Optimizing combination therapies for rare CF variants
Official title: Rational optimization of combinatorial therapies for the treatment of rare cystic fibrosis variants
Funded at Purdue University · NIH-11333209 · Data updated 2026-11-12
A study at Purdue University aims to find better drug combinations to help people with rare cystic fibrosis mutations.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Purdue University NIH-funded |
| Lab location | 1 site (West Lafayette, United States) |
| Project start | September 1, 2023 |
| Project end | May 31, 2027 |
| Funding year | 2026 |
| Award (this year) | $679,809 |
| NIH project number | 5R01HL167046-04 |
| Project ID | NIH-11333209 on NIH RePORTER |
Who could benefit from this research
✓Could help
CF patients with rare or uncommon CFTR variants who may not currently benefit from standard therapies.
✕Less likely to help
Patients with CF caused by well-understood variants already well-managed by existing treatments may see limited incremental benefit.
What this research studies
This project focuses on improving how a mix of drugs works for CF patients who have uncommon CFTR mutations. Researchers will explore biochemistry and CRISPR-based approaches to identify combos that enhance CFTR production and function in cells. The goal is to broaden which CF genotypes can be treated effectively and reduce variability in patient responses. The work emphasizes translating laboratory findings into strategies that could reach patients who currently have fewer treatment options.
Why it matters
Potential benefit: If successful, the research could expand effective therapies to more people with rare CF mutations.
How similar studies have gone: Previous CF drug studies show combination therapies can greatly help many patients, but rare variants require new approaches which this project seeks to develop.
Where this research is happening
-
Purdue University
West Lafayette, United States Active
Researchers
- Principal investigator: Schlebach, Jonathan Patrick — Purdue University
- Study coordinator: Schlebach, Jonathan Patrick
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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.