SGPL1 gene therapy to reduce lung scarring in idiopathic pulmonary fibrosis
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-11187012 · Data updated 2026-11-12
This project uses a one-time viral gene delivery to boost SGPL1 in the lungs and lower sphingosine-1-phosphate to help people with idiopathic pulmonary fibrosis breathe better.
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 | 2025 |
| Award (this year) | $565,250 |
| NIH project number | 5R61HL171786-02 |
| Project ID | NIH-11187012 on NIH RePORTER |
Who could benefit from this research
✓Could help
People diagnosed with idiopathic pulmonary fibrosis who meet safety and eligibility criteria and can travel to a participating center would be the most likely candidates.
✕Less likely to help
Patients with other causes of lung fibrosis, very advanced disease not amenable to intervention, or those with contraindications to viral gene therapy may not receive benefit.
What this research studies
Idiopathic pulmonary fibrosis causes progressive scarring of the lungs, and this project aims to deliver the SGPL1 gene using an adeno-associated virus (AAV9) to increase the enzyme that breaks down sphingosine-1-phosphate (S1P). The team will administer AAV9-SGPL1 intravenously in preclinical models and measure effects on lung fibrosis, inflammation, and profibrotic signaling to build safety and efficacy data. The approach is intended to reduce S1P-driven fibrosis without the long-term immune suppression linked to some S1P receptor drugs. If results support safety and benefit, the researchers plan to advance toward human testing at their clinical center.
Why it matters
Potential benefit: If successful, this could reduce or reverse lung scarring and improve lung function, offering a potential one-time treatment beyond current medicines or transplantation.
How similar studies have gone: Drugs that target S1P receptors exist and gene therapy for lung diseases is emerging, but using AAV to increase SGPL1 is a novel approach that is largely untested in humans.
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
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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.