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saRNA-LNP encoding PAI-1
- Development stage
- Preclinical
- Lead developer
- University of British Columbia
- Modality
- RNA Therapeutics → Nucleic Acid Therapeutics, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Intravenous
01
Overview
saRNA-LNP encoding PAI-1 is a preclinical self-amplifying RNA (saRNA) therapy encapsulated in lipid nanoparticles (LNPs) designed to endogenously synthesize and increase circulating levels of plasminogen activator inhibitor-1 (PAI-1). Developed by the Versiti Blood Research Institute and the University of British Columbia, this platform aims to treat PAI-1 deficiency, a rare blood disorder that causes moderate bleeding and cardiac fibrosis. Unlike conventional mRNA therapies which provide transient expression, the saRNA platform enables sustained hepatic expression and protein production of PAI-1 for over a month in animal models.
- Other names
- saRNA-PAI-1saRNA-PAI1saRNA-PAI 1saRNA-PAI-1 LNPsaRNA-PAI1 LNPsaRNA-PAI 1 LNPself-amplifying RNA encoding PAI-1 in lipid nanoparticlesself-amplifying RNA-lipid nanoparticle encoding plasminogen activator inhibitor-1
02
Targets
PAI-1 (Plasminogen activator inhibitor type 1)PLAT (Tissue-type plasminogen activator)PLAU (uPA)
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