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Mivelsiran

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APP-targeting RNAi therapeutic in Phase 1 and Phase 2 studies across amyloid-related neurological diseases.

Modality
intrathecal RNA interference therapeutic targeting APP mRNA
Availability (US)
Phase 1 study Recruiting; one Phase 2 study Active, not recruiting and another Phase 2 study Recruiting
Biomarkers
CSF soluble APP alpha and beta; amyloid beta 40 and 42; brain amyloid burden; MRI cerebral microbleeds; treatment-emergent adverse events
Hallmark targeted
loss of proteostasis, extracellular amyloid accumulation, neurodegeneration
Kind
investigational RNAi therapeutic
Clinical trials
NCT05231785 — Phase 1, Recruiting on September 14, 2026 update; NCT06393712 — Phase 2, Active, not recruiting on September 18, 2026 update; NCT07636811 — Phase 2, Recruiting on September 4, 2026 update