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CEBPA mRNA LNP
- Development stage
- Preclinical
- Lead developer
- Ostentus Therapeutics
- Modality
- mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Intravenous
01
Overview
CEBPA mRNA LNP is an experimental mRNA-based therapeutic consisting of messenger RNA encoding the CCAAT enhancer-binding protein alpha (CEBPA) encapsulated in lipid nanoparticles (LNPs). CEBPA is a critical transcription factor for myeloid differentiation, and its mutation or downregulation is a hallmark of certain subsets of acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS). By delivering exogenous wild-type CEBPA mRNA, the therapy aims to restore normal CEBPA protein expression, thereby overcoming the differentiation block characteristic of leukemic blasts, inducing apoptosis, and reducing cell proliferation. This approach is being investigated by researchers at the City of Hope in collaboration with Moderna for the treatment of CEBPA-mutated AML and other hematologic malignancies.
- Other names
- CEBPA mRNA lipid nanoparticle
02
Targets
CEBPA (CCAAT enhancer-binding protein alpha)
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