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VCAM-targeted MFSD2A mRNA nanoparticle
- Development stage
- Preclinical
- Lead developer
- University of Pennsylvania
- Modality
- mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Intravenous
01
Overview
VCAM-targeted MFSD2A mRNA nanoparticle is an experimental RNA therapy designed to restore blood-brain barrier (BBB) integrity following ischemic stroke. Developed by researchers at the University of Pennsylvania and the University of Wisconsin, the therapy utilizes lipid nanoparticles (LNPs) functionalized with ligands targeting Vascular Cell Adhesion Molecule (VCAM), which is upregulated on inflamed cerebral endothelium post-stroke. The LNPs encapsulate mRNA encoding Major Facilitator Superfamily Domain Containing 2a (MFSD2A), a critical modulator of BBB integrity that inhibits caveolin-1–mediated vesicular transcytosis. By selectively delivering MFSD2A mRNA to the ischemic vasculature, the treatment aims to reduce endothelial transcytosis, mitigate cerebral edema, and improve neurological recovery. Preclinical studies in mouse models of transient middle cerebral artery occlusion (tMCAO) have demonstrated improved survival and neurobehavioral outcomes.
- Other names
- VCAM-targeted lipid nanoparticles encapsulating MFSD2A mRNA
02
Targets
MFSD2A (Major facilitator superfamily domain-containing protein 2A)CAV1 (Caveolin-1)VCAM1 (Vascular cell adhesion molecule 1)
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