Drug intelligence / Profile preview

antisense oligonucleotide targeting sEV biogenesis regulator

Development stage
Preclinical
Lead developer
Institute of Science Tokyo
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
Intravenous
01

Overview

The antisense oligonucleotide targeting sEV biogenesis regulator is a preclinical antisense oligonucleotide (ASO) candidate being developed by the Institute of Science Tokyo and Gunma University [1091]. It is designed to knock down a key, undisclosed regulator of small extracellular vesicle (sEV) biogenesis and secretion in renal tubular epithelial cells (TECs) [1091]. In diabetic kidney disease (DKD), injured kidneys release sEVs that promote vascular calcification, interstitial fibrosis, and inflammation [1091]. By inhibiting sEV biogenesis, this ASO aims to disrupt pathogenic intercellular communication within the kidney [1091]. Preclinical studies in db/db mouse models of DKD have shown that weekly intravenous administration of the candidate ASO (20 mg/kg) attenuates elevations in serum creatinine and urea levels, suggesting therapeutic potential for diabetic kidney disease [1091].

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