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GCCR ASO
- 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
GCCR ASO is an antisense oligonucleotide (ASO) designed to selectively reduce the expression of the glucocorticoid receptor (GCCR, also known as NR3C1) in the liver and white adipose tissue (WAT). Developed primarily by Ionis Pharmaceuticals, this therapeutic approach utilizes antisense technology to degrade GCCR mRNA, thereby inhibiting hepatic gluconeogenesis and improving insulin sensitivity and lipid profiles. Unlike systemic glucocorticoid antagonists, GCCR ASOs are designed to be tissue-selective, aiming to treat metabolic disorders such as type 2 diabetes and metabolic syndrome without triggering the activation of the hypothalamic-pituitary-adrenal (HPA) axis or causing systemic adrenal insufficiency. Clinical development reached Phase 2 with the lead candidate IONIS-GCCRRx (ISIS 426115).
- Other names
- Glucocorticoid receptor antisense oligonucleotideGR ASO
02
Targets
GR (Glucocorticoid receptor)
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