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IONIS GHR-LRx + somatostatin receptor ligand

Development stage
Unknown
Lead developer
Ionis Pharmaceuticals
Modality
Peptides, 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

IONIS GHR-LRx + somatostatin receptor ligand is a combination therapy being investigated for the treatment of acromegaly in patients already receiving long-acting somatostatin receptor ligands. IONIS GHR-LRx (also known as Cimdelirsen, ISIS 766720) is a ligand-conjugated antisense oligonucleotide designed to reduce growth hormone receptor (GHR) synthesis in the liver, thereby inhibiting excessive growth hormone activity and reducing circulating insulin-like growth factor 1 (IGF-1) levels. The somatostatin receptor ligand component (such as octreotide or lanreotide) is a peptide analog that activates somatostatin receptors (especially SSTR2 and SSTR5), suppressing growth hormone secretion from the pituitary gland. The rationale for combining both is to enhance disease control in inadequately managed acromegaly patients by targeting growth hormone signaling at both the pituitary and peripheral (hepatic) levels[3][4][7].

Other names
IONIS-GHR-LRxCimdelirsenISIS 766720ISIS766720ISIS-766720somatostatin receptor ligandSRLlong-acting somatostatin receptor ligand
02

Targets

GHR (Growth hormone receptor)SSTR5 (Somatostatin receptor 5)SSTR2 (Somatostatin receptor type 2)

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