Oligonucleotide Therapeutics (ASO/siRNA)

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.

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.

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.

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.

Cat. No.

A77977

CAS No.

2273007-95-5

Sapablursen

Synonyms: IONIS-TMPRSS6-Lrx; ISIS 702843

Sapablursen is an antisense oligonucleotide that specifically inhibits TMPRSS6, leading to enhanced expression of hepcidin, a crucial regulator of iron metabolism. This compound is valuable for research focused on blood disorders, particularly those related to iron regulation and homeostasis. Its ability to modulate hepcidin levels makes it a potential tool for studying various pathophysiological conditions associated with dysregulated iron metabolism.

Cat. No.

A77978

CAS No.

2273008-00-5

Sapablursen sodium

Synonyms: IONIS-TMPRSS6-Lrx sodium; ISIS 702843 sodium

Sapablursen sodium is an antisense oligonucleotide that specifically inhibits TMPRSS6, leading to enhanced expression of hepcidin, a crucial regulator of iron homeostasis. This compound is valuable for investigating its effects on iron regulation and potential applications in the study of blood disorders. Researchers can utilize Sapablursen sodium to explore therapeutic strategies for conditions related to iron dysregulation.

Cat. No.

A79388

CAS No.

2131025-75-5

Eplontersen sodium

Eplontersen sodium is a triantennary N-acetyl galactosamine (GalNAc3-7a)-conjugated antisense oligonucleotide that targets transthyretin (TTR) mRNA, effectively inhibiting the production of both variant and wild-type TTR protein. By reducing TTR levels, Eplontersen sodium plays a crucial role in addressing conditions associated with amyloid TTR (ATTR) amyloidosis, where misfolded TTR leads to the formation of amyloid fibrils affecting the heart and peripheral nerves. This compound is pivotal for research in therapeutic strategies aimed at managing hereditary and wild-type forms of amyloidosis.

Cat. No.

A79389

CAS No.

1492984-65-2

Inotersen

Synonyms: GSK-2998728; ISIS-420915

Inotersen is a 2'-O-methoxyethyl-modified antisense oligonucleotide targeting transthyretin (TTR) to inhibit its production. By binding to TTR mRNA, Inotersen promotes RNase H1-mediated degradation, leading to a decrease in both mutant and wild-type TTR synthesis in the liver. This reduction significantly mitigates amyloid fiber deposition, making Inotersen a valuable tool in research focused on hereditary transthyretin amyloidosis, including its related complications such as polyneuropathy and cardiomyopathy. Caution is advised, as high doses may lead to specific toxicities, including inflammation or tumor development in certain animal models.

Cat. No.

A79391

CAS No.

1637600-16-8

Eplontersen

Eplontersen is a triantennary N-acetyl galactosamine (GalNAc3-7a) conjugated antisense oligonucleotide that selectively targets transthyretin (TTR) mRNA. By inhibiting the synthesis of both variant and wild-type TTR proteins, Eplontersen plays a crucial role in disrupting the misfolding of TTR, which contributes to the formation of amyloid fibrils associated with ATTR amyloidosis. This compound is valuable for research applications focused on amyloid-related diseases and exploring therapeutic strategies for TTR-related pathologies.

Cat. No.

A79425

CAS No.

2411937-33-0

Movronersen

Synonyms: BIIB-101; ION-464

Movronersen is an antisense oligonucleotide that specifically inhibits α-synuclein expression. By targeting the mRNA of α-synuclein, this compound effectively reduces its protein levels, making it a valuable tool for research into neurodegenerative diseases such as Parkinson's disease. Movronersen can aid in the study of α-synuclein's role in pathology and the development of potential therapeutic strategies.

Cat. No.

A84904

CAS No.

1152798-48-5

Pixofisiran

Pixofisiran is a small interfering RNA (siRNA) that specifically inhibits transforming growth factor-beta 1 (TGF-β1). This compound plays a significant role in modulating fibrosis and inflammation, making it a valuable tool for research into these biological processes. Applications include studies related to cancer, tissue repair, and the mechanistic exploration of TGF-β1 signaling pathways.