News•Apr 22, 2026
Divalent siRNA Clinical Trial Is Now Recruiting
A first‑in‑human trial of a divalent PrP‑siRNA (2439‑s4) is now enrolling 15 symptomatic prion disease participants. The FDA‑cleared IND permits a single‑ascending‑dose study, testing 50 mg, 100 mg and 200 mg levels to assess safety and target engagement. The trial includes an optional observational arm as a comparator, given the absence of a placebo group. Results will inform dosing frequency and biomarker strategies for future larger studies.
By CureFFI.org
News•Mar 17, 2026
ION717 Trial Re-Opens with 3rd Dosing Regimen
Ionis Pharmaceuticals announced that its Phase 1/2a PrProfile trial of ION717, the first PrP‑lowering antisense oligonucleotide for symptomatic prion disease, has reopened with a third dosing regimen. The study, which enrolled 56 patients in 2024, will now recruit at three...
By CureFFI.org