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Oligonucleotide Therapeutics (ASO/siRNA)

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.

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  • Cimdelirsen sodium
    Cat. No.
    A74928
    CAS No.
    2131025-83-5

    Cimdelirsen sodium

    Synonyms: ISIS 766720 sodium; IONIS-GHR-LRx sodium

    Cimdelirsen sodium is an investigative antisense oligonucleotide designed to inhibit growth hormone receptor (GHR) synthesis through a ligand-conjugated, hepatic-targeted mechanism. This compound effectively reduces the detrimental effects associated with growth hormone (GH) hypersecretion and lowers circulating insulin-like growth factor-1 (IGF-1) levels in patients with acromegaly. Its primary applications include studying the modulation of GH signaling and exploring therapeutic strategies in disorders related to GH excess.

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