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agazisiran
- Development stage
- Phase 2
- Lead developer
- ADARx 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
Agazisiran (ADX-038) is a GalNAc-conjugated small interfering RNA (siRNA) therapeutic designed to target and suppress the synthesis of complement factor B (CFB). Developed by ADARx Pharmaceuticals, agazisiran is designed to inhibit the alternative pathway of the complement system, offering a potential treatment for complement-mediated diseases. By selectively silencing CFB, it aims to provide deep and prolonged alternative pathway inhibition with a convenient dosing schedule (such as every 3 or 6 months) while sparing the classical complement pathway to minimize infection risks. It is currently in Phase 2 clinical development for complement-mediated kidney diseases (including IgA nephropathy, complement 3 glomerulopathy, and immune complex membranoproliferative glomerulonephritis), paroxysmal nocturnal hemoglobinuria (PNH), and geographic atrophy secondary to age-related macular degeneration.
- Other names
- agazisiran sodiumsodium agazisiran
02
Targets
CFB (Complement Factor B)
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