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LDLR mRNA exosomes
- Development stage
- Phase 1
- Lead developer
- Tangdu Hospital
- Modality
- mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Intravenous
01
Overview
LDLR mRNA exosomes are an experimental therapeutic modality consisting of extracellular vesicles (exosomes) engineered to carry messenger RNA (mRNA) encoding the low-density lipoprotein receptor (LDLR). Developed primarily by Codiak BioSciences using their engEx platform, this approach is designed to treat homozygous familial hypercholesterolemia (HoFH), a severe genetic disorder where patients lack functional LDLR, resulting in extremely high levels of LDL cholesterol and premature cardiovascular disease. The therapy leverages the natural ability of exosomes to protect mRNA from degradation in the circulation and facilitate its delivery to the liver. Upon uptake by hepatocytes, the mRNA is translated into functional LDLR proteins that are expressed on the cell surface, where they bind and internalize circulating LDL particles, thereby reducing plasma cholesterol levels. This exosome-based delivery system aims to provide a more targeted and potentially less immunogenic alternative to traditional lipid nanoparticle (LNP) delivery systems.
- Other names
- LDLR mRNA exosomes
02
Targets
LDLR (Low-density lipoprotein receptor)
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