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pivicasiran
- Development stage
- Phase 2
- Lead developer
- Regeneron 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
**Pivicasiran** is an investigational small interfering RNA (siRNA) therapeutic designed to reduce the synthesis of adiponutrin (also known as patatin-like phospholipase domain-containing protein 3 or PNPLA3), a key enzyme implicated in lipid metabolism dysregulation in liver diseases. It functions by inhibiting PNPLA3 expression via RNA interference, aiming to address pathological lipid accumulation. Developed initially by Regeneron Pharmaceuticals, it is in early clinical testing for liver conditions associated with metabolic dysfunction.[1][3]
- Other names
- ALN-PNP
02
Targets
PNPLA3 (Patatin-like phospholipase domain-containing protein 3)
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