Developing a new method to target RNA for treating certain neurological diseases
Official title: Cell-based RNA degradation assay for C9ALS/FTD drug discovery
Funded at Lucerna, INC. · NIH-10822574 · Data updated 2026-11-12
This study is working on a new way to find drugs that can specifically target harmful RNA molecules linked to diseases like C9ALS and FTD, which could lead to better treatments for patients.
Quick facts
| Grant type | Sbir 1 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Lucerna, INC. NIH-funded |
| Lab location | 1 site (Brooklyn, UNITED STATES) |
| Project start | December 1, 2023 |
| Project end | November 30, 2025 |
| Funding year | 2024 |
| Award (this year) | $392,116 |
| NIH project number | 1R43AG085837-01 |
| Project ID | NIH-10822574 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with C9ALS or frontotemporal dementia (FTD).
✕Less likely to help
Patients with conditions unrelated to RNA degradation or those not diagnosed with C9ALS or FTD may not benefit from this research.
What this research studies
This research focuses on creating a novel assay to help discover drugs that can target RNA molecules involved in diseases like C9ALS and FTD. By using innovative techniques to control RNA degradation, the study aims to overcome challenges associated with traditional drug discovery methods that focus on proteins. Patients may benefit from new therapies that specifically target the underlying RNA mechanisms contributing to their conditions. The approach includes high-throughput screening to identify effective compounds that can selectively degrade harmful RNA.
Why it matters
Potential benefit: If successful, this research could lead to new treatments for patients with C9ALS and FTD by targeting the RNA that contributes to these diseases.
How similar studies have gone: While the approach of targeting RNA for drug discovery is relatively novel, there have been some promising early-stage studies exploring RNA modulation in other contexts.
Where this research is happening
-
Lucerna, INC.
Brooklyn, United States Active
Researchers
- Principal investigator: O'hanlon, Ryan — Lucerna, INC.
- Study coordinator: O'hanlon, Ryan
Related research
Other NIH-funded projects on the same topics.
-
Developing a new method to find antiviral drugs by targeting RNA
This study is looking for new antiviral medicines that can safely target and break down the RNA of viruses, helping to create treatments that work better and are less likely to become ineffective over time.
-
Developing a sensor to help discover drugs for diseases like Alzheimer's and Parkinson's.
This study is working on a new way to find potential drugs for conditions like Alzheimer's and Parkinson's by looking for specific RNA structures in the brain, which could help develop treatments that slow down or stop the diseases.
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.