Gene therapy to treat hereditary angioedema
Official title: Safety of AAV-mediated C1 Esterase Inhibitor Gene Therapy for Hereditary Angioedema
Funded at Enyx Therapeutics, LLC · NIH-10818251 · Data updated 2026-11-12
This study is testing a new gene therapy that could help people with hereditary angioedema by using a modified virus to deliver a gene that makes a missing protein, aiming to reduce swelling attacks and keep you feeling better for a longer time.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Enyx Therapeutics, LLC NIH-funded |
| Lab location | 1 site (New York, United States) |
| Project start | March 15, 2024 |
| Project end | November 30, 2025 |
| Funding year | 2024 |
| Award (this year) | $275,737 |
| NIH project number | 1R41AI181097-01 |
| Project ID | NIH-10818251 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with hereditary angioedema who experience recurrent swelling episodes.
✕Less likely to help
Patients who do not have hereditary angioedema or those with other forms of angioedema may not benefit from this research.
What this research studies
This research investigates a new gene therapy approach for hereditary angioedema (HAE), a serious condition that causes recurrent swelling in various body tissues. The therapy aims to restore the function of a protein called C1-esterase inhibitor, which is deficient in patients with HAE. By using a modified virus to deliver a copy of the gene responsible for producing this protein, the treatment seeks to provide long-lasting protection against angioedema attacks. The research is being conducted in collaboration with a biotechnology company and aims to ensure the safety of this innovative therapy in humans.
Why it matters
Potential benefit: If successful, this research could significantly reduce or eliminate the frequency of angioedema attacks in patients with hereditary angioedema.
How similar studies have gone: Previous research has shown promising results with similar gene therapy approaches in animal models, indicating potential for success in human applications.
Where this research is happening
-
Enyx Therapeutics, LLC
New York, United States Active
Researchers
- Principal investigator: Crystal, Ronald G — Enyx Therapeutics, LLC
- Study coordinator: Crystal, Ronald G
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.