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Cas9 mRNA + F11 sgRNA
- Development stage
- Preclinical
- Lead developer
- CIBER-BBN
- Modality
- CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Intravenous
01
Overview
Cas9 mRNA + F11 sgRNA is an in vivo CRISPR/Cas9-based gene editing therapy designed for long-term anticoagulation and the prevention of venous thrombosis. Developed by researchers from the University of Murcia, CIBERER, CIBER-BBN, and CIEMAT, the therapy consists of CleanCap Cas9 mRNA and a single guide RNA (sgRNA) targeting the F11 gene, which encodes coagulation Factor XI (FXI). Delivered via lipid nanoparticles (LNPs) to the liver, the therapy induces double-strand breaks in the F11 gene, leading to permanent gene disruption and a sustained reduction in plasma FXI levels. By targeting FXI, which amplifies thrombin generation during thrombosis but plays a minimal role in baseline hemostasis, this approach aims to provide robust antithrombotic protection while mitigating the bleeding risks and adherence challenges associated with conventional anticoagulants.
- Other names
- CleanCap Cas9 mRNA + F11 sgRNACRISPR/Cas9 F11 gene editing therapy
02
Targets
F11 (Coagulation Factor XI)
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