Cemdisiran — Investigational Agent
Other · Phase 3 · Alnylam / Regeneron
Mechanism of Action
Subcutaneously administered small interfering RNA (siRNA) conjugated to GalNAc for hepatocyte delivery. Cemdisiran silences C5 messenger RNA in the liver (the principal site of complement C5 synthesis), lowering circulating C5 protein and thereby reducing terminal complement activity and the membrane attack complex responsible for intravascular hemolysis in PNH. It is being developed both as monotherapy and in combination with the anti-C5 monoclonal antibody pozelimab to achieve deeper, more durable C5 suppression.
Investigational Indications
- Paroxysmal nocturnal hemoglobinuria (PNH) — as monotherapy and in combination with pozelimab