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ALN-6457 in symptomatic prion disease
2026-526727-21-00Phase 2Authorised
- Sponsor
- Regeneron Pharmaceuticals
- Start
- Oct 2026
- 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
- Countries
- 6
01
Overview
Phase 2, open-label, single-arm trial of ALN-6457 in Prion Disease (PD), Creutzfeldt-Jakob disease (CJD) and Genetic Prion Disease, sponsored by Regeneron Pharmaceuticals. Primary endpoints: Occurrence of treatment-emergent adverse events, Severity of treatment-emergent adverse events and 1 more. Planned start Oct 2026; status: authorised.
02
Drugs
ALN-6457
03
Conditions
Prion Disease (PD)Creutzfeldt-Jakob disease (CJD)Genetic Prion Disease
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