Drug intelligence / Profile preview

olezarsen

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 is an antisense oligonucleotide designed to treat adults with familial chylomicronemia syndrome (FCS) by reducing triglyceride levels in the blood. It works by binding to and degrading apolipoprotein C-III (apoC-III) mRNA in hepatocytes, thereby decreasing apoC-III protein production. ApoC-III normally inhibits lipoprotein lipase and hepatic uptake of triglyceride-rich lipoproteins; thus, lowering apoC-III enhances the breakdown and clearance of plasma triglycerides and very low-density lipoproteins (VLDL). Olezarsen is administered as a subcutaneous injection once monthly. The drug was developed by Ionis Pharmaceuticals and is marketed under the brand name Tryngolza[1][2][5][6][7].

Brand names
Tryngolza
Other names
olezarsenISIS 678354 sodium saltISIS678354 sodium saltISIS-678354 sodium salt
02

Targets

APOC3 (Apolipoprotein C-III)

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