First-in-human trial of siRNA drug for prion disease begins enrolling patients
Clinical trial of a prion disease drug candidate begins enrolling participants
A phase 1 clinical trial has started enrolling 15 patients with symptomatic prion disease to test a divalent siRNA that cuts production of the misfolded prion protein. Developed by Broad Institute and UMass Chan Medical School scientists, the drug previously lowered prion protein by 49% and extended survival by 64% in mice. The trial will assess safety and dosing, with the team committed to openly sharing all data.
To finally advance this drug to a human trial is the long-overdue achievement of a longstanding dream, but it's also the very beginning of learning about this drug's safety and activity in humans.