Oligonucleotide Therapeutics (ASO/siRNA)

Cat. No.

A50901

CAS No.

2563929-84-8

Bepirovirsen sodium

Synonyms: ISIS 505358 sodium

Bepirovirsen sodium is an antisense oligonucleotide designed to specifically target and inhibit all HBV messenger RNAs. This compound effectively reduces HBV-derived RNAs, viral DNA, and associated proteins, making it a valuable tool in the research of chronic HBV infection. The binding site sequence for Bepirovirsen sodium is GCACTTCGCTTCACCTCTGC, underscoring its specificity and utility in virology studies.

Cat. No.

A50905

CAS No.

1403787-62-1

Bepirovirsen

Synonyms: ISIS 505358

Bepirovirsen is an antisense oligonucleotide specifically designed to target all hepatitis B virus (HBV) messenger RNAs. This compound effectively reduces levels of HBV-derived RNAs, HBV DNA, and associated viral proteins. Bepirovirsen is utilized in research focused on chronic HBV infection, offering insights into potential therapeutic strategies against this viral disease.

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.

A50957

CAS No.

2648009-64-5

Elebsiran

Synonyms: VIR-2218

Elebsiran is a small interfering RNA (siRNA) that targets and degrades hepatitis B virus (HBV) and hepatitis D virus (HDV) RNA transcripts. This agent significantly reduces HBV surface antigen (HBsAg) levels and viral load, indicating its potent antiviral activity. By binding to the asialoglycoprotein receptor (ASGPR) on liver cells via the GalNAc ligand, Elebsiran enables liver-targeted delivery while ensuring improved liver safety. This reagent is valuable for research focused on chronic HBV and HDV infections.

Cat. No.

A50960

CAS No.

3061406-96-7

Ozisiran

Ozisiran is a small interfering RNA (siRNA) that targets hepatitis B virus (HBV) RNA transcripts, leading to a reduction in viral load. This compound exhibits potent antiviral activity, making it a valuable tool for research into HBV pathogenesis and therapeutic interventions. Its application in studies of antiviral mechanisms can enhance understanding of HBV infection and facilitate the development of novel antiviral strategies.

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.

A51002

CAS No.

2202783-35-3

Daplusiran

Daplusiran is a small interfering RNA (siRNA) designed to target and promote the degradation of hepatitis B virus (HBV) mRNAs. By inhibiting the replication of HBV, it serves as a vital tool in the development of therapeutic strategies for HBV-related diseases. Research applications include studies focused on liver disease and antiviral interventions.

Cat. No.

A51017

CAS No.

2202789-48-6

Tomligisiran

Tomligisiran is a small interfering RNA (siRNA) designed to target hepatitis B virus (HBV) mRNAs, promoting their degradation and effectively inhibiting HBV replication. This reagent plays a significant role in research focused on antiviral strategies and therapeutic approaches for HBV infections. Its utility in silencing HBV-specific RNA makes it a valuable tool for studying the molecular mechanisms of viral replication and pathogenesis.

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.

A51277

CAS No.

153021-75-1

Trecovirsen

Synonyms: GEM91

Trecovirsen is an antiviral agent that targets HIV gag mRNA by hybridizing with its complementary sequence at the initiation site. This interaction leads to the induction of a reversible, dose-dependent prolongation of activated partial thromboplastin time due to its polyanionic properties. Trecovirsen is applicable for research on HIV infection, providing insights into the mechanisms of viral replication and potential therapeutic strategies.

Cat. No.

A51884

CAS No.

151356-08-0

Afovirsen

Synonyms: ISIS 2105

Afovirsen is a 20-mer phosphorothioate oligonucleotide that specifically targets the mRNA sequence for the translation initiation codon of the E2 protein, essential for the replication of Human Papillomavirus (HPV) types 6 and 11. By disrupting E2 protein synthesis, Afovirsen exhibits potential antiviral activity against HPV, making it a valuable tool for research focused on viral replication mechanisms and therapeutic interventions in HPV-related conditions.

Cat. No.

A51885

CAS No.

138330-98-0

Afovirsen sodium

Synonyms: ISIS 2105 sodium

Afovirsen sodium is a 20-mer phosphorothioate oligonucleotide targeting human papillomavirus (HPV). It is designed to be complementary to the mRNA sequence of the translation initiation codon of the E2 protein, which plays a critical role in the replication of HPV types 6 and 11. This reagent has potential applications in research focused on HPV-related pathogenesis and antiviral strategies.

Cat. No.

A53169

CAS No.

1386946-83-3

Asvasiran sodium

Synonyms: ALN-RSV01 sodium

Asvasiran sodium is a small interfering RNA (siRNA) designed to target the N gene of the respiratory syncytial virus (RSV). This compound effectively inhibits viral replication, making it a valuable tool for researching RSV infections. Its application in gene silencing provides insights into viral mechanisms and potential therapeutic strategies.

Cat. No.

A53196

CAS No.

870094-26-1

Asvasiran

Synonyms: ALN-RSV01

Asvasiran is a small interfering RNA (siRNA) designed to target the N gene of the respiratory syncytial virus (RSV). By inhibiting the expression of this critical viral gene, Asvasiran effectively reduces viral replication. This reagent is valuable for investigating RSV biology and potential therapeutic interventions in respiratory infections caused by this virus.

Cat. No.

A53634

CAS No.

2929240-20-8

Delpacibart zotadirsen

Synonyms: Del-zota; AOC 1044

Delpacibart zotadirsen is an antibody oligonucleotide conjugate that targets the transferrin receptor 1 (TfR1). This innovative reagent is designed to deliver phosphorodiamidate morpholino oligomers (PMOs) specifically to skeletal muscle and heart tissue, facilitating the skipping of exon 44 in the dystrophin gene to promote the production of near-full-length dystrophin. Delpacibart zotadirsen is a valuable tool for investigating myotonic dystrophy type 1 (DM1) and advancing research in therapeutic approaches for muscle disorders.

Cat. No.

A53635

CAS No.

3028750-59-3

Rostudirsen

Synonyms: DYNE-251 Oligomer

Rostudirsen is a phosphorodiamidate morpholino oligomer designed to target Duchenne muscular dystrophy (DMD) by promoting exon skipping in muscle cells. It functions through the selective delivery of therapeutic oligonucleotides to skeletal muscle, facilitating the production of internally shortened dystrophin protein, which is crucial for muscle integrity and function. This reagent is instrumental in advancing research on DMD gene therapies and evaluating the efficacy of treatments aimed at restoring dystrophin expression in affected tissues.

Cat. No.

A53636

CAS No.

3028750-61-7

Basivarsen

Synonyms: DYNE-101 oligomer

Basivarsen (DYNE-101 oligomer) is an antisense oligonucleotide designed to target mutant DMPK RNA for RNase H-mediated degradation, facilitating the correction of splicing defects. Its primary application is in the research of myotonic dystrophy type 1 (DM1), providing a valuable tool for investigating disease mechanisms and potential therapeutic approaches. This compound enhances the understanding of RNA-mediated pathogenesis and aids in the development of targeted treatments for DM1.

Cat. No.

A53806

CAS No.

2169310-72-7

Teprasiran sodium

Synonyms: QPI-1002 sodium

Teprasiran sodium is a small interfering RNA (siRNA) that specifically inhibits p53 activity, modulating p53-mediated apoptotic pathways. This reagent is vital for studying the cellular response to stress and mechanisms involved in acute kidney injury (AKI). Teprasiran sodium facilitates research into potential therapeutic strategies for AKI by temporarily blocking p53-related cell death.

Cat. No.

A53814

CAS No.

1231737-88-4

Teprasiran

Synonyms: QPI-1002

Teprasiran (QPI-1002) is a small interfering RNA that targets p53, a critical regulator of apoptosis. By temporarily inhibiting p53-mediated cell death, Teprasiran is primarily utilized in research to investigate mechanisms underlying acute kidney injury (AKI). Its ability to modulate cellular responses to stress makes it a valuable tool for studies focused on kidney pathology and potential therapeutic interventions.

Cat. No.

A53832

CAS No.

872847-66-0

Cenersen

Synonyms: EL625

Cenersen is an oligonucleotide that specifically targets TP53. It effectively inhibits the activity of gain-of-function mutants of TP53, thereby enhancing the sensitivity of lymphoma cells to cytotoxic chemotherapy in vitro. This reagent is particularly valuable for research applications focused on chronic lymphocytic leukemia (CLL).

Cat. No.

A53984

CAS No.

1065019-70-6

Gataparsen

Synonyms: LY 2181308

Gataparsen is an antisense oligonucleotide that specifically targets survivin mRNA, aiming to inhibit its expression. This intervention has shown potential antitumor activity, making it a valuable tool in cancer research. Gataparsen can be utilized to study the role of survivin in cell survival and proliferation, and to explore therapeutic strategies for tumor suppression.

Cat. No.

A55119

CAS No.

2055732-84-6

Viltolarsen

Synonyms: NS-065/NCNP-01

Viltolarsen is a phosphorodiamidate morpholino antisense oligonucleotide designed to target dystrophin mRNA by binding to exon 53. This mechanism promotes exon skipping, leading to the restoration of the open-reading frame and the production of a truncated dystrophin protein that preserves essential functional elements. Viltolarsen is primarily utilized in research related to Duchenne muscular dystrophy (DMD), making it a valuable tool for advancing therapeutic strategies in this condition.

Cat. No.

A55240

CAS No.

2642382-41-8

Ultevursen

Synonyms: QR-421a

Ultevursen (QR-421a) is a single-stranded RNA oligonucleotide that acts as a splice-switching agent, designed to promote the skipping of exon 13 in the USH2A gene, which encodes usherin. This mechanism aims to halt vision loss associated with retinitis pigmentosa caused by mutations within this exon. Ultevursen has potential applications in gene therapy and research focused on treatments for genetic retinal diseases.