The Broad Institute Announces First-in-Human Clinical Trial to Evaluate Potential New Drug: PrP-siRNA
We are glad to share a research update from the USA and the Broad Institute. The Institute, based in Cambridge Massachusetts, has announced that they have initiated a first-in-human clinical trial to evaluate an investigational drug called PrP-siRNA in patients diagnosed with prion disease. PrP-siRNA is a divalent siRNA drug administered by a lumbar puncture or spinal tap designed to lower the amount of PrP produced in the brain. PrP is the protein that causes prion disease.
From the Broad Institute:
“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,” said Eric Minikel, principal investigator of the trial and codirector of Broad’s Prion Therapeutic Science program. “I am looking forward to finding out whether this candidate has a future as a drug in our disease, but no matter what the outcome, as sponsor-investigators, we will learn a lot about how to run a clinical trial in prion disease, and we plan to broadly and publicly share our data and findings to benefit all sponsors who want to develop drugs for prion disease.”
More information can be found at both the Community Statement and FAQ kindly provided to all Patient Advocacy Organisations by the Institute (Community-Statement-2026-04), and at the study’s ClinicalTrials.gov listing.
We wish everyone involved all the best as this trial moves forward.
Note: The CJD Support Network cannot provide any additional information on this trial other than the information available to all above.
