Developing gene therapy treatments for specific metabolic disorders.
REGULATORY SUPPORT FOR LEAD GENE THERAPY CANDIDATES AS A TREATMENT FOR ORGANIC ACIDEMIAS, PROPIONIC ACIDEMIA (PA) AND METHYLMALONIC ACIDEMIA TYPE B (MMAB)
This study is working on a plan to help develop new gene therapies for people with Propionic Acidemia and Methylmalonic Acidemia Type B, so that one day, patients can have access to better treatments that tackle the root causes of their conditions.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Project ID | NIH-10953994 on NIH RePORTER |
What this research studies
This research focuses on creating a regulatory framework to support the development of gene therapy candidates aimed at treating organic acidemias, specifically Propionic Acidemia and Methylmalonic Acidemia Type B. The approach involves meticulous planning and preparation for interactions with the FDA, including meetings and submissions necessary for advancing these therapies. Patients may benefit from the eventual availability of innovative treatments that target the underlying genetic causes of their conditions.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with Propionic Acidemia or Methylmalonic Acidemia Type B.
Not a fit: Patients with other types of metabolic disorders not targeted by this research may not receive any benefit.
Why it matters
Potential benefit: If successful, this research could lead to new gene therapy options that effectively treat metabolic disorders.
How similar studies have performed: While gene therapy is a rapidly evolving field, this specific regulatory approach for these conditions is relatively novel and untested.
Where this research is happening
Gaithersburg, United States
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.