Oligonucleotide Therapeutics (ASO/siRNA)

Cat. No.

A25500

CAS No.

1639324-58-5

Inclisiran

Synonyms: ALN-PCSsc

Inclisiran is a double-stranded small interfering RNA (siRNA) that specifically targets and inhibits the transcription of proprotein convertase subtilisin/kexin type 9 (PCSK9). By reducing PCSK9 levels, Inclisiran effectively modulates lipid metabolism and demonstrates anti-inflammatory properties, including the inhibition of pyroptosis and a decrease in NLRP3, cleaved caspase-1, IL-1β, and IL-18. This reagent is valuable for research applications focused on hyperlipidemia and cardiovascular diseases (CVD), as it supports studies aimed at understanding lipid regulation and atherosclerosis.

Cat. No.

A30343

CAS No.

1402357-06-5

Danvatirsen

Synonyms: AZD9150

Danvatirsen is an antisense oligonucleotide that specifically inhibits STAT3, a transcription factor involved in cell survival and proliferation. This compound reduces cell viability and promotes apoptosis in leukemia cell lines, while downregulating the expression of endogenous STAT3 and its downstream target genes. Additionally, Danvatirsen effectively diminishes proliferation and tumorigenicity in models of neuroblastoma and lymphoma. In vivo studies demonstrate its capacity to inhibit tumor growth rates in mouse models of neuroblastoma, lymphoma, and non-small cell lung cancer. Danvatirsen is applicable in research focused on lymphoma, myelodysplastic syndrome, acute myeloid leukemia, and neuroblastoma.

Cat. No.

A33800

CAS No.

1146887-67-3

Aganirsen

Aganirsen is a 25-mer DNA antisense oligonucleotide that functions as an inhibitor of the insulin receptor by silencing the expression of insulin receptor substrate-1 (IRS-1). This compound exhibits significant biological activity in modulating insulin signaling pathways, making it a valuable tool for research into metabolic disorders and insulin resistance. Its application extends to studies investigating the role of IRS-1 in various cellular processes and diseases.

Cat. No.

A35187

CAS No.

2272983-11-4

Sefaxersen sodium

Synonyms: IONIS-FB-LRx sodium; RG6299 sodium; ISIS 696844 sodium

Sefaxersen sodium is a specific antisense oligonucleotide (ASO) that targets complement factor B (CFB). It effectively reduces circulating levels of CFB, making it a valuable tool for research into geographic atrophy (GA). This compound facilitates the exploration of therapeutic strategies aimed at modulating the complement pathway in ocular diseases.

Cat. No.

A35204

CAS No.

2272975-74-1

Sefaxersen

Synonyms: IONIS-FB-LRx; RG6299; ISIS 696844

Sefaxersen is a specific antisense oligonucleotide (ASO) that targets complement factor B (CFB), effectively decreasing its circulating levels. This compound plays a critical role in modulating the complement pathway and has significant implications for research related to geographic atrophy (GA). Researchers can utilize Sefaxersen to investigate the therapeutic potential in conditions characterized by complement dysregulation.

Cat. No.

A37461

CAS No.

915443-09-3

Apatorsen sodium

Synonyms: OGX-427 sodium

Apatorsen sodium is a 2'-methoxyethyl-modified antisense oligonucleotide targeting Hsp27. It effectively reduces Hsp27 mRNA and protein levels, disrupting stress-induced cytoprotective functions, promoting apoptosis, inhibiting tumor growth, and preventing metastasis. This compound is relevant for research applications in non-small cell lung cancer, castration-resistant prostate cancer, breast cancer, ovarian cancer, and bladder cancer.

Cat. No.

A37515

CAS No.

1002331-21-6

Apatorsen

Synonyms: OGX-427

Apatorsen is a 2'-methoxyethyl-modified antisense oligonucleotide that functions as a Heat Shock Protein 27 (Hsp27) inhibitor. By reducing Hsp27 mRNA and protein levels, Apatorsen disrupts stress-induced cytoprotective mechanisms, promotes apoptosis, inhibits tumor growth, and prevents metastasis. This compound is particularly relevant for research focused on non-small cell lung cancer, castration-resistant prostate cancer, breast cancer, ovarian cancer, and bladder cancer.

Cat. No.

A37797

CAS No.

2172851-58-8

Pelacarsen sodium

Synonyms: AKCEA-APO(a)-LRx sodium; ISIS 681257 sodium; TQJ230 sodium

Pelacarsen sodium is an apolipoprotein(a) inhibitor that functions as a GalNAc3-conjugated 2′-MOE-modified antisense oligonucleotide. By specifically targeting and reducing levels of apolipoprotein(a), Pelacarsen sodium plays a significant role in lipid metabolism and cardiovascular disease research. It is valuable for studies aimed at exploring new therapies for atherosclerosis and other related conditions.

Cat. No.

A38028

CAS No.

1637637-70-7

Pelacarsen

Synonyms: AKCEA-APO(a)-LRx; ISIS 681257; TQJ230

Pelacarsen is an apolipoprotein(a) inhibitor that functions as a GalNAc3-conjugated 2′-MOE-modified antisense oligonucleotide. This compound effectively reduces levels of apolipoprotein(a), making it valuable in research focused on lipid metabolism and cardiovascular diseases. It is commonly utilized in studies addressing dyslipidemia and atherogenic conditions linked to elevated lipoprotein(a) levels.

Cat. No.

A46154

CAS No.

331257-53-5

Aprinocarsen sodium

Synonyms: ISIS 3521 sodium

Aprinocarsen sodium is a specific antisense oligonucleotide that targets protein kinase C-alpha (PKC-α), functioning as a potent inhibitor. This 20-mer oligonucleotide modulates cellular processes such as differentiation and proliferation. In vivo studies demonstrate its ability to inhibit the growth of human tumor cell lines in nude mice, highlighting its potential utility as a chemotherapeutic agent in the treatment of various human cancers.

Cat. No.

A46296

CAS No.

1386913-72-9

Patisiran sodium

Patisiran sodium is a double-stranded small interfering RNA that targets a specific sequence within the messenger RNA of transthyretin (TTR). It effectively inhibits the hepatic synthesis of both mutant and wild-type TTR. This reagent is primarily used in research applications related to hereditary TTR amyloidosis, providing valuable insights into the disorder's underlying mechanisms.

Cat. No.

A46297

CAS No.

1639325-44-2

Givosiran sodium

Synonyms: ALN-AS1 sodium

Givosiran sodium is a small interfering RNA that specifically inhibits hepatic aminolevulinate synthase 1 (ALAS1) by targeting its messenger RNA. This downregulation leads to a significant reduction in the accumulation of neurotoxic metabolites, such as δ-aminolevulinic acid (ALA) and porphobilinogen (PBG). Givosiran sodium exhibits strong inhibitory effects on ALAS1 in preclinical models, including mouse, rat, and cynomolgus monkey studies. It is valuable for research focused on acute hepatic porphyria (AHP) and related disorders.

Cat. No.

A46300

CAS No.

1639325-43-1

Givosiran

Synonyms: ALN-AS1

Givosiran is a small interfering RNA that specifically inhibits hepatic aminolevulinate synthase 1 (ALAS1) by targeting its messenger RNA. This inhibition leads to decreased levels of the neurotoxic compounds δ-aminolevulinic acid (ALA) and porphobilinogen (PBG). Givosiran exhibits significant efficacy in reducing ALAS1 activity in various preclinical models, including mouse, rat, and cynomolgus monkey studies. This reagent is valuable for research focused on acute hepatic porphyria (AHP) and its associated metabolic disturbances.

Cat. No.

A46301

CAS No.

1834612-06-4

Lumasiran sodium

Lumasiran sodium is a potent glycolate oxidase inhibitor that employs RNA interference (RNAi) mechanisms to effectively decrease hepatic oxalate production. By targeting this specific enzyme, Lumasiran sodium reduces urinary oxalate excretion, addressing the underlying cause of progressive kidney failure in patients with primary hyperoxaluria type 1 (PH1). This therapeutic approach offers significant promise for managing hyperoxalemia and its associated complications.

Cat. No.

A46302

CAS No.

1834610-13-7

Lumasiran

Synonyms: ALN-G01

Lumasiran is a small interfering RNA (siRNA) that specifically inhibits glycolate oxidase, leading to a reduction in hepatic oxalate production. By silencing the gene responsible for glycolate oxidase expression, Lumasiran effectively lowers the synthesis of oxalate, a toxic metabolite implicated in the clinical manifestations of Primary Hyperoxaluria type 1 (PH1). This compound is of particular interest in studies aimed at identifying therapeutic strategies for managing PH1 and related metabolic disorders.

Cat. No.

A50655

CAS No.

160369-77-7

Fomivirsen sodium

Synonyms: ISIS-2922 sodium

Fomivirsen sodium is an antisense 21-mer phosphorothioate oligonucleotide that selectively targets the cytomegalovirus (CMV) immediate-early 2 protein mRNAs. This antiviral agent is primarily utilized in research related to CMV retinitis, particularly in the context of AIDS-related studies. By binding to and facilitating the degradation of CMV mRNAs, Fomivirsen sodium effectively inhibits viral proliferation, making it a valuable tool for investigating CMV pathogenesis and potential therapeutic interventions.

Cat. No.

A50660

CAS No.

144245-52-3

Fomivirsen

Synonyms: ISIS-2922

Fomivirsen is an antisense phosphorothioate oligonucleotide that acts as a cytomegalovirus (CMV) inhibitor by selectively binding to and degrading the mRNA encoding the immediate-early 2 protein of CMV. This mechanism effectively inhibits viral replication, making Fomivirsen a valuable reagent for studying CMV retinitis and other associated CMV diseases. Its application is pertinent in research aimed at understanding CMV pathogenesis and developing therapeutic interventions.

Cat. No.

A50927

CAS No.

2538784-49-3

Xalnesiran sodium

Xalnesiran sodium is a small interfering RNA (siRNA) designed to inhibit hepatitis B virus (HBV) replication. It provides a valuable tool for researching chronic HBV infections and understanding the virus's biology. This compound is particularly relevant in studies aimed at developing therapeutic strategies for managing HBV-related diseases.

Cat. No.

A50970

CAS No.

2538784-48-2

Xalnesiran

Xalnesiran is a small interfering RNA (siRNA) specifically designed to inhibit hepatitis B virus (HBV) replication. By targeting and degrading HBV RNA, it plays a crucial role in reducing viral load and the associated liver pathology. Xalnesiran is valuable for research applications focused on chronic hepatitis B treatment strategies and the investigation of RNA interference mechanisms in viral infections.

Cat. No.

A51069

CAS No.

629167-92-6

Mipomersen sodium

Synonyms: ISIS 301012

Mipomersen sodium is an antisense oligonucleotide targeting apolipoprotein B (apoB), effectively inhibiting its expression. This compound has demonstrated potential anti-hepatitis C virus (HCV) activity by reducing the infectivity of HCV. Mipomersen sodium is primarily utilized in research focused on homozygous familial hypercholesterolemia (HoFH), contributing to a deeper understanding of lipid metabolism and related disorders.

Cat. No.

A51080

CAS No.

1000120-98-8

Mipomersen

Synonyms: ISIS 301012 free base

Mipomersen is an antisense oligonucleotide that specifically inhibits apolipoprotein B (apoB) synthesis. This compound is primarily utilized in research focused on homozygous familial hypercholesterolemia (HoFH), demonstrating significant efficacy in lipid regulation. Additionally, Mipomersen exhibits anti-hepatitis C virus (HCV) activity, contributing to reduced infectivity of the virus. Its unique mechanism makes it a valuable tool for studying lipid disorders and viral infections.

Cat. No.

A51103

CAS No.

1072874-90-8

Miravirsen

Synonyms: SPC-3649

Miravirsen, a specific inhibitor of miR-122, targets the Argonaute (Ago) protein complex to impede the biogenesis of miR-122. This 15-nucleotide locked nucleic acid-modified phosphorothioate antisense oligonucleotide demonstrates significant antiviral activity against hepatitis C virus (HCV) replication. Miravirsen serves as a valuable tool for research focused on HCV infection and its associated mechanisms.

Cat. No.

A57454

CAS No.

185229-68-9

Alicaforsen

Synonyms: ISIS-2302

Alicaforsen is an oligonucleotide that functions as an inhibitor of human ICAM-1 mRNA. By hybridizing to specific regions of the mRNA, Alicaforsen effectively reduces the expression levels of ICAM-1, a key cell adhesion molecule involved in inflammatory responses. This reagent is particularly relevant for research applications focusing on psoriasis, rheumatoid arthritis, and inflammatory bowel disease.

Cat. No.

A57460

CAS No.

331257-52-4

Alicaforsen sodium

Synonyms: ISIS-2302 sodium

Alicaforsen sodium is an oligonucleotide that serves as an inhibitor of intercellular adhesion molecule 1 (ICAM-1) mRNA. By hybridizing to specific mRNA sequences, it effectively reduces ICAM-1 expression levels. This compound is valuable for research into inflammatory conditions, particularly in the study of psoriasis, rheumatoid arthritis, and inflammatory bowel disease.

Cat. No.

A57824

CAS No.

2953012-32-1

Farabursen

Synonyms: RGLS8429; RG1015

Farabursen is a selective inhibitor of miR-17, designed to penetrate the blood-brain barrier. This compound effectively derepresses the target genes Pkd1 and Pkd2, leading to enhanced levels of polycystin-1 (PC1) and polycystin-2 (PC2), while simultaneously reducing cyst growth. In preclinical mouse models, Farabursen has been demonstrated to decrease renal cyst growth, lower kidney weight-to-body weight ratio, reduce cyst and proliferation indices, and decrease blood urea nitrogen levels. This reagent is valuable for research focused on autosomal dominant polycystic kidney disease.