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cimdelirsen
- Development stage
- Phase 2
- Lead developer
- Ionis Pharmaceuticals
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Subcutaneous
01
Overview
Cimdelirsen (IONIS-GHR-LRx; ISIS 766720) is a ligand-conjugated, hepatic-targeted antisense oligonucleotide developed to reduce the synthesis of growth hormone receptor (GHR). By inhibiting GHR production in the liver, it decreases the effects of excess growth hormone and lowers circulating insulin-like growth factor-1 (IGF-1) levels. Cimdelirsen is being investigated primarily for the treatment of acromegaly, particularly in patients with inadequate response to long-acting somatostatin receptor ligands. The drug uses a GalNAc conjugation strategy for targeted delivery to hepatocytes and acts through RNase H1-mediated degradation of GHR mRNA[1][3][4].
- Other names
- cimdelirsen sodium
02
Targets
GHR (Growth hormone receptor)
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