{"doi":"10.7759/cureus.96715","title":"Olezarsen: A Next-Generation Antisense Therapy for Hypertriglyceridemia and Familial Chylomicronemia Syndrome","abstract":null,"journal":"Cureus","year":2025,"id":627925,"datarank":0.0,"base_score":0.0,"endowment":0.0,"self_citation_contribution":0.0,"citation_network_contribution":0.0,"self_endowment_contribution":0.0,"citer_contribution":0.0,"corpus_percentile":null,"corpus_rank":null,"citation_count":0,"citer_count":0,"citers_with_citation_signal":0,"citers_with_endowment":0,"datacite_reuse_total":0,"is_dataset":false,"is_dataset_confidence":null,"is_data_producer":false,"deposit_databanks":null,"is_oa":false,"file_count":0,"downloads":0,"has_version_chain":false,"published_date":null,"fair_score":null,"fair_percentile":null,"algorithm_id":"datarank_citation_only_1hop_v6","ranking_scope":"data_only","authors":[{"id":1625487,"name":"Anna Javed","orcid":null,"position":1,"is_corresponding":false},{"id":120957,"name":"Manish Kumar Singh","orcid":"0000-0002-2232-0350","position":2,"is_corresponding":false},{"id":1625488,"name":"Bhanu Priya Singh","orcid":null,"position":3,"is_corresponding":false},{"id":820385,"name":"Ritesh Kumar","orcid":"0000-0003-3614-1350","position":4,"is_corresponding":false},{"id":1625489,"name":"Sartaj Hussain","orcid":null,"position":5,"is_corresponding":false},{"id":1625486,"name":"Jace Joshy","orcid":null,"position":0,"is_corresponding":false}],"reference_count":0,"raw_metadata":{"has_enrichment":true,"resolved":true,"title":"Olezarsen: A Next-Generation Antisense Therapy for Hypertriglyceridemia and Familial Chylomicronemia Syndrome","abstract":"Olezarsen (Tryngolza) is a next-generation, N-acetylgalactosamine (GalNAc)-conjugated antisense oligonucleotide that selectively inhibits hepatic ApoC-III, a key regulator of triglyceride metabolism. By inhibiting ApoC-III, olezarsen increases triglyceride clearance through both lipoprotein lipase (LPL)-dependent and -independent pathways. In the Phase 3 BALANCE trial, olezarsen reduced fasting triglycerides by approximately 60% at 12 months in patients with familial chylomicronemia syndrome (FCS), with a marked decrease in pancreatitis events versus placebo. Consistent triglyceride reductions (around 50%) were also observed in moderate and severe hypertriglyceridemia, along with improvements in ApoB-containing lipoproteins and high-density lipoprotein (HDL) profiles. In completed trials, olezarsen demonstrated a favorable safety profile, with most adverse events limited to mild injection-site reactions and no clinically significant thrombocytopenia. Ongoing Phase 3 trials (ESSENCE, CORE, and CORE2) will further define its role in cardiovascular risk reduction and pancreatitis prevention in broader hypertriglyceridemic populations. Olezarsen represents a precision medicine advance, offering effective triglyceride lowering with improved tolerability compared with earlier antisense therapies.","is_dataset_classified":null,"base_score":0.0,"endowment":0.0,"datacite_reuse_total":0,"file_count":0,"downloads":0,"views":0,"has_version_chain":false,"is_dataset":false,"is_oa":false,"pmid":"41393714","pmcid":"PMC12700839","openalex_id":"https://openalex.org/W4416158096","authors":[],"funders":[],"total_grants":0,"fwci":0.0,"citation_percentile":0.42567856,"influential_citations":0,"citation_trend":[],"oa_status":"gold","license":"cc-by","oa_locations":[{"url":"https://www.cureus.com/articles/430988-olezarsen-a-next-generation-antisense-therapy-for-hypertriglyceridemia-and-familial-chylomicronemia-syndrome.pdf","host_type":"journal"},{"url":"https://www.cureus.com/articles/430988-olezarsen-a-next-generation-antisense-therapy-for-hypertriglyceridemia-and-familial-chylomicronemia-syndrome.pdf","host_type":"publisher"},{"url":"https://www.cureus.com/articles/430988-olezarsen-a-next-generation-antisense-therapy-for-hypertriglyceridemia-and-familial-chylomicronemia-syndrome","host_type":"publisher"},{"url":"https://doi.org/10.7759/cureus.96715","host_type":"journal"},{"url":"https://pubmed.ncbi.nlm.nih.gov/41393714","host_type":"repository"},{"url":"https://pmc.ncbi.nlm.nih.gov/articles/PMC12700839/","host_type":"repository"},{"url":"https://europepmc.org/articles/PMC12700839","host_type":"Europe_PMC"},{"url":"https://europepmc.org/articles/PMC12700839?pdf=render","host_type":"Europe_PMC"}],"fields_of_study":["Lipid metabolism and disorders","Diabetes, Cardiovascular Risks, and Lipoproteins","Peroxisome Proliferator-Activated Receptors"],"mesh_terms":[],"keywords":["Hypertriglyceridemia","Triglyceride","Tolerability","Lipoprotein lipase","Pancreatitis","Adverse effect","Acute pancreatitis","Antisense therapy","Antisense oligonucleotides","Familial Chylomicronemia Syndrome","Olezarsen","Tryngolza"],"sdg_mappings":[],"linked_datasets":[],"clinical_trials":[],"software_tools":[],"database_accessions":[{"name":"nct"}],"source":"live","citation_network_status":"fetched"},"created_at":"2026-08-05T10:00:31.987701Z","pmid":null,"pmcid":null,"fwci":null,"citation_percentile":null,"influential_citations":0,"oa_status":null,"license":null,"views":0,"total_file_size_bytes":0,"version_count":0,"fair_f":null,"fair_a":null,"fair_i":null,"fair_r":null,"fair_zscore":null,"fair_rationale":null,"fair_model":null,"fair_agent_version":null,"fair_fulltext_source":null,"fair_has_llm":null,"fair_computed_at":null,"clinical_trials":[],"software_tools":[],"db_accessions":[],"linked_datasets":[],"topics":[]}