Stopping antibodies that block AAV gene therapy
Novel strategy to block AAV neutralizing antibody activity
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 ID | NIH-11256166 on NIH RePORTER |
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.
Who could benefit from this research
Good fit: Patients who are candidates for AAV-based gene therapies but have pre-existing neutralizing antibodies against AAV.
Not a fit: People who are not seeking AAV-based treatments or who have other contraindications to gene therapy would not directly benefit from this work.
Why it matters
Potential benefit: Could allow people with pre-existing AAV antibodies to receive effective AAV-based gene therapies.
How similar studies have performed: 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
Durham, United States
- Nabgen, INC. — Durham, United States (Active)
Researchers
- Principal investigator: Askew, Charles H — Nabgen, INC.
- Study coordinator: Askew, Charles H
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 below.