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ION717

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PRNP-lowering antisense medicine in an active Phase 1/2a study for symptomatic prion disease.

Modality
intrathecal antisense RNA-targeted therapeutic against PRNP messenger RNA
Availability (US)
Phase 1/2a PrProfile study Active, not recruiting
Biomarkers
treatment-emergent adverse events; plasma and cerebrospinal-fluid pharmacokinetics; cerebrospinal-fluid prion protein
Hallmark targeted
loss of proteostasis, protein misfolding and aggregation
Kind
investigational oligonucleotide
Clinical trials
NCT06153966 — Phase 1/Phase 2, Active, not recruiting on September 18, 2026 registry update