Single ascending dose results from an ongoing Phase 1 study of mivelsiran (ALN‐APP), the first investigational RNA interference therapeutic targeting amyloid precursor protein for Alzheimer’s disease
Sharon Cohen, Simon Ducharme, Jared R Brosch, Everard GB Vijverberg, Alexandre Sostelly, Sasikiran Goteti, Lynn Farrugia, Andreja Avbersek, Cristin Kaspar, Catherine J Mummery · 2025
Read Single ascending dose results from an ongoing Phase 1 study of mivelsiran (ALN‐APP), the first investigational RNA interference therapeutic targeting amyloid precursor protein for Alzheimer’s disease by Sharon Cohen, Simon Ducharme, Jared R Brosch, Everard GB Vijverberg, Alexandre Sostelly, Sasikiran Goteti, Lynn Farrugia, Andreja Avbersek, Cristin Kaspar, Catherine J Mummery (2025) in the Tower of Babel, a free online library of public-domain works preserved in clean, searchable text. No sign-up. The full text is served as a plain-text file at a predictable URL, where text is present: https://antarctic-labs.com/tower-of-babel/pmc-11712346.txt.
Description
Sharon Cohen and co-authors report early results for an RNA drug aimed at Alzheimer's disease in a 2025 paper. The drug, mivelsiran, is injected into the spinal fluid and silences the gene for the precursor protein that yields amyloid beta, a driver of the disease. The trial tested single rising doses in people with early onset illness.
Twenty patients got one of three doses or placebo and were watched for six months. All side effects reported were mild or moderate. The report covers safety plus drug action and disease markers. The study marks the first test of this gene silencing approach against the precursor protein in patients.
Catalog record
| CREATOR | Sharon Cohen, Simon Ducharme, Jared R Brosch, Everard GB Vijverberg, Alexandre Sostelly, Sasikiran Goteti, Lynn Farrugia, Andreja Avbersek, Cristin Kaspar, Catherine J Mummery |
|---|---|
| YEAR | 2025 |
| COLLECTION | PAPERS |
| FORMAT | TXT |
| FILE SIZE | 3 KB |
| VERSION | 1.0 |
| LICENSE | CC BY 4.0 |
| RIGHTS | CC BY |
| SOURCE | Europe PMC |