#  Clinical trial of a prion disease drug candidate begins enrolling participants 

 





It’s the first time the potential treatment, a small interfering RNA targeting the prion protein, is being tested in humans.



 

May 04, 2026

 

 

A new drug candidate designed to slow the progression of prion disease is entering a phase 1 [clinical trial](https://clinicaltrials.gov/study/NCT07444580), which will evaluate the potential medicine for safety and tolerability.

Prion diseases are a class of neurodegenerative disorders that are caused by the accumulation of misfolded prion protein (PrP) in the brain. There are currently no cures, and the disease is fatal within months or years after symptoms begin. The new drug candidate, developed by Broad Institute and University of Massachusetts Chan Medical School scientists, is a small interfering RNA (siRNA) that binds and snips RNA molecules encoding the prion protein, reducing the amount of the disease-causing protein in the brain. Previous research in animals has shown that lowering prion protein levels can delay onset and slow down the disease. The trial, called PrP-targeting siRNA Safety & Mechanism Study (PRiSM), is enrolling patients exhibiting symptoms of the disease.

Read [full article](https://www.broadinstitute.org/news/clinical-trial-prion-disease-drug-candidate-begins-enrolling-participants).



 

 

 



 

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