Cimdelirsen

**Semantic type:** Nucleic Acid, Nucleoside, or Nucleotide|Pharmacologic Substance

**Definition:** An antisense oligonucleotide targeting the growth hormone receptor (GHR; somatotropin receptor) mRNA, with potential somatotropin-inhibiting activity. Upon subcutaneous administration, cimdelirsen hybridizes with GHR mRNA, which blocks translation of the GHR protein. Reduction of GHR levels in turn decreases the circulating level of insulin-like growth factor-1 (IGF-1). IGF-1 is a hormone that plays an important role in childhood growth and has anabolic effects in adults. Excess production of IGF-1 results in various disease states including acromegaly.

**Synonyms:** - CIMDELIRSEN - GHR Antisense Oligonucleotide IONIS-GHR-LRx - Growth Hormone Receptor Antisense Oligonucleotide IONIS-GHR-LRx - IONIS GHR LRX - IONIS-GHR-LRx - ISIS-766720 - LICA Drug IONIS-GHR-LRx - Ligand-conjugated Antisense Drug IONIS-GHR-LRx