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ALXN2030
- Development stage
- Phase 2
- Lead developer
- Alexion 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
ALXN2030 is a small interfering RNA (siRNA) drug candidate developed by Alexion Pharmaceuticals (now part of AstraZeneca Rare Disease) that targets the complement C3 protein. Its mechanism of action involves inhibiting the expression of complement C3 via RNA interference, thereby modulating the activity of the complement system. The drug is being investigated primarily for its potential to treat antibody-mediated rejection (AMR) in kidney transplant recipients. As of April 2025, it is in Phase 2 clinical trials for this indication[2][3][5]. The therapy is administered subcutaneously and aims to reduce immune-mediated damage to transplanted kidneys by suppressing complement activation[4].
02
Targets
C3
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