Prion diseases are rare, fast and so far untreatable. Past trials of repurposed drugs found no survival benefit, and the first drugs designed to lower prion protein are now in early human trials: an antisense drug from Ionis, in which 56 symptomatic patients enrolled in 2024, and a divalent siRNA in the PRiSM trial. A Regeneron siRNA is also listed, and gene editing and small molecules remain preclinical. Results from the Ionis trial are not expected before 2027, so efficacy is unknown. The Atlas records each program with its sources and stage.
Pages here are written for pharma, insurers, AI companies, funders, policy and journalists. They summarise what is known and what is not, and they point to the Atlas and to research pages for detail. Nothing here is advice for any individual or an endorsement of a program.
Explore
- AI companies and developersFor AI companies and developers working on prion disease: what data and problems exist, and where to find the responsible use and access pages.
- Funders and foundationsFor funders and foundations: who currently supports prion disease trials and preclinical work, where gaps remain and how to give.
- InsurersA brief on genetic information rules in insurance in the US, UK, Canada and Australia. We provide no risk analysis or underwriting tools.
- For journalistsA short brief for journalists on prion disease: the main facts, the terms that get confused, and where to check figures.
- Pharma and biotechEntry point for pharma and biotech: what prion disease trials exist, and where to find unmet need, pipeline, regulatory and feasibility briefs.
- Policy and surveillanceWhy prion disease surveillance and policy are an industry concern, with a guide to surveillance, data protection and evidence for regulators.