Lowering the prion protein (PrP) to treat prion disease
Therapeutic Editing to Lower PrP in Prion Disease
This work tests a one-time gene therapy delivered by a viral vector that aims to lower the harmful prion protein in people with prion disease.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Broad Institute, INC. NIH-funded |
| Lab location | 1 site (Cambridge, United States) |
| Project ID | NIH-11350476 on NIH RePORTER |
What this research studies
From a patient perspective, researchers are developing an experimental gene-based treatment that uses an engineered virus to turn down the PRNP gene and reduce PrP levels in the brain. The team is producing clinical-grade doses and completing the safety and manufacturing steps needed to file for FDA approval to treat people. The plan is to run a first-in-human trial for about 10 patients with prion disease using this novel epigenetic editor and a new viral capsid. If the human test shows it is safe and lowers PrP, the approach could be adapted for other central nervous system conditions.
Who could benefit from this research
Good fit: Ideal candidates are people with a confirmed diagnosis of prion disease who meet the study's eligibility and safety criteria and are able to travel to the trial site for treatment and follow-up.
Not a fit: People without prion disease, or those with very advanced brain damage or medical conditions that make gene therapy unsafe, are unlikely to benefit from this trial.
Why it matters
Potential benefit: If successful, lowering PrP could slow or stop disease progression in people with prion disease and potentially improve survival or function.
How similar studies have performed: AAV-based gene therapies have had successes in other disorders, but this specific PrP-targeting epigenetic editor and the BI-hTfRv2 capsid are new and would be first-in-human uses.
Where this research is happening
Cambridge, United States
- Broad Institute, INC. — Cambridge, United States (Active)
Researchers
- Principal investigator: Vallabh, Sonia Minikel — Broad Institute, INC.
- Study coordinator: Vallabh, Sonia Minikel
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page below.