AAV gene therapy to fix the CFTR defect in cystic fibrosis
Official title: Cystic Fibrosis Gene Therapy with adeno-associated viral vectors
Funded at Johns Hopkins University · NIH-11312654 · Data updated 2026-11-12
A lung-targeted AAV gene therapy aims to deliver a corrected CFTR gene to help people with cystic fibrosis, including those whose mutations are not helped by existing drugs.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Johns Hopkins University NIH-funded |
| Lab location | 1 site (Baltimore, United States) |
| Project start | July 1, 2024 |
| Project end | April 30, 2029 |
| Funding year | 2026 |
| Award (this year) | $788,782 |
| NIH project number | 5R01HL167797-03 |
| Project ID | NIH-11312654 on NIH RePORTER |
Who could benefit from this research
✓Could help
People with cystic fibrosis of various CFTR mutations — particularly those not helped by current modulators — would be the intended candidates for eventual human testing.
✕Less likely to help
Those with advanced, irreversible lung damage or individuals with strong pre-existing immunity to the AAV vector that blocks delivery may not benefit from this approach.
What this research studies
Researchers plan to use a lung-tropic AAV1 viral vector carrying a shortened CFTR gene and a strong promoter to improve delivery to airway cells. They will first study safety and how the therapy behaves in the body using rhesus macaques and ferrets that carry the F508 CF mutation. The work builds on earlier AAV safety data and on recent advances in CFTR-correcting drugs, with the goal of a gene replacement approach that could work across many CF mutations. If these preclinical studies show acceptable safety and promising activity, the team would use that data to support future human trials.
Why it matters
Potential benefit: If successful, the approach could restore CFTR function across many mutations and substantially reduce lung infections and breathing problems for people with CF.
How similar studies have gone: Previous AAV-based CF trials have shown safety but not consistent clinical benefit, so this program uses newer vector choices and a shortened CFTR to try to improve on past results.
Where this research is happening
-
Johns Hopkins University
Baltimore, United States Active
Researchers
- Principal investigator: Cebotaru, Liudmila — Johns Hopkins University
- Study coordinator: Cebotaru, Liudmila
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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.