Gene therapy to treat idiopathic pulmonary fibrosis by reducing harmful signaling in the lungs
Official title: Adeno-associated virus-mediated SGPL1 gene therapy as a first-in-class treatment for idiopathic pulmonary fibrosis that acts by reducing sphingosine-1-phosphate and downstream profibrotic signaling
Funded at University of California, San Francisco · NIH-10793257 · Data updated 2026-11-12
This study is testing a new gene therapy that aims to help people with idiopathic pulmonary fibrosis (IPF) by using a virus to deliver a gene that can reduce lung inflammation and scarring, which could lead to better lung function and a new treatment option for patients.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California, San Francisco NIH-funded |
| Lab location | 1 site (San Francisco, United States) |
| Project start | September 6, 2024 |
| Project end | July 31, 2026 |
| Funding year | 2024 |
| Award (this year) | $565,250 |
| NIH project number | 1R61HL171786-01 |
| Project ID | NIH-10793257 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with idiopathic pulmonary fibrosis who are experiencing progressive lung dysfunction.
✕Less likely to help
Patients with other forms of lung disease or those who are not diagnosed with idiopathic pulmonary fibrosis may not benefit from this research.
What this research studies
This research investigates a novel gene therapy approach for idiopathic pulmonary fibrosis (IPF), a serious lung disease that leads to progressive lung dysfunction. The therapy uses an adeno-associated virus to deliver the SGPL1 gene, which helps break down a harmful lipid called sphingosine-1-phosphate (S1P) that contributes to lung inflammation and fibrosis. By enhancing the activity of S1P lyase, the therapy aims to reduce fibrosis and improve lung function, potentially offering a new treatment option for patients with IPF. The study will involve assessing the safety and effectiveness of this gene therapy in a clinical setting.
Why it matters
Potential benefit: If successful, this research could provide a groundbreaking treatment option that may cure idiopathic pulmonary fibrosis and significantly improve patient outcomes.
How similar studies have gone: While targeting sphingosine-1-phosphate has shown promise in other conditions, this specific gene therapy approach for IPF is novel and has not been extensively tested in clinical settings.
Where this research is happening
-
University of California, San Francisco
San Francisco, United States Active
Researchers
- Principal investigator: Saba, Julie D — University of California, San Francisco
- Study coordinator: Saba, Julie D
Related research
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Repurposing Pyrvinium as an Inhaled Therapy for Idiopathic Pulmonary Fibrosis
A patient-friendly look at a project trying to use an existing drug, pyrvinium, as an inhaled treatment option for idiopathic pulmonary fibrosis (IPF).
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Screening new compounds to reverse gene signatures in IPF lungs
A researcher hopes to find compounds that reverse harmful gene changes in the lungs of people with idiopathic pulmonary fibrosis (IPF).
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Unraveling IPF subtypes to guide prognosis and therapy
This grant aims to identify different IPF subtypes in the lung to improve how we predict outcomes and tailor treatments for people with IPF.
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Novel antibody therapy to treat idiopathic pulmonary fibrosis
A small business gig is developing a targeted antibody drug (VSR-H5) to treat idiopathic pulmonary fibrosis in patients, aiming for better safety and effectiveness than current medicines.
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Repurposing Vonoprazan as an inhaled therapy for IPF
A high-profile NIH grant is testing whether an existing drug, vonoprazan, can be inhaled to slow or reverse lung fibrosis in people with idiopathic pulmonary fibrosis (IPF).
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Proteomic profile of IPF progression to help tailor treatments
A study will look for blood protein patterns that predict how IPF lung disease will progress in patients.
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.