Removing antibodies that block AAV gene therapy
Official title: Protein depleting pre-existing antibodies for viral gene therapy
Funded at Neurogt, INC. · NIH-11164842 · Data updated 2026-11-12
A short-acting IV treatment that temporarily removes antibodies that stop AAV-based gene therapies so more people can get these treatments.
Quick facts
| Grant type | Sbir 2 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Neurogt, INC. NIH-funded |
| Lab location | 1 site (Chapel Hill, UNITED STATES) |
| Project start | August 1, 2023 |
| Project end | July 31, 2026 |
| Funding year | 2025 |
| Award (this year) | $969,817 |
| NIH project number | 5R44AI172670-03 |
| Project ID | NIH-11164842 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates would be people who are eligible for AAV-based gene therapy but are currently excluded because they have pre-existing anti-AAV antibodies.
✕Less likely to help
People without anti-AAV antibodies, those who are not candidates for AAV therapy for other medical reasons, or those who cannot tolerate an IV enzyme treatment may not benefit.
What this research studies
This project is developing an IV protein called AbC that uses an enzyme to cut specific IgG antibodies that bind AAV gene therapy vectors. Given before AAV delivery, the treatment would transiently clear anti-AAV antibodies to allow the viral vector to reach target cells. The team has tested the approach in rabbitized and mouse models and saw restored AAV transduction after treatment. The goal is to advance the product toward clinical use so patients who currently are excluded for having anti-AAV antibodies could become eligible.
Why it matters
Potential benefit: If successful, this could allow people with pre-existing anti-AAV antibodies to receive or be re-dosed with AAV gene therapies.
How similar studies have gone: Related IgG-cleaving enzymes such as IdeS (imlifidase) have shown transient IgG degradation in humans and animals without dose-limiting toxicity, and preclinical work restored AAV delivery in antibody-positive animal models.
Where this research is happening
-
Neurogt, INC.
Chapel Hill, United States Active
Researchers
- Principal investigator: Mccarty, Douglas M — Neurogt, INC.
- Study coordinator: Mccarty, Douglas M
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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.