Creating a process to manufacture gene therapy candidates using AAV vectors
NCATS-DPI-22-0101 - CREATION OF AN OPEN-SOURCE PROCESS TO MANUFACTURE AAV VECTOR-BASED GENE THERAPY CANDIDATES FOR NON-COMMERCIAL CLINICAL STUDIES
This study is working on a new way to make special viruses that can help with gene therapy, which is important for treating certain conditions, and it aims to create a reliable process that can produce these viruses in large amounts while ensuring they are safe and effective for patients.
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 ID | NIH-11196042 on NIH RePORTER |
What this research studies
This research focuses on developing an open-access method for producing adeno-associated virus (AAV) vectors, which are essential for gene therapy. The project aims to generate at least two widely used AAV serotypes, utilizing a green fluorescent protein (GFP) gene and a gene of interest identified by the National Center for Advancing Translational Sciences (NCATS). The researchers will demonstrate the scalability of this manufacturing process in bioreactors, ensuring that the produced vectors meet quality standards for clinical use. Additionally, they will establish necessary testing methods to support regulatory applications for these therapies.
Who could benefit from this research
Good fit: Ideal candidates for this research are patients with genetic disorders that could potentially be treated with AAV-based gene therapies.
Not a fit: Patients with conditions not amenable to gene therapy or those who do not have access to the clinical trials may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to more accessible and effective gene therapies for various genetic conditions.
How similar studies have performed: Other research has shown success in developing AAV vectors for gene therapy, indicating that this approach is promising and builds on established methodologies.
Where this research is happening
Frederick, United States
- Leidos Biomedical Research, INC. — Frederick, United States (Active)
Researchers
- Principal investigator: Mangalampalli, Venkata — Leidos Biomedical Research, INC.
- Study coordinator: Mangalampalli, Venkata
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.