olezarsen

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Ligands
olezarsen

GtoPdb Ligand ID: 13658

Synonyms: AKCEA-APOCIII-LRX | IONIS-APOCIII-LRX | ISIS-678354 | ISIS-APOCIII-LRX | Tryngolza®

olezarsen is an approved drug (FDA (2024))

Comment: Olezarsen is an antisense GalNAc₃-conjugated oligonucleotide (ASO) that targets the messenger RNA for apolipoprotein C-III (APOC3) as a mechanism to treat dyslipidemias [1-3,5]. The nucleotide sequence is chemically modified to optimise in vivo stability and hepatic delivery. We have been unable to resolve a full SMILES or HELM notation for the entire ASO.

References
1. Chebli J, Larouche M, Gaudet D. (2024) APOC3 siRNA and ASO therapy for dyslipidemia. Curr Opin Endocrinol Diabetes Obes, 31 (2): 70-77. [PMID:38334488]
2. Hooper AJ, Bell DA, Burnett JR. (2024) Olezarsen, a liver-directed APOC3 ASO therapy for hypertriglyceridemia. Expert Opin Pharmacother, 25 (14): 1861-1866. [PMID:39305266]
3. Shamsudeen I, Hegele RA. (2022) Safety and efficacy of therapies for chylomicronemia. Expert Rev Clin Pharmacol, 15 (4): 395-405. [PMID:35762447]
4. Syed YY. (2025) Olezarsen: First Approval. Drugs, 85 (4): 571-576. [PMID:40074987]
5. Valdivielso P, Coca Prieto I. (2024) Apolipoprotein C-III, familial chylomicronemia syndrome, and olezarsen. Med, 5 (6): 493-494. [PMID:38878767]

References

1. Chebli J, Larouche M, Gaudet D. (2024)
APOC3 siRNA and ASO therapy for dyslipidemia.
Curr Opin Endocrinol Diabetes Obes, 31 (2): 70-77. [PMID:38334488]

2. Hooper AJ, Bell DA, Burnett JR. (2024)
Olezarsen, a liver-directed APOC3 ASO therapy for hypertriglyceridemia.
Expert Opin Pharmacother, 25 (14): 1861-1866. [PMID:39305266]

3. Shamsudeen I, Hegele RA. (2022)
Safety and efficacy of therapies for chylomicronemia.
Expert Rev Clin Pharmacol, 15 (4): 395-405. [PMID:35762447]

4. Syed YY. (2025)
Olezarsen: First Approval.
Drugs, 85 (4): 571-576. [PMID:40074987]

5. Valdivielso P, Coca Prieto I. (2024)
Apolipoprotein C-III, familial chylomicronemia syndrome, and olezarsen.
Med, 5 (6): 493-494. [PMID:38878767]