Oligonucleotide Therapeutics (ASO/siRNA)

Cat. No.

A64981

CAS No.

2982817-49-0

Onvuzosiran

Onvuzosiran is a small interfering RNA (siRNA) that effectively reduces the synthesis of prekallikrein, a key component in the contact activation pathway of blood coagulation. By targeting and silencing the gene responsible for prekallikrein production, Onvuzosiran plays a crucial role in research focused on thrombosis, inflammatory responses, and related cardiovascular disorders. This reagent is essential for studies investigating the modulation of the coagulation cascade and the development of novel therapeutic strategies.

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.

A68428

CAS No.

2415330-05-9

Zorevunersen sodium

Synonyms: STK-001 sodium

Zorevunersen sodium is an antisense oligonucleotide that acts as an activator of the Scn1a gene through TANGO technology. This compound enhances Scn1a mRNA transcripts, leading to increased expression of the NaV1.1 protein. By restoring the excitability of parvalbumin interneurons, Zorevunersen sodium effectively reduces seizure frequency and improves survival in mouse models. It is a valuable tool for research focused on Dravet syndrome and related epileptic disorders.

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.

A68669

CAS No.

2415330-04-8

Zorevunersen

Synonyms: STK-001

Zorevunersen is an antisense oligonucleotide that acts as an activator of the Scn1a gene through TANGO technology. It enhances the production of Scn1a mRNA transcripts, leading to an increased expression of the NaV1.1 protein. This compound has demonstrated the ability to restore the excitability of parvalbumin interneurons, effectively reducing seizure activity and improving survival rates in murine models. Zorevunersen is a valuable tool for research focused on Dravet syndrome and related channelopathies.

Cat. No.

A68707

CAS No.

1848239-71-3

Tivanisiran sodium

Synonyms: SYL1001 sodium

Tivanisiran sodium is a small interfering RNA (siRNA) that targets the mRNA of transient receptor potential vanilloid 1 (TRPV1). It is primarily utilized in the research of dry eye disease by effectively silencing TRPV1 expression, which is involved in pain and sensory signaling. This agent facilitates the exploration of TRPV1's role in ocular conditions, contributing to the development of potential therapeutic strategies for managing dry eye symptoms.

Cat. No.

A68984

CAS No.

2755197-77-2

Rapirosiran sodium

Synonyms: ALN-HSD sodium

Rapirosiran sodium is an N-acetylgalactosamine-conjugated small-interfering RNA that specifically targets hydroxysteroid 17-beta dehydrogenase 13 (HSD17B13) mRNA in the liver. It is associated with a reduction in the risk of chronic liver diseases due to loss-of-function variants in the HSD17B13 gene. This reagent is particularly valuable for research focused on non-alcoholic steatohepatitis, facilitating the exploration of therapeutic strategies targeting liver metabolism and inflammation.

Cat. No.

A68985

CAS No.

2755196-97-3

Rapirosiran

Synonyms: ALN-HSD; ALN-288996; AD-288996

Rapirosiran is an N-acetylgalactosamine-conjugated small interfering RNA that specifically targets mRNA of hydroxysteroid 17-beta dehydrogenase 13 (HSD17B13) in the liver. Inhibition of HSD17B13 via Rapirosiran is associated with a reduced risk of chronic liver disease. This reagent is valuable for investigating the mechanisms of non-alcoholic steatohepatitis and exploring potential therapeutic approaches.

Cat. No.

A69055

CAS No.

2409783-34-0

Gatuzosiran

Gatuzosiran is a small interfering RNA (siRNA) that targets and reduces the synthesis of 17-beta-hydroxysteroid dehydrogenase 13 (HSD17B13). This compound plays a crucial role in research focused on fatty liver disease, providing insights into the molecular mechanisms underlying lipid metabolism and liver function. Its ability to modulate HSD17B13 expression makes it a valuable tool for studying the pathophysiology of liver diseases.

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.

A69324

CAS No.

2728713-54-8

Lefelsiran sodium

Synonyms: NN-6020 sodium

Lefelsiran sodium is a small interfering RNA (siRNA) that targets aldehyde dehydrogenase 2 (ALDH2). It is utilized in research to investigate the mechanisms underlying alcohol use disorder. By downregulating ALDH2, Lefelsiran sodium may provide insights into metabolic pathways associated with alcohol consumption and the potential therapeutic approaches for treating alcohol-related conditions.

Cat. No.

A69359

CAS No.

2765645-87-0

Lefelsiran

Synonyms: NN-6020

Lefelsiran is a small interfering RNA (siRNA) that specifically targets aldehyde dehydrogenase 2 (ALDH2). This reagent is primarily employed in research focused on alcohol use disorder, investigating the molecular mechanisms underlying this condition. By modulating ALDH2 expression, Lefelsiran facilitates the study of its role in alcohol metabolism and the potential therapeutic interventions for alcohol dependence.

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.

A69524

CAS No.

3078403-66-1

Lexosiran

Lexosiran is a small interfering RNA (siRNA) that reduces the synthesis of angiopoietin-related protein 3 (ANGPTL3). By inhibiting ANGPTL3, Lexosiran plays a significant role in lipid metabolism and may serve as a valuable tool in the study of antihyperlipidemic strategies. Its application extends to elucidating the biological pathways involved in lipid regulation and exploring therapeutic avenues for dyslipidemia.

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.

A69528

CAS No.

1573402-50-2

Volanesorsen sodium

Synonyms: ISIS 304801 sodium

Volanesorsen sodium is an antisense oligonucleotide designed to inhibit apolipoprotein CIII (apo-CIII) mRNA, effectively lowering triglyceride levels and enhancing insulin sensitivity. This compound is under investigation for its therapeutic potential in hypertriglyceridemia, familial chylomicronemia syndrome, and type 2 diabetes. Its unique mechanism positions it as a valuable tool for research into lipid metabolism and related metabolic disorders.

Cat. No.

A69530

CAS No.

2639854-33-2

Opemalirsen sodium

Synonyms: AZD2373 sodium; ION-972190 sodium

Opemalirsen sodium is an antisense oligonucleotide that specifically targets and reduces the synthesis of apolipoprotein L1 (APOL1). This compound is primarily utilized in research focusing on kidney disorders, providing insights into the molecular mechanisms underlying APOL1-related pathologies. Its ability to modulate APOL1 levels makes it a valuable tool for investigating therapeutic strategies in renal diseases.

Cat. No.

A69531

CAS No.

915430-78-3

Volanesorsen

Synonyms: ISIS 304801

Volanesorsen is an antisense oligonucleotide that specifically inhibits the mRNA of apolipoprotein CIII (apo-CIII), leading to a reduction in triglyceride levels and enhancement of insulin sensitivity. This compound is under investigation for its therapeutic potential in conditions such as hypertriglyceridemia, familial chylomicronemia syndrome, and type 2 diabetes. Volanesorsen offers a targeted approach for modulating lipid metabolism and improving metabolic outcomes.

Cat. No.

A69537

CAS No.

2379776-41-5

Plozasiran sodium

Synonyms: ARO-APOC3 sodium

Plozasiran sodium is a small interfering RNA (siRNA) that targets the apolipoprotein C-III (APOC3) gene. It plays a crucial role in regulating lipid metabolism and is utilized in research applications focused on mixed hyperlipidemia. By silencing APOC3, this reagent can help elucidate the molecular mechanisms underlying lipid disorders and explore potential therapeutic strategies for cardiovascular diseases.

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.