Analyzing the immune response to gene therapy for rare diseases
Official title: INSILICO AND EX-VIVO IMMUNOGENICITY ANALYSIS OF NCATS GENE THERAPY TEST
Funded at Leidos Biomedical Research, INC. · NIH-11196158 · Data updated 2026-11-12
This study is looking at how well new gene therapies for rare diseases work with the immune system, using computer models and lab tests, to make sure they are safe and effective before being given to patients like you.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Leidos Biomedical Research, INC. NIH-funded |
| Lab location | 1 site (Frederick, United States) |
| Project start | July 3, 2023 |
| Project end | July 2, 2026 |
| Funding year | 2024 |
| Award (this year) | $564,711 |
| NIH project number | 75N91019D00024-P00002-759102300009-1 |
| Project ID | NIH-11196158 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with rare diseases that may benefit from gene therapy.
✕Less likely to help
Patients with common diseases or those not eligible for gene therapy may not receive any benefit from this research.
What this research studies
This research focuses on evaluating the immune response to gene therapy products designed for rare diseases. It employs both in-silico (computer-based) and ex-vivo (lab-based) analyses to assess how these therapies might perform in human subjects. By identifying potential risks early in the development process, the research aims to improve the safety and effectiveness of gene therapies before they are tested in patients. This approach is crucial for ensuring that new treatments can be safely administered to individuals with rare conditions.
Why it matters
Potential benefit: If successful, this research could lead to safer and more effective gene therapies for patients with rare diseases.
How similar studies have gone: Previous research has shown promise in using similar in-silico and ex-vivo approaches to evaluate gene therapies, indicating a potential for success in this area.
Where this research is happening
-
Leidos Biomedical Research, INC.
Frederick, United States Active
Researchers
- Principal investigator: Moore, Max — Leidos Biomedical Research, INC.
- Study coordinator: Moore, Max
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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.