical
- Modality
- TCR-Engineered T Cells → Engineered T Cells → Adoptive Cell Transfer → Cell Therapies, CAR-T Cells → Engineered T Cells → Adoptive Cell Transfer → Cell Therapies, CAR-NK Cells → Other Engineered Cells → Adoptive Cell Transfer → Cell Therapies, CAR-Macrophages → Other Engineered Cells → Adoptive Cell Transfer → Cell Therapies, Stem Cell Therapies → Cell Therapies
- Administration
- Intraperitoneal
01
Overview
Mesenchymal stem cells engineered to express a fusion protein of **cytosine deaminase (CD)** and **uracil phosphoribosyl transferase (UPRT)** are known as CDUPRT-engineered MSCs. These cells may also be further engineered to co-express **Interferon-beta (IFNb)**, resulting in CDUPRT-IFNb_MSC. The main mechanism involves the local conversion of the prodrug 5-fluorocytosine (5FC) into cytotoxic 5-fluorouracil (5-FU) at tumor sites, providing targeted chemotherapy with reduced systemic toxicity. The addition of IFNb confers immunomodulatory and potent anti-tumor activity, enhancing the cGAS-STING pathway. These MSCs retain normal stem cell phenotype, migratory properties, and differentiation capacity. Preclinical evidence supports potent suppression of tumor growth (e.g., peritoneal carcinomatosis, hepatocellular carcinoma) and reduced metastasis when administered intraperitoneally and co-dosed with 5FC[1][2][3]. Safety profiles in animal models are favorable, and ongoing investigations are focused on establishing suitability for clinical cell therapy in oncology.
- Brand names
- CDUPRT-engineered MSC
- Other names
- MSCs expressing cytosine deaminase and uracil phosphoribosyl transferaseMSCs co-expressing CDUPRT and IFNbCD/UPRT-modified MSC
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