Universal approach to block inhibitors in protein replacement therapy for AAV-based treatments
Official title: Development of a universal approach to block inhibitors for protein replacement therapy
Funded at Nabgen, INC. · NIH-11311027 · Data updated 2026-11-12
A new approach aims to block immune inhibitors that reduce the effectiveness of protein therapies used in conditions like hemophilia and certain storage diseases.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Nabgen, INC. NIH-funded |
| Lab location | 1 site (Durham, United States) |
| Project start | August 1, 2026 |
| Project end | July 31, 2027 |
| Funding year | 2026 |
| Award (this year) | $312,461 |
| NIH project number | 1R41TR006064-01 |
| Project ID | NIH-11311027 on NIH RePORTER |
Who could benefit from this research
✓Could help
Patients with conditions treated by protein replacement therapies who develop inhibitors/all antibodies that block treatment could potentially benefit in the future.
✕Less likely to help
Patients without inhibitor formation or those not receiving protein replacement therapy may not directly benefit.
What this research studies
NabGen is developing Protein M, a long-lasting, engineered protein derived from mycoplasma, to block alloantibodies that interfere with protein replacement therapies. The project focuses on using this blocker alongside AAV-based gene delivery to improve the activity of infused biologics by preventing inhibitor formation. The research includes preclinical data showing Protein M binds immunoglobulins broadly and can dramatically reduce neutralizing antibody activity in vitro and in animal models. If successful, this strategy could make protein therapies more reliable and reduce treatment costs and hospitalizations for patients with inhibitors.
Why it matters
Potential benefit: If successful, patients could experience more effective protein therapies with fewer immune-related complications and potentially lower treatment burdens.
How similar studies have gone: Preclinical results show substantial reductions in antibody activity when using Protein M, supporting further development in this area.
Where this research is happening
-
Nabgen, INC.
Durham, United States Active
Researchers
- Principal investigator: Li, Chengwen — Nabgen, INC.
- Study coordinator: Li, Chengwen
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.