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Bap-Notch engineered mesenchymal stromal cells
- Development stage
- Preclinical
- Lead developer
- Vanderbilt University
- Modality
- Gene Therapies, Stem Cell Therapies → Cell Therapies
- Administration
- Intracerebral
01
Overview
Bap-Notch engineered mesenchymal stromal cells (MSCs) are an experimental cell-based therapy designed for the treatment of Alzheimer's disease. Developed by researchers at Vanderbilt University, these cells are modified using the synthetic Notch (synNotch) platform to express a chimeric receptor. The extracellular domain of this receptor utilizes a single-chain variable fragment (scFv) derived from the anti-amyloid-beta (Aβ) monoclonal antibody bapineuzumab. Upon encountering and binding to Aβ aggregates in the brain, the synNotch receptor undergoes intramembrane proteolysis, releasing an intracellular synthetic transcription factor. This factor then translocates to the nucleus to drive the expression of programmed therapeutic transgenes, such as anti-inflammatory agents (e.g., soluble TNF receptor 1 and IL-1 receptor antagonist). This localized, inducible approach aims to combine the innate immunomodulatory properties of MSCs with targeted, disease-responsive delivery of potent therapeutics to mitigate neuroinflammation and protein aggregation.
- Other names
- Bap-Notch MSCsbapineuzumab-synNotch engineered mesenchymal stromal cells
02
Targets
Aβ (Amyloid-beta peptides and aggregates)TNFA (Tumor necrosis factor alpha (soluble))IL-1R (Interleukin-1 receptor type 1 (IL-1R1))
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