Analyzing gene therapy candidates for rare diseases
INSILICO AND EX-VIVO IMMUNOGENICITY ANALYSIS OF NCATS GENE THERAPY TEST
['FUNDING_OTHER'] · LEIDOS BIOMEDICAL RESEARCH, INC. · NIH-10891948
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
| Phase | ['FUNDING_OTHER'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | LEIDOS BIOMEDICAL RESEARCH, INC. (nih funded) |
| Locations | 1 site (FREDERICK, UNITED STATES) |
| Trial ID | NIH-10891948 on ClinicalTrials.gov |
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.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with rare diseases that may benefit from gene therapy interventions.
Not a fit: Patients with common diseases or those not eligible for gene therapy may not receive any benefit from this research.
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 performed: 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
FREDERICK, UNITED STATES
- LEIDOS BIOMEDICAL RESEARCH, INC. — FREDERICK, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: MOORE, MAX — LEIDOS BIOMEDICAL RESEARCH, INC.
- Study coordinator: MOORE, MAX
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.