First human trial of RNA therapy for fatal brain disease begins
Disease control
Recruiting now
This early-stage trial tests a new drug called PrP-siRNA in 30 adults with symptomatic prion disease, a rare and fatal brain condition. The drug is given via a spinal injection and aims to reduce levels of the harmful prion protein. The main goal is to check safety, but researche…
Phase: PHASE1 • Sponsor: Broad Institute of MIT and Harvard • Aim: Disease control
Last updated May 15, 2026 11:57 UTC