Therapeutic NR1H3 ASO to reprogram microglia for ALS/FTD
Official title: The Therapeutic Development of a Clinical NR1H3 Antisense Oligonucleotide to Reprogram Microglia into a Neuroprotective State in ALS and FTD
Funded at Modulo Bio, INC. · NIH-11409366 · Data updated 2026-11-12
A targeted antisense therapy aims to shift brain immune cells to a protective state to slow ALS and FTD in patients.
Quick facts
| Grant type | Sbir 2 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Modulo Bio, INC. NIH-funded |
| Lab location | 1 site (San Diego, UNITED STATES) |
| Project start | September 21, 2026 |
| Project end | August 31, 2028 |
| Funding year | 2026 |
| Award (this year) | $847,695 |
| NIH project number | 1R44NS143633-01A1 |
| Project ID | NIH-11409366 on NIH RePORTER |
Who could benefit from this research
✓Could help
Patients with ALS or related frontotemporal degeneration who could participate in future trials of ASO therapies or contribute samples for validation.
✕Less likely to help
Individuals without ALS/FTD or without eligibility for translational ASO trials may not benefit.
What this research studies
Researchers are developing a short RNA-based treatment that lowers NR1H3 in microglia to convert them from a harmful to a protective state in ALS and FTD. They use patient-derived cell models and animal studies to show the approach can reduce neurodegeneration. The project also addresses brain-penetrant ways to deliver this therapy safely in people who have ALS or FTD.
Why it matters
Potential benefit: If successful, this therapy could slow disease progression and protect neurons in ALS and related frontotemporal disorders.
How similar studies have gone: Early models suggest reprogramming microglia can alter neurodegeneration; translation to humans remains investigational.
Where this research is happening
-
Modulo Bio, INC.
San Diego, United States Active
Researchers
- Principal investigator: Lim, Ryan Gar-Lok — Modulo Bio, INC.
- Study coordinator: Lim, Ryan Gar-Lok
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.