Oligonucleotide Therapeutics (ASO/siRNA)

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.

A37798

CAS No.

2589926-27-0

Ulefnersen sodium

Synonyms: ION-363 sodium

Ulefnersen sodium is a targeted antisense oligonucleotide that specifically modulates the fused-in sarcoma (FUS) gene by silencing the 6th intron of its transcript. This compound effectively reduces FUS protein levels in the brain and spinal cord of disease-relevant mouse models of amyotrophic lateral sclerosis (ALS-FUS), thereby delaying the progression of motor neuron degeneration. Ulefnersen sodium is valuable for research in ALS and other conditions associated with FUS dysregulation.

Cat. No.

A37810

CAS No.

1251830-50-8

Drisapersen

Synonyms: Kyndrisa; GSK2402968A; PRO051

Drisapersen is an antisense oligonucleotide designed to promote exon 51 skipping in dystrophin pre-mRNA splicing. This mechanism facilitates the production of partially functional dystrophin, offering a potential therapeutic approach for patients with Duchenne muscular dystrophy (DMD) harboring specific amenable mutations. Its biological activity underscores its role in advancing research for targeted genetic therapies in muscle degeneration disorders.

Cat. No.

A37821

CAS No.

1422959-91-8

Golodirsen

Synonyms: SRP-4053

Golodirsen is an antisense oligonucleotide designed to target the Duchenne muscular dystrophy (DMD) gene by utilizing a phosphorodiamidate morpholino oligomer (PMO) structure. It functions by modifying the splicing of pre-mRNA to skip exon 53, effectively restoring the reading frame of the DMD gene. Golodirsen can be employed in research focused on DMD, helping to investigate potential therapies aimed at restoring the expression of the anti-myostatin protein and improving muscle function.

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.

A38206

CAS No.

2576358-63-7

Mulnitorsen

Synonyms: ANDES-1537

Mulnitorsen is an antisense non-coding mitochondrial RNA (ASncmtRNA) synthesis reducer targeting mitochondrial RNA processing. This compound demonstrates significant antitumor activity by modulating mitochondrial gene expression, contributing to cancer cell apoptosis and inhibition of tumor growth. Mulnitorsen is applicable in cancer research, particularly in studies focusing on mitochondrial function and the development of novel therapeutic strategies for malignancies.

Cat. No.

A42458

CAS No.

2591587-57-2

Rugonersen

Synonyms: RG6091; RO7248824

Rugonersen is a locked-nucleic acid (LNA)-modified antisense oligonucleotide targeting the ubiquitin-protein ligase E3A (UBE3A). This compound effectively reduces the silencing of UBE3A, which is implicated in Angelman syndrome, a severe neurodevelopmental disorder. Rugonersen is utilized in research aiming to restore UBE3A expression, providing insights into potential therapeutic strategies for this condition.

Cat. No.

A42469

CAS No.

2591588-58-6

Rugonersen sodium

Synonyms: RG6091 sodium; RO7248824 sodium

Rugonersen sodium is a locked-nucleic acid (LNA)-modified antisense oligonucleotide (ASO) targeting ubiquitin-protein ligase E3A (UBE3A). This compound effectively reduces the silencing of UBE3A, making it a valuable tool for studying Angelman syndrome, a severe neurodevelopmental disorder resulting from the loss of functional UBE3A in neurons. Rugonersen sodium facilitates research into potential therapeutic strategies for this condition, contributing to the understanding of UBE3A function and regulation in neuronal development.

Cat. No.

A42513

CAS No.

2834724-31-9

Obudanersen

Synonyms: ION-582

Obudanersen is an antisense oligonucleotide designed to specifically target the ubiquitin protein ligase E3A-antisense transcript (UBE3A-ATS). This compound plays a critical role in the investigation of Angelman syndrome by modulating UBE3A expression. Its ability to alter gene expression makes it a valuable tool for studying the underlying mechanisms of this neurodevelopmental disorder and for assessing potential therapeutic strategies.

Cat. No.

A42523

CAS No.

2919324-01-7

Apazunersen

Synonyms: GTX-102

Apazunersen is an antisense oligonucleotide (ASO) designed to target and inhibit the expression of the UBE3A antisense transcript (UBE3A-AS). This mechanism prevents the silencing of the paternally inherited allele of the UBE3A gene, facilitating the reactivation of the deficient protein. Apazunersen holds potential for research applications related to Angelman syndrome, offering insights into genetic regulation and therapeutic strategies for this neurodevelopmental disorder.

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.

A46298

CAS No.

2808361-18-2

Lepodisiran

Synonyms: LY3819469

Lepodisiran is a small interfering RNA (siRNA) that specifically targets lipoprotein(a) to diminish its synthesis. By reducing lipoprotein(a) levels, lepodisiran may have therapeutic potential in addressing cardiovascular diseases associated with elevated lipoprotein(a). Its research applications include the study of lipid metabolism and the development of innovative treatments for atherosclerosis 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.

A46303

CAS No.

1499251-18-1

Fitusiran

Synonyms: ALN-AT3SC; SAR439774

Fitusiran is a small interfering RNA (siRNA) reagent that specifically targets the SERPINC1 gene in hepatocytes, effectively inhibiting the synthesis of antithrombin. By doing so, Fitusiran promotes enhanced thrombin activity and improves thrombin generation, which may restore normal hemostatic function. This compound is primarily utilized in research focused on hemophilia and related coagulopathy studies.

Cat. No.

A46304

CAS No.

2247026-22-6

Nedosiran sodium

Synonyms: DCR-PHXC sodium

Nedosiran sodium is an RNA interference agent that targets lactate dehydrogenase (LDH). This GalNAc-dsRNA conjugate demonstrates significant potential as a therapeutic option for primary hyperoxaluria (PH), particularly in patients with end-stage renal disease (ESRD). Its mechanism of action offers a novel approach to reducing LDH levels, thereby addressing metabolic imbalances associated with these conditions.

Cat. No.

A46305

CAS No.

1867157-35-4

Vutrisiran

Synonyms: ALN-TTRsc02

Vutrisiran is an investigational small interfering ribonucleic acid (siRNA) agent targeting transthyretin (TTR). This liver-directed compound is designed to reduce the production of TTR, making it a valuable tool for research into TTR-mediated amyloidosis. Vutrisiran facilitates studies aimed at understanding the molecular mechanisms of amyloidogenesis and potential therapeutic strategies for related diseases.

Cat. No.

A46306

CAS No.

2175009-09-1

Fazirsiran sodium

Synonyms: ARO-AAT sodium

Fazirsiran sodium is a second-generation RNA interference (RNAi) agent that targets Alpha-1 antitrypsin (AAT) mRNA. It comprises a cholesterol-conjugated RNAi trigger (chol-RNAi) designed for specific degradation of AAT mRNA, along with an endosomal escape-enhancing peptide derived from melittin, conjugated to N-acetylgalactosamine (NAG). This formulation facilitates effective delivery in hepatocytes. Fazirsiran sodium is primarily utilized in research related to Alpha-1 Antitrypsin Deficiency (AATD), a condition resulting from mutations in the SERPINA1 gene, which leads to liver disease.

Cat. No.

A46307

CAS No.

1848224-71-4

Tivanisiran

Synonyms: SYL1001

Tivanisiran is a small interfering RNA (siRNA) targeting the transient receptor potential vanilloid 1 (TRPV1) mRNA. It is primarily used in research focused on dry eye disease, facilitating the study of TRPV1's role in ocular inflammatory responses. By silencing TRPV1 expression, Tivanisiran enables the investigation of therapeutic strategies aimed at alleviating symptoms associated with dry eye disorders.

Cat. No.

A46308

CAS No.

2646703-64-0

Zerlasiran

Synonyms: SLN360

Zerlasiran is an apolipoprotein A (ApoA) synthesis reducer that targets RNA to inhibit ApoA production. This compound demonstrates significant biological activity in lowering ApoA levels, making it a valuable tool in research focused on lipid metabolism and cardiovascular diseases. Zerlasiran is applicable in studies investigating the modulation of lipid profiles and the effects of reduced ApoA synthesis on atherogenesis and related conditions.

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.