Gene therapy to treat hereditary angioedema
Safety of AAV-mediated C1 Esterase Inhibitor Gene Therapy for Hereditary Angioedema
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 ID | NIH-10818251 on NIH RePORTER |
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.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with hereditary angioedema who experience recurrent swelling episodes.
Not a fit: Patients who do not have hereditary angioedema or those with other forms of angioedema may not benefit from this research.
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 performed: 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
New York, United States
- 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 below.