Oligonucleotide Therapeutics (ASO/siRNA)

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.

A78286

CAS No.

2572991-83-2

Mivelsiran

Synonyms: ALN-APP

Mivelsiran is a small interfering RNA (siRNA) that specifically reduces the synthesis of amyloid-beta precursor protein (APP). This compound is primarily utilized in research focusing on Alzheimer's disease, providing insights into the mechanisms of amyloid plaque formation and the associated neurodegenerative processes. Mivelsiran serves as a valuable tool for exploring therapeutic strategies aimed at mitigating APP-related pathologies.

Cat. No.

A78748

CAS No.

864021-55-6

Monarsen

Synonyms: EN101; ODN 7040; BL 7040

Monarsen is a synthetic 20-base antisense oligodeoxynucleotide targeting the human acetylcholinesterase (AChE) gene. This compound is utilized in research related to autoimmune myasthenia gravis (MG), a neuromuscular disorder characterized by autoantibodies against the acetylcholine receptor (AChR). Monarsen's mechanism of action allows for the inhibition of AChE expression, making it a valuable tool for studying disease mechanisms and potential therapeutic interventions in MG.

Cat. No.

A78950

CAS No.

3059403-91-4

Brezosiran

Brezosiran is a small interfering RNA (siRNA) designed to reduce the synthesis of huntingtin protein. By targeting the mRNA coding for huntingtin, it effectively decreases its expression, making it a valuable tool in research related to Huntington's disease and other neurodegenerative disorders. This compound enables scientists to investigate the molecular mechanisms underlying these conditions and assess potential therapeutic strategies.

Cat. No.

A78953

CAS No.

1709886-74-7

Tominersen

Synonyms: RG6042; IONIS-HTTRx

Tominersen is a second-generation 2′-O-(2-methoxyethyl) antisense oligonucleotide that targets huntingtin protein (HTT) mRNA, leading to significant suppression of HTT production. This compound has demonstrated potential in enhancing survival and reducing brain atrophy in mouse models. Tominersen is valuable for research applications focused on Huntington's disease (HD), providing insights into therapeutic strategies for neurodegenerative disorders.

Cat. No.

A78954

CAS No.

2072901-32-5

Rovanersen

Synonyms: WVE-120101

Rovanersen is an antisense oligonucleotide designed to selectively target mutated mRNA transcripts of the huntingtin (HTT) gene, while sparing the normal mRNA. This specificity helps to inhibit the production of the abnormal Huntingtin protein, which is implicated in Huntington's disease pathology. Rovanersen is valuable for research related to Huntington’s disease and for investigating therapeutic strategies aimed at reducing the detrimental effects of mutant HTT expression.

Cat. No.

A78955

CAS No.

2231347-41-2

Rovanersen sodium

Synonyms: WVE-120101 sodium

Rovanersen sodium is an antisense oligonucleotide that selectively targets mutated mRNA of the huntingtin (HTT) gene, allowing for the inhibition of faulty Huntingtin protein production while sparing the healthy mRNA. This compound is essential for research into Huntington's disease, providing insights into potential therapeutic approaches and mechanisms underlying the disease pathology.

Cat. No.

A79173

CAS No.

2834724-51-3

Erisonersen

Synonyms: ION 717

Erisonersen is an antisense oligonucleotide that targets prion protein synthesis. Its primary mechanism involves reducing the expression of prion proteins, making it a valuable tool for investigating prion diseases. This reagent is essential for research applications focused on understanding the molecular mechanisms and pathophysiology of prion-related disorders.

Cat. No.

A79181

CAS No.

2834724-81-9

Erisonersen sodium

Synonyms: ION 717 sodium

Erisonersen sodium targets prion protein synthesis through its action as an antisense oligonucleotide. This compound effectively reduces the production of prion proteins, making it a valuable tool in the research of prion diseases. Its applications extend to understanding the molecular mechanisms underlying neurodegenerative diseases associated with prion pathogenesis.

Cat. No.

A79329

CAS No.

2304711-06-4

Diranersen

Synonyms: IONIS-MAPTRx; BIIB080; ISIS 814907

Diranersen is a Tau-lowering antisense oligonucleotide designed to target and inhibit tau protein expression. This compound holds potential for advancing research in Alzheimer’s disease and related neurodegenerative conditions. By reducing tau levels, Diranersen aims to facilitate studies focused on tau pathology and its implications in Alzheimer's progression.

Cat. No.

A79387

CAS No.

1432726-13-0

Inotersen sodium

Synonyms: ISIS-420915 sodium

Inotersen sodium is a 2′-O-methoxyethyl-modified antisense oligonucleotide that targets the transthyretin (TTR) RNA transcript to inhibit the production of TTR protein. This reduction in TTR levels makes Inotersen sodium a valuable tool for studying hereditary TTR amyloidosis polyneuropathy. Its mechanism of action provides insight into potential therapeutic strategies for related disorders.

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.

A79397

CAS No.

1438322-82-7

Revusiran

Synonyms: ALN-TTRSC

Revusiran is a first-generation short interfering RNA (siRNA) that targets and inhibits transthyretin (TTR) mRNA. By reducing TTR expression, Revusiran is instrumental in studying TTR-mediated amyloidosis and its associated pathologies. This reagent serves as a valuable tool for researchers investigating the molecular mechanisms of amyloid formation and potential therapeutic interventions.

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.

Cat. No.

B18744

CAS No.

2756821-85-7

Zotresiran

Zotresiran is a small interfering RNA (siRNA) designed to reduce the synthesis of xanthine dehydrogenase, an enzyme involved in purine metabolism. By downregulating this target, Zotresiran can modulate uric acid levels, making it valuable for research in conditions such as gout and hyperuricemia. Its application in gene silencing offers researchers a powerful tool for investigating metabolic disorders and developing therapeutic strategies.

Cat. No.

B19034

CAS No.

2701584-16-7

Salanersen

Synonyms: BIIB115; ION306

Salanersen is an antisense oligonucleotide that specifically targets the survival motor neuron 2 (SMN2) gene, effectively reducing its expression. This compound plays a crucial role in the investigation of spinal muscular atrophy (SMA) by modulating SMN2 levels. Its application is essential for understanding the pathophysiology of SMA and developing potential therapeutic strategies.

Cat. No.

B19704

CAS No.

2756001-75-7

Elsunersen

Synonyms: PRAX-222

Elsunersen is an antisense oligonucleotide designed to selectively reduce the expression of the SCN2A gene, which encodes the voltage-gated sodium channel Nav1.2. This compound is instrumental in researching SCN2A developmental and epileptic encephalopathy (SCN2A-DEE), a neurological disorder characterized by severe seizures and developmental impairments. By targeting SCN2A, Elsunersen facilitates the exploration of therapeutic approaches for conditions arising from SCN2A mutations.

Cat. No.

B19894

CAS No.

2757908-12-4

Marpinersen

Marpinersen is an antisense oligonucleotide targeting ataxin-2 synthesis. It plays a crucial role in reducing ataxin-2 levels, making it a valuable tool for studying the pathogenesis of amyotrophic lateral sclerosis (ALS). This compound is instrumental in investigating the molecular mechanisms underlying ALS and potential therapeutic interventions.

Cat. No.

B22085

CAS No.

2766131-24-0

Lepodisiran sodium

Synonyms: LY3819469 sodium

Lepodisiran sodium is a small interfering RNA (siRNA) that specifically targets lipoprotein(a) to reduce its levels in the bloodstream. By inhibiting the synthesis of this atherogenic lipoprotein, Lepodisiran sodium plays a crucial role in cardiovascular research and may contribute to the development of innovative therapies for conditions related to elevated lipoprotein(a). This reagent is valuable for studies focused on lipid metabolism, cardiovascular disease mechanisms, and gene silencing techniques.