Developing gene therapy for a rare metabolic disorder.
CMC SUPPORT FOR NCATS RARE DISEASE PLATFORM GENE THERAPY TRIAL PROJECTS
This study is working on a new gene therapy for people with methylmalonic acidemia (MMA) to help treat the condition by fixing the genetic issues behind it, and it aims to create a reliable way to produce the therapy for future clinical trials.
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-11196049 on NIH RePORTER |
What this research studies
This research focuses on creating and manufacturing gene therapy products aimed at treating methylmalonic acidemia (MMA), a rare metabolic disorder. The project involves developing a scalable process for producing AAV9 gene vectors, which are essential for delivering the gene therapy. The research will also ensure that the necessary preclinical and clinical materials are prepared to support upcoming clinical trials. Patients may benefit from innovative treatments that address the underlying genetic causes of their condition.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with methylmalonic acidemia or related rare metabolic disorders.
Not a fit: Patients with conditions unrelated to methylmalonic acidemia may not receive any benefit from this research.
Why it matters
Potential benefit: If successful, this research could provide a new gene therapy option for patients suffering from methylmalonic acidemia.
How similar studies have performed: Previous research in gene therapy for rare diseases has shown promising results, indicating potential for success in this area.
Where this research is happening
Frederick, United States
- Leidos Biomedical Research, INC. — Frederick, United States (Active)
Researchers
- Principal investigator: Mitra, George — Leidos Biomedical Research, INC.
- Study coordinator: Mitra, George
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.