Exploring a new drug for treating cystic fibrosis
Official title: Therapeutic potential of CC-90009 for cystic fibrosis
Funded at Univ of North Carolina Chapel Hill · NIH-10826733 · Data updated 2026-11-12
This study is looking at a new drug called CC-90009 to see if it can help people with cystic fibrosis who have certain genetic mutations that stop their bodies from making a key protein, with the hope that it can improve lung function and help clear mucus better.
Quick facts
| Grant type | Fellowship grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Univ of North Carolina Chapel Hill NIH-funded |
| Lab location | 1 site (Chapel Hill, United States) |
| Project start | September 1, 2024 |
| Project end | August 31, 2029 |
| Funding year | 2024 |
| Award (this year) | $40,620 |
| NIH project number | 1F30HL172628-01 |
| Project ID | NIH-10826733 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are cystic fibrosis patients with class I CFTR mutations who are currently ineligible for existing CFTR modulator therapies.
✕Less likely to help
Patients with cystic fibrosis who do not have class I CFTR mutations or those who are already responding well to current CFTR modulators may not benefit from this research.
What this research studies
This research investigates the potential of CC-90009, a small-molecule drug, to treat cystic fibrosis (CF) patients with specific genetic mutations that prevent the production of the CFTR protein. The study focuses on how CC-90009 can rescue the function of defective CFTR variants and reduce the activity of the epithelial sodium channel (ENaC), which may help improve mucus hydration and mucociliary clearance in the lungs. By using primary human airway epithelial cells, the research aims to understand the drug's effects and its ability to address the unmet needs of patients who currently have no treatment options due to their genetic profile.
Why it matters
Potential benefit: If successful, this research could provide a new therapeutic option for cystic fibrosis patients with class I CFTR mutations, improving their lung function and quality of life.
How similar studies have gone: While CC-90009 is currently being tested for acute myeloid leukemia, its application for cystic fibrosis represents a novel approach that has not been extensively studied in this context.
Where this research is happening
-
Univ of North Carolina Chapel Hill
Chapel Hill, United States Active
Researchers
- Principal investigator: Bulik-Sullivan, Emily Claire — Univ of North Carolina Chapel Hill
- Study coordinator: Bulik-Sullivan, Emily Claire
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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.