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ALN-XDH
- Development stage
- Discontinued
- Lead developer
- Alnylam Pharmaceuticals
- 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
ALN-XDH is an investigational RNA interference (RNAi) therapeutic developed for the treatment of gout. It is designed to be administered subcutaneously and specifically targets xanthine dehydrogenase (XDH), a key enzyme involved in uric acid production. By silencing XDH expression, ALN-XDH aims to lower urate levels in patients, potentially reducing gout flares and improving disease management. The drug utilizes Alnylam’s Enhanced Stabilization Chemistry Plus (ESC+) GalNAc-conjugate technology, which enables selective delivery and infrequent dosing. Development was led by Alnylam Pharmaceuticals, with clinical trials reaching up to Phase 2 before being discontinued[1][2][3][4][5].
02
Targets
XO (Xanthine Oxidoreductase)
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