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Oligonucleotide Therapeutics lists antisense oligonucleotides and small interfering RNAs that reduce or modify gene expression. Each antisense oligonucleotide (ASO) binds complementary RNA and, depending on its chemistry, triggers ribonuclease H (RNase H) cleavage, alters splicing or blocks translation. Antisense examples include eteplirsen, a phosphorodiamidate morpholino oligomer, and danvatirsen, which is directed at signal transducer and activator of transcription 3 (STAT3). Each small interfering RNA (siRNA) is a short double-stranded RNA that guides the RNA-induced silencing complex (RISC) to cleave a matching messenger RNA (mRNA). Inclisiran, for example, is an siRNA directed at proprotein convertase subtilisin/kexin type 9 (PCSK9). Phosphoramidite building blocks for oligonucleotide synthesis are listed separately under Phosphoramidites.
Lufepirsen is an unmodified antisense oligonucleotide that specifically targets Connexin43 (Cx43). This protein is critical for cellular communication and plays a significant role in wound healing processes within the eye. Lufepirsen has potential applications in research focused on ocular health and regenerative medicine.
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Synonyms: DS-5141b
Renadirsen is an antisense oligonucleotide designed to promote exon 45 skipping of the dystrophin gene. This mechanism facilitates the production of a functional dystrophin protein, which is crucial for muscle function. Renadirsen is primarily utilized in research related to Duchenne muscular dystrophy (DMD), aiming to advance therapeutic strategies targeting dystrophinopathies.
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Rimigorsen is an antisense oligonucleotide designed to induce skipping of exon 44 in the pre-mRNA of dystrophin, targeting the underlying genetic mutation associated with Duchenne muscular dystrophy (DMD). This compound effectively promotes the production of partially functional dystrophin, which is critical for muscle integrity and function. Rimigorsen is a valuable tool for research in the development of therapies aimed at treating DMD and other related neuromuscular disorders.
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Tacadirsen is an oligonucleotide designed to promote the synthesis of functional dystrophin, a crucial protein for muscle integrity. It is primarily utilized in research related to Duchenne muscular dystrophy (DMD), a genetic disorder characterized by progressive muscle degeneration. This compound serves as a valuable tool for understanding dystrophin deficiencies and exploring potential therapeutic strategies for DMD.
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Nivudirsen is an antisense oligonucleotide specifically designed to target dystrophin mRNA, promoting the production of functional dystrophin protein. This compound plays a crucial role in gene therapy research, particularly in the context of Duchenne muscular dystrophy (DMD), where restoring dystrophin expression is essential for muscle function improvement. Its application extends toward investigating dystrophin-related pathology and developing innovative therapeutic strategies.
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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.
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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.
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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.
Synonyms: RGLS8429 sodium; RG1015 sodium
Farabursen sodium is a selective inhibitor of the microRNA-17 family. By effectively inhibiting the functionality of miR-17, it enhances the expression of target genes such as PKD1 and PKD2, leading to increased levels of polycystin-1 and -2 (PC1/2). This compound demonstrates significant potential in attenuating the growth of renal cysts and reducing kidney weight-to-body weight ratios, making it valuable for research into autosomal dominant polycystic kidney disease.
Synonyms: MRG-106 sodium
Cobomarsen sodium is a targeted miR-155 inhibitor that modulates various signaling pathways, including JAK/STAT, MAPK/ERK, and PI3K/AKT, contributing to the regulation of cell survival. This oligonucleotide is primarily employed in research focused on B-cell lymphoma, enabling insights into the molecular mechanisms of this malignancy and potential therapeutic strategies.
Synonyms: MRG-106
Cobomarsen is an oligonucleotide inhibitor targeting miR-155. This compound modulates various signaling pathways associated with cell survival, including the JAK/STAT, MAPK/ERK, and PI3K/AKT pathways. Cobomarsen is primarily utilized in research focused on B-cell lymphoma, providing insights into miRNA-mediated gene regulation and potential therapeutic approaches.
Synonyms: MRG-201
Remlarsen (MRG-201) is a miR-29b mimic that functions as an agonist of the miR-29b pathway. It is primarily involved in inhibiting the formation of fibrotic scars and addressing cutaneous fibrosis. This reagent is valuable for research applications focused on fibrosis-related conditions and the modulation of miRNA pathways in cellular processes.
Dimovarsen is an oligonucleotide designed as an inhibitor of micro RNA-132. This compound plays a crucial role in the modulation of gene expression related to cardiac function and has potential applications in the study of heart failure. By inhibiting micro RNA-132, Dimovarsen may aid in elucidating the underlying mechanisms of heart disease and facilitate the exploration of novel therapeutic strategies.
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Synonyms: SYL040012
Bamosiran is a small interfering RNA that specifically targets the β-adrenergic receptor 2. This compound is recognized for its ability to significantly reduce intraocular pressure, making it valuable in the study of ocular conditions. Its role as an adrenergic receptor inhibitor aids in investigating therapeutic strategies for glaucoma and other eye disorders.
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Synonyms: ISIS-757456
Evazarsen is an angiotensinogen synthesis inhibitor that effectively reduces the production of angiotensinogen, a precursor to angiotensin II, a peptide that plays a crucial role in blood pressure regulation. This compound exhibits antihypertensive properties, making it valuable for research focused on cardiovascular diseases and hypertension management. Its ability to modulate the renin-angiotensin system supports investigations into therapeutic strategies for hypertension and related disorders.
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Synonyms: AD-85481; ALN-AGT
Zilebesiran is a small interfering RNA (siRNA) that selectively inhibits hepatic angiotensinogen expression via RNA interference. This reagent plays a crucial role in the investigation of mild to moderate hypertension, as angiotensinogen is the primary precursor of angiotensin peptides and a significant regulator of systemic blood pressure. Researchers can utilize Zilebesiran to explore therapeutic strategies targeting the renin-angiotensin system in cardiovascular studies.
Synonyms: ISIS-757456 sodium
Evazarsen sodium is an angiotensinogen synthesis inhibitor that exhibits antihypertensive properties. By targeting the synthesis of angiotensinogen, it plays a significant role in regulating blood pressure and can be utilized in research related to cardiovascular diseases and hypertension management. This compound is valuable for studies focusing on the molecular mechanisms of blood pressure regulation and the development of potential therapeutic interventions.
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Synonyms: AD-85481 sodium; ALN-AGT sodium
Zilebesiran sodium is a small interfering RNA (siRNA) that effectively reduces hepatic angiotensinogen levels through RNA interference. By targeting angiotensinogen, a primary precursor of angiotensin peptides, it plays a significant role in regulating blood pressure. This reagent is valuable for research applications related to mild to moderate hypertension and cardiovascular disease mechanisms.
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ISIS 449884 is a 2'-O-methoxyethyl antisense oligonucleotide that targets the glucagon receptor gene (GCGR). It effectively reduces hepatic glucose output and lowers blood glucose levels, making it a valuable tool for studying type 2 diabetes mellitus (T2DM). This compound is instrumental for researchers investigating the molecular mechanisms underlying glucose metabolism and diabetes pathophysiology.
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Lixadesiran is an siRNA designed to inhibit cyclooxygenase-2 (COX-2) by targeting its mRNA. This compound is part of the STP705 formulation, which includes two siRNA oligonucleotides, with Pixofisiran targeting TGF-β1. Lixadesiran's mechanism of action contributes to the modulation of inflammatory pathways, making it relevant for research in cancer and fibrosis studies.
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Synonyms: ION373 sodium
Zilganersen sodium is a gapmer antisense oligonucleotide that specifically inhibits glial fibrillary acidic protein (GFAP) synthesis. By targeting the GFAP gene, Zilganersen sodium effectively reduces the accumulation of GFAP associated with pathogenic variants, making it a valuable tool for studying Alexander disease. This compound facilitates research into the mechanisms of glial cell dysfunction and therapeutic approaches in related neurodegenerative disorders.
Synonyms: ION373
Zilganersen is a gapmer antisense oligonucleotide that specifically targets glial fibrillary acidic protein (GFAP). By inhibiting the synthesis of GFAP, Zilganersen reduces excess levels produced by disease-associated variants in the GFAP gene. This reagent is valuable for research focused on the pathophysiology of Alexander disease and the roles of glial cells in neurological disorders.
Synonyms: ION-935918 sodium; ION251 sodium
Frenlosirsen sodium is an antisense oligonucleotide that specifically targets IRF4, a critical regulator in immune responses. This compound has demonstrated potential in the study of relapsed or refractory multiple myeloma (RRMM), facilitating research into therapeutic interventions for this challenging malignancy. Its mechanisms may provide insight into the modulation of immune pathways associated with multiple myeloma progression and treatment resistance.
Synonyms: ION-935918; ION251
Frenlosirsen is an antisense oligonucleotide that specifically targets interferon regulatory factor 4 (IRF4). It is utilized in research focused on relapsed or refractory multiple myeloma (RRMM), enabling the examination of therapeutic strategies aimed at modulating this transcription factor. This reagent serves as a valuable tool for understanding the role of IRF4 in tumorigenesis and resistance mechanisms in hematological malignancies.
Synonyms: ISIS-721744; IONIS-PKK-LRX
Donidalorsen sodium is an antisense oligonucleotide that selectively targets prekallikrein (PKK). By binding to and promoting the degradation of PKK mRNA in the liver, Donidalorsen sodium effectively inhibits kallikrein activity and diminishes Bradykinin production. This reagent is particularly valuable for research applications related to hereditary angioedema.
