Developing gene therapy for methylmalonic acidemia
CMC SUPPORT FOR NCATS RARE DISEASE PLATFORM GENE THERAPY TRIAL PROJECTS
['FUNDING_OTHER'] · LEIDOS BIOMEDICAL RESEARCH, INC. · NIH-10928692
This study is working on a new gene therapy to help people with methylmalonic acidemia (MMA) by creating a way to produce the treatment more easily, so that it can be tested in clinical trials and hopefully provide better options for managing this condition.
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-10928692 on ClinicalTrials.gov |
What this research studies
This research focuses on creating and manufacturing gene therapy products aimed at treating methylmalonic acidemia (MMA), a rare genetic disorder. The project involves developing a scalable process for producing AAV9 gene vectors and preparing clinical trial materials. By conducting necessary chemistry, manufacturing, and controls (CMC) tasks, the research aims to facilitate regulatory approvals and initiate clinical studies. 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 metabolic disorders.
Not a fit: Patients with conditions unrelated to methylmalonic acidemia may not 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 success for this approach.
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.