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Synonyms: AKCEA-APO(a)-LRx | Ionis-apo(a)-lrx | ISIS 681257 | ISIS-681257 | TQJ-230 | TQJ230
Compound class:
Nucleic acid
Comment: Pelacarsen (TQJ230) is an apolipoprotein A (Lp(a)) synthesis reducer. It is a liver-targeted (GalNAc3-conjugated) antisense oligonucleotide that was designed to lower genetically elevated Lp(a) as a mechanism to reduce atherosclerotic cardiovascular disease risk [1-2,4-5].
We have been unable to locate a full chemical SMILES for the drug, or its HELM notation. We provide the basic nucleic acid structure for the molecule here. Full specifications for all modifications are available from its INN record. |
| References |
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1. Alhomoud IS. (2025)
Emerging therapies targeting lipoprotein(a): the next frontier in cardiovascular risk reduction. Front Med (Lausanne), 12: 1727918. [PMID:41488100] |
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2. Karwatowska-Prokopczuk E, Lesogor A, Yan JH, Hurh E, Hoenlinger A, Margolskee A, Xia S, Tsimikas S. (2023)
Efficacy and safety of pelacarsen in lowering Lp(a) in healthy Japanese subjects. J Clin Lipidol, 17 (1): 181-188. [PMID:36529659] |
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3. Parhofer KG, Julius U, Herzog AL, Krüger T, Weinmann-Menke J, Heine GH, Cao H, Schorr J, Ferber P, Wang J et al.. (2026)
Pelacarsen and lipoprotein(a) apheresis in secondary prevention: the Lp(a)FRONTIERS APHERESIS trial. Eur Heart J, [Epub ahead of print]. [PMID:41721795] |
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4. Yeang C, Karwatowska-Prokopczuk E, Su F, Dinh B, Xia S, Witztum JL, Tsimikas S. (2022)
Effect of Pelacarsen on Lipoprotein(a) Cholesterol and Corrected Low-Density Lipoprotein Cholesterol. J Am Coll Cardiol, 79 (11): 1035-1046. [PMID:35300814] |
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5. Yu RZ, Gunawan R, Post N, Zanardi T, Hall S, Burkey J, Kim TW, Graham MJ, Prakash TP, Seth PP et al.. (2016)
Disposition and Pharmacokinetics of a GalNAc3-Conjugated Antisense Oligonucleotide Targeting Human Lipoprotein (a) in Monkeys. Nucleic Acid Ther, 26 (6): 372-380. [PMID:27500733] |
