Cemdisiran cemdisiran PHASE3
Drug Profile
ModalitysiRNA
RouteSC
Therapy AreaRare Disease
Formulations[]
Companies
REGN (ORIGINATOR)100%
Mechanism: C5 siRNA (liver-targeted gene silencing)
Expert: GalNAc-conjugated small interfering RNA targeting hepatic C5 mRNA. Silences C5 production in hepatocytes via RISC pathway. GalNAc moiety enables liver-targeted uptake via ASGPR. SC Q4-8W dosing. Combined with pozelimab (anti-C5 mAb) for complete and durable C5 inhibition.
Everyday: Uses a tiny piece of genetic code (siRNA) to silence the gene that makes complement C5 protein in the liver. By shutting off C5 production at the source, it provides long-lasting complement inhibition with infrequent dosing — like turning off the factory instead of blocking each product one by one.
Targets: ["C5"]
Notes
C5-targeting siRNA (GalNAc-conjugated). Used in combination with pozelimab (anti-C5 mAb, marketed as Veopoz). The combo provides dual C5 inhibition: siRNA reduces C5 production while mAb neutralizes circulating C5. Together they achieve near-complete C5 suppression.
Data from Supabase · Updated 2026-03-24