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farabursen
- Development stage
- Phase 1
- Lead developer
- Regulus Therapeutics
- 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
- Subcutaneous
01
Overview
Farabursen (RGLS8429) is a novel, next-generation oligonucleotide drug candidate developed for the treatment of autosomal dominant polycystic kidney disease (ADPKD). It is designed to inhibit microRNA-17 (miR-17), a key regulator implicated in the pathogenesis of ADPKD, and preferentially targets the kidney. Preclinical studies have shown that administration of farabursen leads to improvements in kidney function, reduction in kidney size, and other measures of disease severity. Clinical trials have demonstrated favorable safety and pharmacokinetic profiles with no serious adverse events reported. Farabursen has shown dose-dependent biological activity as measured by increases in urinary polycystin 1 and 2 levels (PC1/PC2) and reductions in total kidney cyst volume[1][3][5][8].
- Brand names
- farabursen
- Other names
- farabursen
02
Targets
MicroRNA-17
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