Oligonucleotide Therapeutics (ASO/siRNA)

Cat. No.

A57825

CAS No.

2921918-19-4

Farabursen sodium

Synonyms: RGLS8429 sodium; RG1015 sodium

Farabursen sodium is a selective inhibitor of the microRNA-17 family. By effectively inhibiting the functionality of miR-17, it enhances the expression of target genes such as PKD1 and PKD2, leading to increased levels of polycystin-1 and -2 (PC1/2). This compound demonstrates significant potential in attenuating the growth of renal cysts and reducing kidney weight-to-body weight ratios, making it valuable for research into autosomal dominant polycystic kidney disease.

Cat. No.

A57831

CAS No.

1848257-85-1

Cobomarsen sodium

Synonyms: MRG-106 sodium

Cobomarsen sodium is a targeted miR-155 inhibitor that modulates various signaling pathways, including JAK/STAT, MAPK/ERK, and PI3K/AKT, contributing to the regulation of cell survival. This oligonucleotide is primarily employed in research focused on B-cell lymphoma, enabling insights into the molecular mechanisms of this malignancy and potential therapeutic strategies.

Cat. No.

A57837

CAS No.

1848257-52-2

Cobomarsen

Synonyms: MRG-106

Cobomarsen is an oligonucleotide inhibitor targeting miR-155. This compound modulates various signaling pathways associated with cell survival, including the JAK/STAT, MAPK/ERK, and PI3K/AKT pathways. Cobomarsen is primarily utilized in research focused on B-cell lymphoma, providing insights into miRNA-mediated gene regulation and potential therapeutic approaches.

Cat. No.

A57842

CAS No.

2568631-54-7

Dimovarsen

Dimovarsen is an oligonucleotide designed as an inhibitor of micro RNA-132. This compound plays a crucial role in the modulation of gene expression related to cardiac function and has potential applications in the study of heart failure. By inhibiting micro RNA-132, Dimovarsen may aid in elucidating the underlying mechanisms of heart disease and facilitate the exploration of novel therapeutic strategies.

Cat. No.

A58689

CAS No.

1337968-84-9

Bamosiran

Synonyms: SYL040012

Bamosiran is a small interfering RNA that specifically targets the β-adrenergic receptor 2. This compound is recognized for its ability to significantly reduce intraocular pressure, making it valuable in the study of ocular conditions. Its role as an adrenergic receptor inhibitor aids in investigating therapeutic strategies for glaucoma and other eye disorders.

Cat. No.

A58949

CAS No.

2589926-22-5

Evazarsen

Synonyms: ISIS-757456

Evazarsen is an angiotensinogen synthesis inhibitor that effectively reduces the production of angiotensinogen, a precursor to angiotensin II, a peptide that plays a crucial role in blood pressure regulation. This compound exhibits antihypertensive properties, making it valuable for research focused on cardiovascular diseases and hypertension management. Its ability to modulate the renin-angiotensin system supports investigations into therapeutic strategies for hypertension and related disorders.

Cat. No.

A58974

CAS No.

2380166-33-4

Zilebesiran

Synonyms: AD-85481; ALN-AGT

Zilebesiran is a small interfering RNA (siRNA) that selectively inhibits hepatic angiotensinogen expression via RNA interference. This reagent plays a crucial role in the investigation of mild to moderate hypertension, as angiotensinogen is the primary precursor of angiotensin peptides and a significant regulator of systemic blood pressure. Researchers can utilize Zilebesiran to explore therapeutic strategies targeting the renin-angiotensin system in cardiovascular studies.

Cat. No.

A59001

CAS No.

2589926-24-7

Evazarsen sodium

Synonyms: ISIS-757456 sodium

Evazarsen sodium is an angiotensinogen synthesis inhibitor that exhibits antihypertensive properties. By targeting the synthesis of angiotensinogen, it plays a significant role in regulating blood pressure and can be utilized in research related to cardiovascular diseases and hypertension management. This compound is valuable for studies focusing on the molecular mechanisms of blood pressure regulation and the development of potential therapeutic interventions.

Cat. No.

A59004

CAS No.

2380166-34-5

Zilebesiran sodium

Synonyms: AD-85481 sodium; ALN-AGT sodium

Zilebesiran sodium is a small interfering RNA (siRNA) that effectively reduces hepatic angiotensinogen levels through RNA interference. By targeting angiotensinogen, a primary precursor of angiotensin peptides, it plays a significant role in regulating blood pressure. This reagent is valuable for research applications related to mild to moderate hypertension and cardiovascular disease mechanisms.

Cat. No.

A64411

CAS No.

1152798-49-6

Lixadesiran

Lixadesiran is an siRNA designed to inhibit cyclooxygenase-2 (COX-2) by targeting its mRNA. This compound is part of the STP705 formulation, which includes two siRNA oligonucleotides, with Pixofisiran targeting TGF-β1. Lixadesiran's mechanism of action contributes to the modulation of inflammatory pathways, making it relevant for research in cancer and fibrosis studies.

Cat. No.

A64626

CAS No.

2305355-64-8

Zilganersen sodium

Synonyms: ION373 sodium

Zilganersen sodium is a gapmer antisense oligonucleotide that specifically inhibits glial fibrillary acidic protein (GFAP) synthesis. By targeting the GFAP gene, Zilganersen sodium effectively reduces the accumulation of GFAP associated with pathogenic variants, making it a valuable tool for studying Alexander disease. This compound facilitates research into the mechanisms of glial cell dysfunction and therapeutic approaches in related neurodegenerative disorders.

Cat. No.

A64643

CAS No.

2305355-56-8

Zilganersen

Synonyms: ION373

Zilganersen is a gapmer antisense oligonucleotide that specifically targets glial fibrillary acidic protein (GFAP). By inhibiting the synthesis of GFAP, Zilganersen reduces excess levels produced by disease-associated variants in the GFAP gene. This reagent is valuable for research focused on the pathophysiology of Alexander disease and the roles of glial cells in neurological disorders.

Cat. No.

A64972

CAS No.

2304701-45-7

Donidalorsen sodium

Synonyms: ISIS-721744; IONIS-PKK-LRX

Donidalorsen sodium is an antisense oligonucleotide that selectively targets prekallikrein (PKK). By binding to and promoting the degradation of PKK mRNA in the liver, Donidalorsen sodium effectively inhibits kallikrein activity and diminishes Bradykinin production. This reagent is particularly valuable for research applications related to hereditary angioedema.

Cat. No.

A64987

CAS No.

2304692-48-4

Donidalorsen

Synonyms: ISIS-721744 free acid; IONIS-PKK-LRX free acid

Donidalorsen is an antisense oligonucleotide that selectively targets prekallikrein (PKK). By binding to and degrading PKK mRNA in the liver, Donidalorsen effectively inhibits kallikrein activity and reduces bradykinin production. This reagent is valuable for research focused on hereditary angioedema and related pathophysiological studies.

Cat. No.

A68542

CAS No.

2247669-96-9

Cofirasersen

Synonyms: ION-827359

Cofirasersen is a sodium channel inhibitor that targets the epithelial sodium channel (ENaC). It is specifically developed to downregulate ENaC expression in the lungs, where hyperactivity of ENaC is implicated in cystic fibrosis, a disorder linked to mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. This compound has potential applications in research related to cystic fibrosis and the modulation of sodium transport in pulmonary tissues.

Cat. No.

A69236

CAS No.

2304711-30-4

Obeversen

Synonyms: ION-769357

Obeversen is an antisense oligonucleotide that specifically inhibits diacylglycerol acyltransferase 2 (DGAT-2). This inhibition plays a crucial role in disrupting lipid metabolism and has been implicated in the research of nonalcoholic fatty liver disease. Obeversen serves as a valuable tool for studying the molecular mechanisms underlying lipid dysregulation and offers potential insights into therapeutic targets for metabolic disorders.

Cat. No.

A69241

CAS No.

2304711-35-9

Obeversen sodium

Synonyms: ION-769357 sodium

Obeversen sodium is an antisense oligonucleotide that selectively inhibits Diacylglycerol acyltransferase 2 (DGAT-2) synthesis. This inhibition plays a critical role in modulating lipid metabolism, making it a valuable tool for studying nonalcoholic fatty liver disease and related metabolic disorders. Researchers can utilize Obeversen sodium to explore the pathways associated with lipid accumulation and investigate potential therapeutic targets in hepatic diseases.

Cat. No.

A69517

CAS No.

2172852-12-7

Vupanorsen sodium

Synonyms: IONIS ANGPT-L3Rx sodium; ISIS 703802 sodium

Vupanorsen sodium is an N-acetyl galactosamine-conjugated antisense oligonucleotide that specifically inhibits the synthesis of Angiopoietin-like 3 (ANGPTL3) protein. This compound is effective in lowering triglyceride levels and reducing atherogenic lipoproteins, making it a valuable tool for research in lipid metabolism and cardiovascular disease. Its potential applications extend to studies aimed at understanding the role of ANGPTL3 in dyslipidemia and associated disorders.

Cat. No.

A69518

CAS No.

2232918-66-8

Zodasiran

Synonyms: ARO-ANG3

Zodasiran is a small interfering RNA (siRNA) designed to inhibit angiopoietin-like protein 3 (ANGPTL3) in the liver. This inhibition plays a crucial role in lipid metabolism regulation, contributing to potential therapeutic applications in dyslipidemia and cardiovascular diseases. Zodasiran is a valuable tool for research studies focused on lipid regulation and metabolic disorders.

Cat. No.

A69520

CAS No.

2232918-74-8

Zodasiran sodium

Synonyms: ARO-ANG3 sodium

Zodasiran sodium is a small interfering RNA (siRNA) designed to specifically inhibit angiopoietin-like protein 3 (ANGPTL3) in hepatic tissues. This compound plays a critical role in lipid metabolism and has significant implications for the study of dyslipidemia and cardiovascular diseases. Zodasiran sodium is utilized in research applications aimed at investigating the therapeutic potential of ANGPTL3 modulation in metabolic disorders.

Cat. No.

A69521

CAS No.

2707429-60-3

Solbinsiran sodium

Synonyms: LY3561774 sodium

Solbinsiran sodium is a small interfering RNA (siRNA) designed to inhibit the production of angiopoietin-like protein 3 (ANGPTL3) in hepatic tissues. By silencing ANGPTL3 expression, it plays a crucial role in lipid metabolism and is being investigated for its potential therapeutic applications in dyslipidemia and cardiovascular diseases. This reagent serves as a valuable tool for studying the impact of ANGPTL3 modulation on lipid profiles and associated metabolic disorders.

Cat. No.

A69522

CAS No.

2097587-62-5

Vupanorsen

Synonyms: IONIS ANGPT-L3Rx; ISIS 703802

Vupanorsen is an N-acetyl galactosamine-conjugated antisense oligonucleotide that specifically targets and inhibits the synthesis of Angiopoietin-like 3 (ANGPTL3). This compound is demonstrated to effectively lower triglyceride levels and reduce atherogenic lipoprotein concentrations. Its primary applications include research on lipid metabolism, cardiovascular disease, and the therapeutic potential for dyslipidemia management.

Cat. No.

A69525

CAS No.

2718150-11-7

Solbinsiran

Synonyms: LY3561774

Solbinsiran is a small interfering RNA (siRNA) that specifically targets and inhibits angiopoietin-like 3 (ANGPTL3), a key regulator of lipid metabolism. By silencing ANGPTL3, Solbinsiran effectively reduces lipid levels and has significant potential in the treatment of dyslipidemia and metabolic disorders. This reagent is intended for research applications focused on lipid regulation and cardiovascular health.

Cat. No.

A69538

CAS No.

2298451-31-5

Olezarsen sodium

Synonyms: ISIS 678354 sodium; IONIS-APOCIII-LRx sodium; AKCEA-APOCIII-LRx sodium

Olezarsen sodium is an APOC3 inhibitor that functions as a GalNAc-modified antisense oligonucleotide. It binds to APOC3 mRNA, leading to its degradation through ribonuclease H1-mediated sense strand cleavage, which decreases the hepatic synthesis of apolipoprotein C-III. This mechanism results in the reduction of plasma triglycerides, apolipoprotein B, and non-high-density lipoprotein cholesterol levels. Olezarsen sodium is valuable for research into familial chylomicronemia syndrome, hypertriglyceridemia, and atherosclerotic cardiovascular disease.

Cat. No.

A73694

CAS No.

2589926-20-3

Tonlamarsen

Synonyms: ION-904

Tonlamarsen is an angiotensinogen synthesis inhibitor, primarily acting as an antihypertensive agent. It effectively reduces blood pressure by decreasing the production of angiotensinogen, leading to lower levels of angiotensin II, a potent vasoconstrictor. This compound is valuable for research in cardiovascular physiology and the development of novel antihypertensive therapies.