Developing a new treatment for prion disease using siRNA technology
Official title: Regulatory Consulting to Support Advancement of a Divalent siRNA for Prion Disease
Funded at Broad Institute, INC. · NIH-11194089 · Data updated 2026-11-12
This study is working on a new treatment for prion disease that could help lower the harmful protein levels in the body, and it's getting ready to ask the FDA for permission to start testing it in people.
Quick facts
| Grant type | U01 cooperative agreement |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Broad Institute, INC. NIH-funded |
| Lab location | 1 site (Cambridge, United States) |
| Project start | August 1, 2023 |
| Project end | July 31, 2025 |
| Funding year | 2025 |
| Award (this year) | $98,280 |
| NIH project number | 3U01NS132994-02S1 |
| Project ID | NIH-11194089 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with prion disease or those at risk of developing it.
✕Less likely to help
Patients with other neurological disorders unrelated to prion disease may not benefit from this research.
What this research studies
This research aims to advance a novel therapeutic approach for prion disease by filing an Investigational New Drug (IND) application with the FDA. The focus is on a divalent siRNA that lowers the levels of the prion protein, which is implicated in the disease. Regulatory consultants will be engaged to ensure that the IND application is thorough and meets all necessary compliance standards, increasing the likelihood of successful clinical trials. If successful, this could lead to new treatment options for patients suffering from this challenging condition.
Why it matters
Potential benefit: If successful, this research could provide a new therapeutic option for patients with prion disease, potentially improving their outcomes.
How similar studies have gone: While the approach of using siRNA technology is promising, it is still relatively novel in the context of prion disease, and similar studies have shown potential but are not yet widely established.
Where this research is happening
-
Broad Institute, INC.
Cambridge, United States Active
Researchers
- Principal investigator: Minikel, Eric Vallabh — Broad Institute, INC.
- Study coordinator: Minikel, Eric Vallabh
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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.