- Early research
- First human studies
- Clinical trials
- Approved
A simplified view. No treatment is proven yet. See the Atlas for each record and its date.
Prion disease is rare, and the registered clinical programmes are early. As of the registry records read on 31 October 2026, three trials of drugs that lower prion protein are listed: PrProfile (ION717, sponsor Ionis, Phase 1/2, active and not recruiting), PRiSM (a PrP-targeting siRNA, sponsor the Broad Institute, Phase 1, recruiting) and a Phase 1/2 trial of ALN-6457 (sponsor Regeneron, not yet recruiting). All three study safety first. No efficacy claim is made on this page, and sponsor registry text should be checked for current status.
The pages below set out the case for developers. Start with unmet need and the pipeline overview, then regulatory pathway, biomarkers and endpoints, trial feasibility and working with patient groups. Related briefs are for journalists, policy and surveillance and the partners hub.
Explore
- Biomarkers and endpointsThe outcome measures registered for three PrP-lowering prion disease trials, and what earlier drug trials used as endpoints.
- Pipeline overviewA summary of prion disease drug programmes in clinical trials and in the laboratory, with the stage and the limits stated by their own sponsors and authors.
- Regulatory pathwayWhat orphan designation means for a prion disease medicine in the EU and US, what the one EU designation for CJD is, and what remains open.
- Trial feasibility: sites, registries, cohortsWhere prion disease trials recruit, how many sites are registered, and what limits enrolment in a rare, fast disease.
- Unmet need and patient numbersPatient numbers and the treatment gap in prion disease, from CDC and NHS figures, with what the public sources do not say.
- Working with patient groupsHow prion disease patient organisations engage with drug developers: panels, trial information, travel support and data sharing, with their contact routes.