Developing a faster and cheaper way to produce personalized gene therapy products.
Platform for Rapid Expression, Purification, and Analysis of Patient-Specific Gene Therapy Products
This study is working on making it easier and cheaper to produce special viruses that help deliver gene therapies to patients, so they can get the treatments they need faster and more affordably.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | North Carolina State University Raleigh NIH-funded |
| Lab location | 1 site (Raleigh, United States) |
| Project ID | NIH-10694917 on NIH RePORTER |
What this research studies
This research focuses on improving the manufacturing process of adeno-associated viruses (AAVs), which are crucial for delivering gene therapies to patients. By integrating advanced purification and sensing technologies, the project aims to make the production of patient-specific AAVs more efficient and affordable. The approach involves using specialized materials to isolate AAVs and advanced sensors to measure their effectiveness, ultimately reducing costs and wait times for patients needing these therapies.
Who could benefit from this research
Good fit: Ideal candidates for this research are patients who require gene therapy and would benefit from more affordable and rapidly produced treatment options.
Not a fit: Patients who do not require gene therapy or those whose conditions are not addressed by AAV-based treatments may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could significantly lower the cost and improve the accessibility of personalized gene therapies for patients.
How similar studies have performed: Other research has shown promise in improving biomanufacturing processes for gene therapies, indicating that this approach could lead to significant advancements.
Where this research is happening
Raleigh, United States
- North Carolina State University Raleigh — Raleigh, United States (Active)
Researchers
- Principal investigator: Daniele, Michael — North Carolina State University Raleigh
- Study coordinator: Daniele, Michael
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.