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Today we’re announcing the launch of PROSPECT (NCT07732608).

We’ve just published a new paper updating our estimates of penetrance and age of onset for a few of the most common genetic mutations associated with prion disease risk [Lian & Kotobelli 2026 (full text)].

Below are some of the most common questions we have been getting about PRiSM (NCT07444580), our clinical trial of PrP-siRNA in prion disease.

Today we’re proud to announce that PRiSM (NN112), our first-in-human clinical trial of PrP-siRNA, has begun enrolling pre-symptomatic participants at high genetic risk of prion disease.

Earlier this summer, FDA posted docket FDA-2026-N-4699 to the Federal Register, describing a pilot program to accelerate Investigational New Drug (IND) applications and shorten time to first-in-human clnical trials, and requesting comments from the public.

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This blog post is a call to action.

We announced today that a first-in-human clinical trial of PrP-siRNA is now enrolling symptomatic participants with prion disease.

This morning Ionis Pharmaceuticals announced that PrProfile (NCT06153966), their Phase 1/2a trial of ION717, a PrP-lowering antisense oligonucleotide for symptomatic prion disease patients, has re-opened. background If you’re just tuning in now, here’s a…

Byron Caughey lecturing at Prion2019 in Edmonton, Alberta.

Above: change in cUHDRS, the primary endpoint of Uniqure’s Huntington’s disease trial.

Last year I blogged about PRNP in gnomAD v4, which now contains DNA from 807,192 people, and with it, possibly some new insights about prion biology.

Today, an exciting announcement: we have obtained permission from the U.S.