Multiple‐Dose Results from an Ongoing Phase 1 Study of Mivelsiran, an Investigational RNA Interference Therapeutic Targeting Amyloid‐Beta Precursor Protein for Alzheimer’s Disease
In this first report of multiple-dose clinical data for a CNS-targeting RNAi therapeutic, single and multiple doses of mivelsiran were generally well tolerated and continued to demonstrate robust, durable, dose-dependent CSF sAPPβ reductions. Further sAPPβ lowering was observed after a second dose of mivelsiran 50mg. These results support further evaluation of mivelsiran in patients with Alzheimer's disease or cerebral amyloid angiopathy.
Authors
- D. Blackburn (ORCID: https://orcid.org/0000-0001-8886-1283)
- Eric McDade (ORCID: https://orcid.org/0000-0002-6764-3866)
- Alexandre Sostelly (ORCID: https://orcid.org/0000-0002-7300-979X)
- Jared R. Brosch (ORCID: https://orcid.org/0000-0002-6578-0584)
- Julia Shirvan
- Everard G.B. Vijverberg (ORCID: https://orcid.org/0000-0003-3879-884X)
- Robert Deering
- Simon Ducharme (ORCID: https://orcid.org/0000-0002-7309-1113)
- Lynn Farrugia
- Sharon Cohen
- Catherine J. Mummery
- Sandeep Chaudhari
Institutions
- Montreal Neurological Institute and Hospital (CA)
- Alnylam Pharmaceuticals (United States) (US)
- Washington University in St. Louis (US)
- Amsterdam University Medical Centers (NL)
- Indiana University School of Medicine
- University College London (GB)
- McGill University (CA)
- Indiana University – Purdue University Indianapolis (US)
- Vrije Universiteit Amsterdam (NL)
- University of Sheffield (GB)
Publication Details
- Journal
- Alzheimer s & Dementia
- Published
- 2025-12-01
- DOI
- https://doi.org/10.1002/alz70859_097989
- Primary Topic
- RNA Interference and Gene Delivery
- Type
- article
- Field-Weighted Citation Impact
- 0.00