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onvuzosiran
- Development stage
- Phase 3
- 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
Onvuzosiran (ADX-324) is an investigational small interfering RNA (siRNA) therapeutic developed by ADARx Pharmaceuticals for the prophylactic treatment of hereditary angioedema (HAE). It is designed to target and inhibit the expression of prekallikrein (PKK) mRNA in the liver, thereby reducing the production of plasma prekallikrein. By lowering these levels, the drug prevents the subsequent activation of plasma kallikrein and the excessive release of bradykinin, which is the primary mediator of the painful and life-threatening swelling attacks characteristic of HAE. Onvuzosiran is engineered for high potency and durability, with clinical data supporting a potential twice-yearly (semi-annual) subcutaneous dosing regimen to maintain high attack-free rates.
- Brand names
- Onvuzosiran
- Other names
- onvuzosiran
02
Targets
PKK (Plasma prekallikrein (KLKB1))
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