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Asns-ASO
- Development stage
- Preclinical
- Lead developer
- Ionis Pharmaceuticals
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intraperitoneal
01
Overview
Asns-ASO is an antisense oligonucleotide (ASO) designed to target and silence the expression of asparagine synthetase (ASNS). Developed by researchers at IRCCS Ospedale San Raffaele in collaboration with Ionis Pharmaceuticals, Asns-ASO is being investigated for the treatment of autosomal dominant polycystic kidney disease (ADPKD). In ADPKD, cystic epithelial cells undergo metabolic reprogramming, including increased dependence on glutamine and de novo pyrimidine synthesis, which is driven by the upregulation of ASNS. By silencing Asns, the ASO reduces total kidney volume, halts cyst expansion, and restores renal function in preclinical models. Combining Asns-ASO with the glycolytic inhibitor 2-deoxy-D-glucose (2DG) has shown synergistic therapeutic efficacy.
- Other names
- Asns antisense oligonucleotideasparagine synthetase antisense oligonucleotide
02
Targets
ASNS (Aspartate-ammonia ligase)
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