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olezarsen (Ionis Pharmaceuticals)
- Development stage
- Approved
- 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
Olezarsen (formerly APOCIII-LRx) is a GalNAc-conjugated antisense oligonucleotide (ASO) designed to inhibit the production of apolipoprotein C-III (apoC-III), a key regulator of triglyceride metabolism. By targeting apoC-III mRNA in the liver, olezarsen promotes the clearance of triglyceride-rich lipoproteins and inhibits the assembly of very-low-density lipoproteins (VLDL). Developed by Ionis Pharmaceuticals, it is being investigated for the treatment of familial chylomicronemia syndrome (FCS) and severe hypertriglyceridemia (SHTG). The GalNAc modification allows for targeted delivery to hepatocytes, enhancing potency and allowing for lower, less frequent dosing compared to non-conjugated ASOs.
- Other names
- olezarsen
02
Targets
APOC3 (Apolipoprotein C-III)ASGPR (Asialoglycoprotein Receptor 1)
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