First-Ever human trial launches for devastating brain disease treatment
Disease control
Not yet recruiting
This is the first study in people to test the safety of a new drug called PrP-siRNA for those already showing symptoms of prion disease, a rare and fatal brain condition. Researchers will give a single injection of the drug into the spinal fluid and monitor 30 participants for 24…
Phase: PHASE1 • Sponsor: Broad Institute of MIT and Harvard • Aim: Disease control
Last updated Mar 27, 2026 12:39 UTC