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pelacarsen
- Development stage
- Phase 3
- Lead developer
- Novartis
- 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
Pelacarsen is an investigational antisense oligonucleotide designed to reduce the production of apolipoprotein(a) in the liver, thereby lowering circulating levels of lipoprotein(a) [Lp(a)], a known risk factor for cardiovascular disease. It specifically targets the mRNA of the LPA gene in hepatocytes, preventing translation and subsequent production of apo(a). Pelacarsen has demonstrated dose-dependent reductions in Lp(a) levels by 35–80% in clinical trials and is being developed primarily for patients with elevated Lp(a) who are at increased risk for cardiovascular events. The drug is currently undergoing Phase 3 clinical trials (Lp[a]HORIZON study), with its development led by Ionis Pharmaceuticals and licensed to Novartis for worldwide development, manufacturing, and commercialization[1][3][5][7].
- Other names
- pelacarsen
02
Targets
LPA (Lysophosphatidic acid)
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