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HTRA1 mRNA-engineered induced mesenchymal stem cells
- Development stage
- Preclinical
- Lead developer
- Factor Bioscience
- Modality
- mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, iPSCs → Pluripotent Stem Cells → Stem Cell Therapies → Cell Therapies
- Administration
- Intravenous
01
Overview
HTRA1 mRNA-engineered induced mesenchymal stem cells are an experimental allogeneic cell therapy candidate developed by Factor Bioscience. These cells are derived from induced pluripotent stem cells (iPSCs) and engineered via mRNA electroporation to transiently express and secrete High Temperature Requirement A1 (HTRA1), a serine protease with chaperone activity. HTRA1 is a protein quality control enzyme that degrades misfolded amyloidogenic protein aggregates, such as tau fibrils and alpha-synuclein, which are central to the pathology of Alzheimer's disease and other neurodegenerative proteinopathies. By utilizing iPSC-derived mesenchymal stem cells (iMSCs) as a delivery vehicle, the therapy aims to combine the inherent anti-inflammatory and neuroprotective properties of MSCs with the targeted proteolytic clearance of toxic protein aggregates mediated by HTRA1.
- Other names
- HTRA1-expressing iMSCsHTRA-1-expressing iMSCsHTRA 1-expressing iMSCsHTRA1-iMSCsHTRA-1-iMSCsHTRA 1-iMSCsHTRA1 mRNA-engineered iMSCsHTRA-1 mRNA-engineered iMSCsHTRA 1 mRNA-engineered iMSCs
02
Targets
IGFBP5 (Insulin-like growth factor-binding protein 5)NOTCH (Neurogenic locus notch homolog protein 3)Tau aggregates (Microtubule-associated protein tau (pathological form))
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