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IONIS-FGB-LRx
- Development stage
- Unknown
- Lead developer
- Ionis Pharmaceuticals
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Subcutaneous
01
Overview
IONIS-FGB-LRx (formerly AKCEA-FGB-LRx) is a ligand-conjugated antisense (LICA) oligonucleotide designed to reduce the production of fibrinogen, a key glycoprotein involved in blood coagulation. The drug specifically targets the mRNA of the fibrinogen beta chain (FGB), which is the rate-limiting component for the assembly of the fibrinogen hexamer in the liver. By utilizing RNase H1-mediated degradation of the target mRNA, IONIS-FGB-LRx lowers circulating levels of fibrinogen. It is primarily being developed for the prevention of thrombotic disorders. Additionally, preclinical research has highlighted its potential in oncology, demonstrating that depletion of fibrinogen can disrupt the tumor microenvironment, suppress primary tumor growth, and inhibit metastasis in aggressive cancers like pancreatic ductal adenocarcinoma (PDAC) by reducing extravascular fibrin deposits.
- Other names
- fibrinogen-specific antisense oligonucleotideFg-ASOfibrinogen B-beta chain antisense oligonucleotidefibrinogen beta chain ASO
02
Targets
CFB (Complement Factor B)
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