Analyzing gene therapy candidates for rare diseases
Official title: INSILICO AND EX-VIVO IMMUNOGENICITY ANALYSIS OF NCATS GENE THERAPY TEST
Funded at Leidos Biomedical Research, INC. · NIH-10891948 · Data updated 2026-11-12
This study is looking at new gene therapies for rare diseases to make sure they are safe and effective for patients by understanding how they might affect the immune system before testing them in people.
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 | 2023 |
| Award (this year) | $442,590 |
| NIH project number | 75N91019D00024-0-759102300009-1 |
| Project ID | NIH-10891948 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 interventions.
✕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 the development and evaluation of gene therapy products specifically designed for rare diseases. It employs advanced computational (in-silico) and laboratory (ex-vivo) techniques to assess the immunogenicity of these gene therapy candidates, which helps to identify potential risks before they are tested in humans. By understanding how these therapies interact with the immune system, the research aims to improve the safety and effectiveness of gene therapies for patients with rare conditions.
Why it matters
Potential benefit: If successful, this research could lead to safer and more effective gene therapies for patients suffering from rare diseases.
How similar studies have gone: Other research initiatives in gene therapy have shown promising results, indicating that this approach has the potential for significant advancements in treatment.
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
Related research
Other NIH-funded projects on the same topics.
-
Improving public health responses to COVID-19 through standardized testing.
This study is looking to improve how we fight COVID-19 by testing blood samples to see if people have had the virus, and it’s designed for anyone interested in better ways to manage the disease.
-
Creating a large library of cancer models for research use
This study is creating a collection of new cancer models to help researchers and companies find better ways to treat cancer, making it easier for everyone to work together and share ideas.
-
Advancing global cancer control through data analysis and international collaboration
This study is all about helping countries around the world work better together to fight cancer by sharing important information and training scientists, especially in places that need extra support.
-
Support for HIV and HPV cancer prevention clinical trials in the Americas
This study is working on new vaccines and treatments to help prevent cancers related to HIV and HPV, making sure that patients in the US, Latin America, and the Caribbean can access better options as these exciting trials move forward.
-
Development and production of clinical-grade biopharmaceuticals
This study is all about making safe and effective new medicines, like vaccines and treatments for various conditions, so that patients can have access to the latest therapies that meet high-quality standards.
-
Understanding the immune response to COVID-19
This study is looking for ways to improve COVID-19 testing and understand how our immune system fights the virus, and you can help by sharing samples to make these tests better for everyone.
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.