Oligonucleotide Therapeutics (ASO/siRNA)

Cat. No.

A69539

CAS No.

2097587-83-0

Olezarsen

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

Olezarsen is a GalNAc-modified antisense oligonucleotide that specifically targets APOC3 mRNA. By inducing degradation of this mRNA through ribonuclease H1-mediated cleavage, Olezarsen effectively decreases the synthesis of hepatic apolipoprotein C-III (apoC-III). This compound has demonstrated efficacy in reducing levels of plasma triglycerides, apolipoprotein B, and non-high-density lipoprotein cholesterol. Olezarsen is relevant for research focused on familial chylomicronemia syndrome, hypertriglyceridemia, and atherosclerotic cardiovascular disease.

Cat. No.

A69540

CAS No.

2379776-40-4

Plozasiran

Synonyms: ARO-APOC3

Plozasiran is an siRNA designed to target APOC3, a key regulator of lipid metabolism. This reagent is utilized in research focused on mixed hyperlipidemia, allowing for the investigation of APOC3's role in dyslipidemia and cardiovascular diseases. Its application aids in elucidating the underlying mechanisms of lipid regulation and potential therapeutic strategies for metabolic disorders.

Cat. No.

A69550

CAS No.

2639853-99-7

Opemalirsen

Synonyms: AZD2373; ION-972190

Opemalirsen is an antisense oligonucleotide that specifically reduces the synthesis of apolipoprotein L1 (APOL1). It plays a crucial role in investigating the underlying mechanisms of kidney disorders, particularly those associated with APOL1 mutations. This reagent is a valuable tool for researchers studying APOL1-related pathologies and exploring novel therapeutic strategies.

Cat. No.

A72936

CAS No.

2589926-21-4

Tonlamarsen sodium

Synonyms: ION-904 sodium

Tonlamarsen sodium is an angiotensinogen synthesis reducer that exhibits antihypertensive activity. It functions by inhibiting the production of angiotensinogen, leading to decreased angiotensin II levels and subsequent reduction in blood pressure. This compound is primarily utilized in research focusing on hypertension and related cardiovascular disorders.

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.

Cat. No.

A74731

CAS No.

959716-29-1

Anivamersen

Anivamersen is an RNA aptamer that functions as a reversal agent for the anticoagulant effects of the parenteral factor IXa inhibitor, pegnivacogin. This innovative compound is integral to the REG1 anticoagulation system, which combines pegnivacogin's inhibition of coagulation factor IXa with anivamersen to effectively restore hemostasis. Anivamersen is essential for research applications focused on anticoagulant therapies and the management of bleeding complications in clinical settings.

Cat. No.

A74740

CAS No.

959716-31-5

Anivamersen sodium

Anivamersen sodium is an RNA aptamer designed to reverse the anticoagulant effects of the factor IXa inhibitor, pegnivacogin. This compound acts as a complementary sequence reversal oligonucleotide within the novel REG1 anticoagulation system, facilitating the restoration of hemostasis. Anivamersen sodium is essential for studying mechanisms of anticoagulation and potential therapeutic applications in coagulation disorders.

Cat. No.

A74774

CAS No.

3061406-99-0

Vortosiran

Vortosiran is a small interfering RNA (siRNA) that targets and reduces the synthesis of coagulation factor XI, thereby exerting anticoagulant effects. This compound has potential applications in the study of thrombotic disorders and the development of novel anticoagulant therapies. Vortosiran can be utilized in research settings to explore the modulation of coagulation pathways and the impact of factor XI on thrombosis.

Cat. No.

A74924

CAS No.

1826859-79-3

Atesidorsen sodium

Synonyms: COR-004 sodium; ATL1103 sodium; ISIS 227452 sodium

Atesidorsen sodium is an antisense oligomer that functions as a selective inhibitor of human growth hormone receptor (GHR) mRNA translation. By targeting GHR, this reagent reduces its expression, impacting growth and metabolism pathways. It is primarily utilized in research focused on elucidating the role of GHR in various diseases and understanding growth hormone signaling mechanisms.

Cat. No.

A74926

CAS No.

2131025-82-4

Cimdelirsen

Synonyms: ISIS 766720; IONIS-GHR-LRx

Cimdelirsen is a ligand-conjugated, hepatic-targeted antisense oligonucleotide that selectively inhibits growth hormone receptor (GHR) synthesis. This mechanism effectively reduces the adverse effects associated with growth hormone (GH) hypersecretion and lowers circulating insulin-like growth factor-1 (IGF-1) levels. It has potential applications in the treatment of acromegaly, providing a novel therapeutic approach for managing this condition.

Cat. No.

A74927

CAS No.

872063-57-5

Atesidorsen

Synonyms: COR-004; ATL1103; ISIS 227452

Atesidorsen is a second-generation antisense oligomer that specifically inhibits the translation of human growth hormone receptor (GHR) mRNA. This selective inhibition effectively reduces GHR expression, leading to decreased signaling pathways associated with growth hormone activity. Atesidorsen is primarily utilized in research focused on growth hormone regulation and its implications in various metabolic disorders and conditions related to abnormal growth patterns.

Cat. No.

A74928

CAS No.

2131025-83-5

Cimdelirsen sodium

Synonyms: ISIS 766720 sodium; IONIS-GHR-LRx sodium

Cimdelirsen sodium is an investigative antisense oligonucleotide designed to inhibit growth hormone receptor (GHR) synthesis through a ligand-conjugated, hepatic-targeted mechanism. This compound effectively reduces the detrimental effects associated with growth hormone (GH) hypersecretion and lowers circulating insulin-like growth factor-1 (IGF-1) levels in patients with acromegaly. Its primary applications include studying the modulation of GH signaling and exploring therapeutic strategies in disorders related to GH excess.

Cat. No.

A75525

CAS No.

2437257-12-8

Zifcasiran sodium

Synonyms: ADS-007 sodium; ARO HIF2 sodium

Zifcasiran sodium is a synthetic double-stranded siRNA designed to target hypoxia-inducible factor-2α (HIF2α), modulating its expression and disrupting pro-oncogenic signaling pathways. This reagent effectively engages the RNA interference (RNAi) machinery, facilitating the degradation of HIF2α (EPAS1) mRNA, which leads to a decrease in translated HIF2α protein levels. Zifcasiran sodium is particularly relevant in research focused on clear cell renal cell carcinoma (ccRCC) and the mechanisms of tumor growth regulation.

Cat. No.

A75571

CAS No.

2437257-11-7

Zifcasiran

Synonyms: ADS-007; ARO HIF2

Zifcasiran is a synthetic double-stranded siRNA designed to target hypoxia-inducible factor-2 alpha (HIF2α). By engaging the RNA interference (RNAi) pathway, Zifcasiran effectively degrades HIF2α mRNA, thereby inhibiting the expression of this key oncogenic factor in clear cell renal cell carcinoma (ccRCC). This mechanism reduces downstream pro-oncogenic signaling, making Zifcasiran a valuable tool for research on ccRCC and potential therapeutic applications in targeting hypoxia-related pathways.

Cat. No.

A75821

CAS No.

2894810-55-8

Senvesiran

Senvesiran is a small interfering RNA (siRNA) that targets and reduces the synthesis of ketohexokinase. By downregulating this enzyme, Senvesiran plays a critical role in metabolic research, particularly in studies related to fructose metabolism and its implications in metabolic disorders. This reagent is essential for investigating the pathways involved in sugar regulation and potential therapeutic interventions for conditions such as obesity and type 2 diabetes.

Cat. No.

A75838

CAS No.

2266591-83-5

Nedosiran

Synonyms: DCR-PHXC

Nedosiran is an RNA interference agent that selectively targets lactate dehydrogenase (LDH). It has shown potential as a therapeutic for primary hyperoxaluria (PH), particularly in cases progressing to end-stage renal disease (ESRD). As a GalNAc-dsRNA conjugate, Nedosiran facilitates tissue-specific delivery, making it a promising candidate for research applications in metabolic disorders and kidney function studies.

Cat. No.

A77042

CAS No.

2639640-84-7

Bezeparsen

Bezeparsen is a potent PCSK9 synthesis inhibitor that regulates LDL cholesterol levels by enhancing hepatic LDL receptor activity. It plays a critical role in lipid metabolism and is used in cardiovascular research to study cholesterol homeostasis and potential therapeutic interventions for hyperlipidemia. This compound is valuable for investigations focusing on the modulation of cholesterol levels and cardiovascular disease management.

Cat. No.

A77060

CAS No.

2252375-14-5

Cepadacursen sodium

Synonyms: CIVI007 sodium

Cepadacursen sodium is an antisense oligonucleotide (ASO) that targets and inhibits the synthesis of proprotein convertase subtilisin/kexin type 9 (PCSK9) in the liver. By reducing PCSK9 levels, it plays a critical role in regulating cholesterol metabolism. This reagent is particularly relevant for research focused on hypercholesterolemia and the prevention strategies for atherosclerotic cardiovascular disease (ASCVD).

Cat. No.

A77064

CAS No.

2604215-74-7

AZD8233

AZD8233 is an antisense oligonucleotide (ASO) that targets proprotein convertase subtilisin/kexin type 9 (PCSK9) to inhibit its protein synthesis. By reducing levels of PCSK9, AZD8233 enhances the availability of LDL receptors, which facilitates the clearance of low-density lipoprotein (LDL) from circulation and contributes to lowering LDL cholesterol (LDL-C) levels. This compound is applicable in research focused on lipid metabolism and cardiovascular disease.

Cat. No.

A77260

CAS No.

3056860-75-1

Pexamsiran

Pexamsiran is a small interfering RNA (siRNA) that effectively inhibits plasminogen synthesis. This compound is instrumental in research related to hereditary hemorrhagic telangiectasia, providing insights into the molecular mechanisms underlying this condition and potential therapeutic approaches. Pexamsiran's ability to modulate plasminogen levels makes it a valuable reagent for studying vascular biology and related disorders.

Cat. No.

A77262

CAS No.

3034567-90-0

Edutirsen

Edutirsen is an antisense oligonucleotide that specifically targets and inhibits the synthesis of patatin-like phospholipase domain containing protein 3 (PNPLA3). By reducing PNPLA3 levels, Edutirsen can elucidate the role of this enzyme in lipid metabolism and associated metabolic pathways. This reagent is particularly useful in research focused on liver disease, obesity, and related metabolic disorders, facilitating the understanding of PNPLA3’s contribution to these conditions.

Cat. No.

A77263

CAS No.

2654847-29-5

Pivicasiran

Pivicasiran is a synthetic small interfering RNA (siRNA) targeting adiponutrin synthesis. This compound effectively reduces the expression of adiponutrin, a protein implicated in lipid metabolism and obesity-related diseases. Pivicasiran is valuable for research applications focused on understanding metabolic disorders and the regulation of fat storage and energy homeostasis.

Cat. No.

A77623

CAS No.

1698048-23-5

Baliforsen

Synonyms: ISIS 598769; IONIS 598769; BIIB 065; ISIS-DMPK-2.5Rx

Baliforsen is an antisense oligonucleotide composed of 16 nucleotides, specifically designed to target the mRNA of myotonic dystrophy protein kinase (DMPK). This compound plays a crucial role in the study of myotonic dystrophy by modulating the expression of pathogenic transcripts. Its application in research facilitates a deeper understanding of the molecular mechanisms underlying myotonic dystrophy, offering potential insights for therapeutic advancements.

Cat. No.

A77765

CAS No.

2646704-10-9

Manusiran

Synonyms: Divesiran

Manusiran (SLN124) is a GalNAc-conjugated 19-mer siRNA targeting TMPRSS6 (transmembrane protease serine 6). It effectively reduces plasma iron levels while increasing hepcidin expression in healthy individuals. This compound is primarily used in research applications focused on iron metabolism and disorders related to iron homeostasis.

Cat. No.

A77793

CAS No.

2375562-54-0

Belcesiran

Synonyms: DCR-S1459

Belcesiran is a small interfering RNA (siRNA) that targets and inhibits alpha-1 antitrypsin (A1AT) production. By downregulating A1AT synthesis in the liver, it promotes the understanding of A1AT-related conditions and potential therapeutic interventions. This compound is primarily utilized in research focused on liver function and A1AT deficiency disorders.