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JUD-001
- Development stage
- Preclinical
- Lead developer
- Judo Bio
- Modality
- Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Subcutaneous
01
Overview
JUD-001 is a preclinical-stage, kidney-targeted small interfering RNA (siRNA) therapeutic being developed by Judo Bio for the treatment of gout. It is designed using Judo Bio's proprietary STRIKE™ (Selectively Targeting RNA Into KidnEy) platform, which utilizes ligand-siRNA conjugates that bind to megalin (LRP2) receptors highly expressed on proximal tubular epithelial cells (PTECs) in the kidney. This receptor-mediated endocytosis enables selective delivery and intracellular uptake of the siRNA. Once inside the PTECs, JUD-001 targets and silences the mRNA of the renal urate transporter URAT1 (encoded by the *SLC22A12* gene), thereby reducing transporter expression, inhibiting renal urate reabsorption, and promoting urinary excretion of uric acid to lower systemic urate levels in patients with hyperuricemia and gout.
02
Targets
URAT1 (Uric Acid Transporter 1)
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