Developing a new method to target RNA for treating certain neurological diseases
Cell-based RNA degradation assay for C9ALS/FTD drug discovery
['FUNDING_SBIR_1'] · LUCERNA, INC. · NIH-10822574
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
| Phase | ['FUNDING_SBIR_1'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | LUCERNA, INC. (nih funded) |
| Locations | 1 site (Brooklyn, UNITED STATES) |
| Trial ID | NIH-10822574 on ClinicalTrials.gov |
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.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with C9ALS or frontotemporal dementia (FTD).
Not a fit: Patients with conditions unrelated to RNA degradation or those not diagnosed with C9ALS or FTD may not benefit from this research.
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 performed: 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
Brooklyn, UNITED STATES
- LUCERNA, INC. — Brooklyn, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: O'HANLON, RYAN — LUCERNA, INC.
- Study coordinator: O'HANLON, RYAN
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.
Conditions: Disorder