Oligonucleotide Therapeutics (ASO/siRNA)

Cat. No.

A13867

CAS No.

1173755-55-9

Eteplirsen (AVI-4658)

Synonyms: AVI 4658

Eteplirsen is an experimental drug, currently in clinical trials.

Cat. No.

A24476

CAS No.

1639264-46-2

Cemdisiran (ALN-CC5)

Synonyms: ALN-CC5

Cemdisiran is an N-acetylgalactosamine (GalNAc)-conjugated small interfering RNA (siRNA) designed to suppress hepatic production of complement component 5 (C5). It is used in research focused on complement-mediated diseases.

Cat. No.

A24793

CAS No.

2170507-65-8

Tadnersen

Synonyms: BIIB078; IONIS-C9Rx

Tadnersen is an antisense oligonucleotide (ASO) designed to selectively target C9ORF72 transcript variants 1 and 3 with the hexanucleotide expansion. This targeted mechanism of action allows for modulation of gene expression associated with neurodegenerative diseases, particularly associated with amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). Tadnersen is utilized in research applications aimed at understanding the role of C9ORF72 in these conditions and evaluating potential therapeutic strategies.

Cat. No.

A25085

CAS No.

1834560-88-1

Cosdosiran

Synonyms: QPI-1007

Cosdosiran is a chemically modified small interfering RNA (siRNA) that specifically targets and inhibits the expression of caspase 2 protein. This reagent is instrumental in researching nonarteritic anterior ischemic optic neuropathy and various optic neuropathies, including glaucoma, that lead to the degeneration of retinal ganglion cells. Cosdosiran facilitates the exploration of pathways involved in retinal cell survival and offers insights into potential therapeutic strategies for neurodegenerative eye diseases.

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.

A30320

CAS No.

890056-27-6

Custirsen

Synonyms: OGX-011

Custirsen is a targeted antisense oligonucleotide that inhibits the expression of clusterin, an antiapoptotic protein often upregulated in response to chemotherapy. By downregulating clusterin, custirsen enhances apoptosis in cancer cells, thereby overcoming treatment resistance. This compound is primarily utilized in cancer research, particularly in studies focused on improving therapeutic efficacy and understanding apoptotic mechanisms in tumor biology.

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.

A34715

CAS No.

160614-28-8

Cibrigirsen

Cibrigirsen is an antisense oligonucleotide that specifically targets and reduces the expression of the insulin-like growth factor I receptor (IGF-1R). By inhibiting IGF-1R synthesis, it plays a crucial role in modulating signaling pathways associated with cell proliferation and survival. This reagent is valuable for research applications focused on cancer biology, particularly in studies investigating the role of IGF-1R in tumor growth and resistance to therapy.

Cat. No.

A35180

CAS No.

2985646-78-2

Agazisiran

Agazisiran is a small interfering RNA (siRNA) that targets and reduces the synthesis of complement factor B, a key component in the alternative complement pathway. By modulating complement activation, Agazisiran has potential applications in research focused on autoimmune diseases, inflammatory conditions, and other disorders where complement dysregulation plays a critical role.

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.

A35229

CAS No.

3069641-77-3

Cevolisiran

Cevolisiran is a small interfering RNA (siRNA) that specifically targets and reduces the synthesis of complement component 3 (C3). This reduction in C3 levels may modulate the complement pathway, which is pivotal in immune responses and inflammation. Cevolisiran is primarily used in research applications aimed at understanding the role of the complement system in various diseases and therapeutic strategies.

Cat. No.

A35723

CAS No.

2924115-64-8

Cadapersen

Synonyms: RG6084; RO-7191863

Cadapersen is an antisense oligonucleotide that selectively induces the degradation of PD-L1 mRNA. By inhibiting PD-L1 expression, this reagent serves as a valuable tool in the investigation of immune responses and mechanisms underlying chronic hepatitis B (CHB). Its application in research enhances the understanding of immune checkpoint regulation, facilitating studies geared towards novel therapeutic strategies.

Cat. No.

A36986

CAS No.

1443994-98-6

Mongersen sodium

Synonyms: GED-0301 sodium

Mongersen sodium is a targeted SMAD7 antisense oligonucleotide that functions to restore TGF-β1 activity, resulting in the inhibition of inflammatory signaling pathways. This compound has demonstrated efficacy in attenuating experimental colitis resembling Crohn's disease in mouse models. It is a valuable tool for studying inflammatory bowel disease mechanisms and the modulation of TGF-β signaling in various research applications.

Cat. No.

A36996

CAS No.

925681-61-4

Trabedersen

Synonyms: AP 12009

Trabedersen is an antisense oligodeoxynucleotide that specifically targets and inhibits TGF-β2, a key regulator in the TGF-beta/Smad signaling pathway. This reagent is critical for research in malignant brain tumors and various solid tumors, including those affecting the skin, pancreas, and colon, which exhibit overexpression of TGF-β2. By modulating this pathway, Trabedersen offers insights into tumor biology and potential therapeutic strategies.

Cat. No.

A37001

CAS No.

1443994-46-4

Mongersen

Synonyms: GED-0301

Mongersen is a targeted SMAD7 antisense oligonucleotide that restores TGF-β1 activity, thereby inhibiting inflammatory pathways. This compound has demonstrated the ability to attenuate experimental colitis resembling Crohn's disease in murine models. Its application is particularly relevant in studying inflammation and fibrosis related to gastrointestinal disorders.

Cat. No.

A37018

CAS No.

2179222-27-4

ISTH0036

ISTH0036 is a selective antisense oligonucleotide targeting transforming growth factor beta 2 (TGF-β2). It exhibits inhibitory activity against TGF-β2 expression, making it a valuable tool for investigating the pathophysiological mechanisms of primary open-angle glaucoma (POAG) and wet age-related macular degeneration. This reagent is suitable for both in vitro and in vivo studies aimed at understanding the role of TGF-β2 in these ocular conditions.

Cat. No.

A37095

CAS No.

2897552-25-7

Surbisiran

Surbisiran is a small interfering RNA (siRNA) that functions as a β-catenin synthesis reducer. By inhibiting the synthesis of β-catenin, it exhibits antineoplastic activity, making it a valuable tool for cancer research. Surbisiran can be utilized to explore the role of β-catenin in various malignancies and investigate therapeutic strategies targeting this pathway.

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.

A37665

CAS No.

1258984-36-9

Nusinersen

Nusinersen is an antisense oligonucleotide that targets the SMN2 gene to enhance pre-messenger RNA splicing. By promoting the production of full-length survival motor neuron (SMN) protein, Nusinersen plays a crucial role in the treatment of spinal muscular atrophy (SMA). This compound is valuable in research focused on genetic disorders related to neuronal degeneration and SMN protein deficiency.

Cat. No.

A37670

CAS No.

2088232-70-4

Tofersen

Synonyms: BIIB067; ISIS-SOD1Rx; ISIS 333611

Tofersen is an antisense oligonucleotide designed to induce RNase H-mediated degradation of superoxide dismutase 1 (SOD1) mRNA, thereby decreasing SOD1 protein synthesis. This compound serves as a valuable tool in the study of amyotrophic lateral sclerosis (ALS) pathology and therapeutic interventions targeting SOD1 mutations. Researchers can utilize Tofersen to explore its potential effects on motor neuron preservation and neurodegeneration associated with ALS.

Cat. No.

A37672

CAS No.

1898254-60-8

Tofersen sodium

Synonyms: BIIB067 sodium; ISIS-SOD1Rx sodium; ISIS 333611 sodium

Tofersen sodium is an antisense oligonucleotide that specifically induces RNase H-mediated degradation of superoxide dismutase 1 (SOD1) mRNA, leading to decreased SOD1 protein synthesis. This compound is primarily utilized in research related to amyotrophic lateral sclerosis (ALS), providing insights into the pathophysiology and potential therapeutic strategies targeting SOD1 mutations.

Cat. No.

A37727

CAS No.

2589926-25-8

Ulefnersen

Synonyms: ION-363

Ulefnersen (ION363) is an antisense oligonucleotide designed to target the sixth intron of the fused-in sarcoma (FUS) transcript, facilitating the silencing of FUS in a non-allele-specific manner. This reagent effectively reduces FUS protein levels in the brain and spinal cord of relevant mouse models, thereby delaying the progression of motor neuron degeneration associated with amyotrophic lateral sclerosis (ALS-FUS). Ulefnersen is a valuable tool for research into ALS and related neurodegenerative disorders.