Engineered regulatory T cells as a therapy for Gaucher disease
Official title: Engineered Tregs as a therapy to Gaucher syndrome
Funded at Celdara Medical, LLC · NIH-11405182 · Data updated 2026-11-12
A new cell-based therapy using engineered regulatory T cells to help people with Gaucher disease, a type of lysosomal storage disorder.
Quick facts
| Grant type | Sbir 1 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Celdara Medical, LLC NIH-funded |
| Lab location | 1 site (Lebanon, United States) |
| Project start | July 1, 2026 |
| Project end | June 30, 2027 |
| Funding year | 2026 |
| Award (this year) | $310,564 |
| NIH project number | 1R43TR006062-01A1 |
| Project ID | NIH-11405182 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are individuals with Gaucher disease or related lysosomal storage disorders who have or are at risk for neurological involvement and are eligible for cell-based therapies.
✕Less likely to help
Patients without access to cell-based therapies or those with conditions not related to Gaucher disease may not benefit.
What this research studies
This project aims to develop a cell therapy using regulatory T cells that can cross the blood-brain barrier and reduce inflammation in Gaucher disease. Researchers will design and test these cells in preclinical models to understand their safety and how well they can reach affected tissues. If successful, this approach could become a future treatment option that helps lessen symptoms and improve quality of life for patients with Gaucher disease and related lysosomal storage disorders.
Why it matters
Potential benefit: If successful, patients could gain a safer, inflammation-reducing therapy that may complement or replace some current treatments.
How similar studies have gone: Similar cell-based approaches have shown promise in modulating immune responses in other diseases, though this exact therapy for Gaucher disease is investigational.
Where this research is happening
-
Celdara Medical, LLC
Lebanon, United States Active
Researchers
- Principal investigator: Murad, Joana M — Celdara Medical, LLC
- Study coordinator: Murad, Joana M
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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.