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 could be really helpful for people needing treatment for certain conditions, and they’ll be testing it using a green fluorescent protein and another important gene.
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-10716633 on NIH RePORTER |
What this research studies
This research focuses on developing an open-source method to produce adeno-associated virus (AAV) vectors, which are essential for gene therapy. The project aims to generate at least two widely used AAV serotypes and validate the process using a green fluorescent protein (GFP) gene and a gene of interest identified by NCATS. The researchers will demonstrate the scalability of this manufacturing process by producing the gene therapy candidates at both pilot and larger bioreactor scales. Additionally, they will establish necessary analytical methods to ensure the quality and stability of the produced materials for clinical use.
Who could benefit from this research
Good fit: Ideal candidates for this research are patients with genetic conditions that may benefit from AAV vector-based gene therapies.
Not a fit: Patients with conditions not addressed by AAV vector-based therapies may not receive any benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to more accessible and affordable gene therapies for patients with genetic disorders.
How similar studies have performed: Other research has shown success in developing AAV-based gene therapies, indicating that this approach has potential for significant advancements.
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.