Stopping antibodies that block AAV gene therapy
Official title: Novel strategy to block AAV neutralizing antibody activity
Funded at Nabgen, INC. · NIH-11256166 · Data updated 2026-11-12
A protein-based approach aims to block the antibodies that stop AAV gene therapies so more patients can benefit from AAV treatments.
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 | April 1, 2023 |
| Project end | August 31, 2028 |
| Funding year | 2025 |
| Award (this year) | $993,865 |
| NIH project number | 2R42TR004338-02 |
| Project ID | NIH-11256166 on NIH RePORTER |
Who could benefit from this research
✓Could help
Patients who are candidates for AAV-based gene therapies but have pre-existing neutralizing antibodies against AAV.
✕Less likely to help
People who are not seeking AAV-based treatments or who have other contraindications to gene therapy would not directly benefit from this work.
What this research studies
This project is developing Protein-M, a protein that binds many kinds of antibodies and may prevent them from neutralizing AAV viral vectors used in gene therapy. The team is engineering a more stable version of Protein-M that remains active at body temperature and testing it in lab assays and animal models. Early work showed large reductions in antibody activity in vitro and in mice, and the current work focuses on improving stability and safety. If preclinical results remain strong, the approach could move toward human testing to help patients who currently cannot receive AAV therapies because of pre-existing antibodies.
Why it matters
Potential benefit: Could allow people with pre-existing AAV antibodies to receive effective AAV-based gene therapies.
How similar studies have gone: Other methods (different serotypes, capsid changes, plasmapheresis, B-cell depletion, IgG-cleaving enzymes) have had limited success or side effects, and this Protein-M approach is novel with promising preclinical data.
Where this research is happening
-
Nabgen, INC.
Durham, United States Active
Researchers
- Principal investigator: Askew, Charles H — Nabgen, INC.
- Study coordinator: Askew, Charles H
Related research
Other NIH-funded projects on the same topics.
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New way to block antibodies that stop AAV gene therapy
A new approach uses a special protein to block antibodies that prevent AAV gene therapies from working in people.
-
Universal approach to block inhibitors in protein replacement therapy for AAV-based treatments
A new approach aims to block immune inhibitors that reduce the effectiveness of protein therapies used in conditions like hemophilia and certain storage diseases.
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.